340B Drug Program Contract Pharmacy Service Guidance

On March 5, 2010, the Health Resources and Services Administration (HRSA) published a final notice on 340B drug pricing program contract pharmacy services. The notice announces the availability of final guidelines regarding the utilization of multiple contract pharmacies and suggested contract pharmacy provisions, which previously had been limited to the “Alternative Methods Demonstration Project” program. These final guidelines, which are effective April 5, 2010, replace all previous 340B program guidance documents addressing non-network contract pharmacy services.  Additional analysis is available on our Life Sciences Legal Update blog.  

House Energy & Commerce Drug Safety Hearing Set for March 10

The House Energy and Commerce Health Subcommittee has scheduled a hearing for March 10 on "Drug Safety: An Update from the FDA." At the hearing, the FDA will detail its current challenges and successes in the area of drug safety.  Joshua M. Sharfstein, M.D., FDA Principal Deputy Commissioner, is slated to testify. 

2010 HCPCS Public Meetings Announced

CMS has announced the dates for the 2010 Healthcare Common Procedure Coding System (HCPCS) public meetings, at which the agency will discuss its preliminary coding and payment determinations for all new public requests for revisions to the HCPCS. On May 4-5, CMS will review applications for codes for Drugs/Biologicals/Radiopharmaceuticals/Radiologic Imaging Agents; the May 25-26 sessions will review applications for Supplies; on May 27 CMS will focus on Orthotics and Prosthetics codes; and the June 8 session will review Durable Medical Equipment and Accessories coding applications. Draft agendas, including CMS' preliminary decision, will be posted on the HCPCS website at least 4 weeks before each meeting. 

FDA And NIH Announce Collaboration to Speed Patient Access to New Therapies

This post was written by Paul Sheives.

FDA and the National Institutes of Health (NIH) have announced a new initiative aimed at improving efficiencies in translational science and regulatory science to close the gap in time between discovery of new technology and availability of therapies to patients. A new entity established under the initiative, the Joint Leadership Council, will seek to increase the consideration of regulatory issues early in the development of new technologies and therapies. In addition, the agencies jointly will issue grants totaling almost $7 million to develop better approaches to evaluating safety and efficacy for the development of new technologies and therapies. Additional information is available here

Draft Guidance on Assessment of Abuse Potential of Drugs

This post was written by Paul Sheives.

FDA published a guidance document aimed at sponsors that are developing drug products with the potential for abuse that may need to be scheduled by the Drug Enforcement Agency (DEA) under the Controlled Substances Act.    Within the draft guidance document, FDA addresses both the definition of abuse potential and information on submitting an adequate abuse potential assessment and scheduling request – covering such topics as study design and administrative recommendations.  Comments should be submitted by March 29, 2010 to be considered when the FDA drafts its final guidance. 

Guidance on Complete Submissions for the Evaluation of Proprietary Names

This post was written by Paul Sheives.

To maximize the agency’s efforts to process submissions for the evaluation of proprietary names within the deadlines under the Prescription Drug User Fee Act (PDUFA), FDA issued a guidance document to assist manufacturers in making a complete submission, which is required for the PDUFA review clock to begin. FDA discusses the collection of information that the agency requires to assess: (1) the safety aspects of a proposed proprietary name in order to reduce medication errors, and (2) the promotional implications of a proposed proprietary name, to ensure compliance with other requirements for labeling and promotion using traditional FDA review methods.

CMS Manual Transmittal on Authorized Compendia for Off-Label Uses of Cancer Drugs

On January 30, CMS issued a Medicare Benefit Policy Manual transmittal entitled "Revision of Definition of Compendia as Authoritative Source for Use in the Determination of a Medically-Accepted Indication of Drugs/Biologicals Used Off-label in Anti-Cancer Chemotherapeutic Regimens." The transmittal makes changes to conform to the MIPPA compendia requirement that provides that effective January 1, 2010, no compendia may be included on the list of authorized compendia unless it has a publicly-transparent process for evaluating therapies and for identifying potential conflicts of interests.

FTC Report on Drug Company "Pay-for-Delay" Agreements

The Federal Trade Commission has released a “staff study” entitled “Pay for Delay: How Drug Company Pay-Offs Cost Consumers Billions.” The report examines agreements in patent litigation settlements in which a brand-name pharmaceutical company compensates a generic pharmaceutical company for delays in generic entry. According to the study, over the past six years, such agreements delayed generic entry by an average of 48 months. Over the next 10 years, the study concluded that pay-for-delay agreements will cost American consumers an estimated $35 billion ($3.5 billion per year). While the FTC intends to continue litigating pay-for-delay cases under antitrust laws, the report contends that “a legislative solution offers the quickest and clearest way to deter these agreements and obtain the benefits of generic competition for consumers.”

Proposed Rule Amending Informed Consent Disclosure

This post was written by Paul Sheives.

On December 29, 2009, the Food & Drug Administration Act (FDA) issued a proposed rule that would amend the informed consent regulations to require the addition of an element regarding disclosure of information to the National Institute of Health (NIH) clinical trials database. Under the Food and Drug Administration Amendments Act of 2007, sponsors of “applicable clinical trials” are required to submit information for listing in the NIH clinical trial database. The proposed rule would require all informed consents for such “applicable clinical trials” – including specified drug, biologic, and device clinical investigations -- to include language informing the subject of the potential disclosure of the de-identified data to the clinical trials database (www.clinicaltrials.gov). FDA is accepting comments on the proposal until March 1, 2010.

FDA Guidance Document: Planning for the Effects of High Absenteeism to Ensure Availability of Medically Necessary Drug Products

This post was written by Paul Sheives.

The FDA has issued a guidance document seeking to encourage the creation of contingency production plans for the manufacture of medically-necessary drug products and their components during emergencies that result in high absenteeism at production facilities. Medically-necessary products are those used to treat or prevent a serious disease or medical condition for which there is no other adequately available drug product that is judged by medical staff to be an appropriate substitute.  

CBO Report on Pharmaceutical Manufacturer Spending on Prescription Drug Promotion

The Congressional Budget Office has issued an Economic and Budget Issue Brief on "Promotional Spending for Prescription Drugs." The report discusses trends in how pharmaceutical manufacturers market to consumers and prescribers, in such areas as detailing, sponsorship of meetings, direct-to-consumer (DTC) advertising, and journal advertising. The report also discusses how market factors (e.g., size of the market, current competition in that market, and the time that has elapsed since FDA approval) affect a manufacturer’s decision to use marketing tools, particularly DTC advertising and detailing. The report does not make formal recommendations for policy changes.

Public Hearing on Promotion of FDA-Regulated Medical Products Using the Internet and Social Media Tools

This post was written by Paul Sheives.

The FDA recently held a hearing to help the agency determine how the statutory provisions, regulations, and policies concerning advertising and promotional labeling should be applied to product-related information on the internet and newer technologies. Although the hearing was only an information gathering exercise for FDA, FDA acknowledged the industry’s concern that the Internet is a different medium with unique concerns that must be addressed. 

FDA Issues Warning Letters to Web Site Operators

This post was written by Paul Sheives.

On November 19, 2009, FDA completed a coordinated and collaborative international effort intended to curb illegal actions involving medical products sold via the internet. The agency issued 22 warning letters to the operators of these web sites and notified internet service providers and domain name registrars that the web sites were selling products in violation of U.S. law. FDA noted that, in many cases, because of these violations, internet service providers and domain name registrars may have grounds to terminate the websites and suspend the use of domain names. 

FDA Guidance Documents

This post was written by Paul Sheives.

FDA released a number of final and draft guidance documents in November, including the following:

  • Draft Guidance for Industry on Assay Development for Immunogenicity Testing of Therapeutic Proteins;
  • Draft Guidance for Industry: Factors That Distinguish Liquid Dietary Supplements From Beverages, Considerations Regarding Novel Ingredients, and Labeling for Beverages and Other Conventional Foods;
  • Draft Guidance for Industry on Dosage Delivery Devices for Over-the-Counter Liquid Drug Products;
  • International Conference on Harmonisation; Draft Guidance on E7 Studies in Support of Special Populations; Geriatrics; Questions and Answers;
  • Guidance for Industry on Residual Solvents in Drug Products Marketed in the United States; and
  • Several final and draft guidance documents on tobacco products, including guidance on listing of tobacco ingredients, applications for modified risk tobacco products, and registration of owners and operators of tobacco product establishments.

FDA Public Meeting on ICH Genetic Toxicology Guidance

This post was written by Paul Sheives.

FDA is hosting a one-day public workshop on January 25, 2010 to seek constructive input from experts in the field of genetic toxicology on proposed changes to the International Conference on Harmonisation (ICH) guidance ‘‘S2(R1) Genotoxicity Testing and Data Interpretation for Pharmaceuticals Intended for Human Use’’ from March 2008.  

CMS Meetings on Applications for IPPS/OPPS New Medical Service/Technology Payments (Feb. 10)

CMS is hosting a town hall meeting on February 10, 2010 to discuss FY 2011 applications for add-on payments for new medical services and technologies under the hospital inpatient prospective payment system (IPPS).  Also on February 10, CMS is hosting a workshop on the application process and criteria for new medical services and technologies under the IPPS and on the outpatient prospective payment system (OPPS) transitional pass-through payment for drugs, biologicals, and devices and new technology Ambulatory Payment Classification assignment for new services application processes.

AHRQ Review of Impact of Payment Policy on Clinical Trials

The Agency for Healthcare Research and Quality (AHRQ) has released a "Horizon Scan" entitled "To What Extent do Changes in Third-Party Payment Affect Clinical Trials and the Evidence Base?" The analysis examines the extent to which payment policies may be influencing clinical trial participant recruitment, participation, and retention rates   The report found that several large-scale clinical trials have encountered substantial difficulties due to the deterrent effect of payment policy on participation, with a bigger impact on medical device trials than drug trial. The report called for better coordination among all parties involved in trials, including a strategy covering which costs will be covered and which party will be responsible for such costs.

Generic Drug Competition

The Senate Judiciary Committee has approved S. 369, the Preserve Access to Affordable Generics Act, which generally would prohibit brand name drug companies from compensating generic drug companies to delay the entry of a generic drug into the market. As approved by the Committee, the bill would allow certain settlement agreements between drug companies if they enhance competition, and it would establish penalties for violations of the act. 

Medical Isotopes Production

On October 21, 2009, the House Commerce Committee approved H.R. 3276, the American Medical Isotopes Production Act. The legislation is intended to provide the Department of Energy with new authority and funding to assist private sector projects to establish a steady domestic supply of the medical isotope molybdenum-99.  

FDA Guidance Documents

This post was written by Paul Sheives.

FDA recently released two guidance documents (one in draft form) concerning hematopoietic reconstitution for specified indications as hematopoietic progenitor cells, cord (HPC-C), which provide information to manufacturers seeking licensure and potential sponsors for Investigational New Drugs Applications (INDs). FDA announced that it no longer intends to exercise enforcement discretion regarding IND and Biologics License Application (BLA) requirements for these products. The final guidance document is entitled “Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications,” and the draft guidance is entitled “Investigational New Drug Applications (INDs) for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications.” Other recent FDA guidance documents include the following:

Off-Label Cancer Therapies

The Agency for Healthcare Research and Quality (AHRQ) has released for public comment its draft Technology Assessment, "Report on the Evidence Regarding Off-Label Indications for Targeted Therapies used in Cancer Treatment." The document reviews the medical literature regarding 19 different drug/diseases indications that, at the time of their selection, were all off-label. The report concludes that for "some diseases, despite limited and/or ambiguous data, the use of an off-label indication may be a reasonable clinical decision." The report suggests additional research in this area.  Comments will be accepted until November 16, 2009 (extended from initial November 9 deadline). 

Congressional Hearings

A number of Congressional committees have held hearings recently on health policy issues, including the following:

Upcoming Hearings and Markups

On September 29, 2009, the House Oversight and Government Reform Committee is holding a hearing on the "Administration's Flu Vaccine Program: Health, Safety, and Distribution." On September 30, the Senate HELP Committee is scheduled to vote on the “Ryan White HIV/AIDS Treatment Extension Act of 2009.” Also on September 30, the Senate VA Committee is holding a hearing on VA's contracts for health servicesthe Aging Committee will examine how successful health systems keep costs low and quality highand the Senate Homeland Security Committee is holding a hearing on controlled substance abuse in Medicaid. On October 1, the Senate Judiciary Committee is scheduled to vote on S. 369, the "Preserve Access to Affordable Generics Act."

Current Good Manufacturing Practice Requirements for Combination Products

On September 23, 2009, the FDA published a proposed rule to codify current good manufacturing practice (cGMP) requirements that apply when drugs, devices, and biological products are combined to create a combination product. In addition, the proposed rule sets forth what the FDA characterizes as a “transparent and streamlined” regulatory framework for demonstrating compliance with cGMP requirements for “single-entity” and “co-packaged'” combination products. The FDA is accepting comments on the proposed rule until February 5, 2010 (extended from December 22, 2009).

Promotion of Medical Products Using the Internet and Social Media

The FDA is seeking public input on the promotion of FDA-regulated medical products (including prescription drugs and biologics and medical devices) using the internet and social media tools (e.g., blogs, microblogs, podcasts, social networks and online communities, video sharing, widgets, and wikis). To that end, the FDA is hosting a public hearing in Washington, D.C. on November 12-13, 2009 to discuss this topic, and the agency is soliciting public comments through February 28, 2010. In particular, the FDA is requesting input on questions such as: (1) for what online communications are manufacturers, packers, or distributors accountable; (2) how can such entities fulfill regulatory requirements in their internet and social media promotion, particularly when using tools that are associated with space limitations and tools that allow for real-time communications; (3) what parameters should apply to the posting of corrective information on web sites controlled by third parties; (4) when is the use of links appropriate; and (5) how do adverse event reporting requirements apply to these formats.  

FDA Guidance Documents

The FDA is accepting comments on the following new draft guidance documents for industry:

In addition, the FDA recently has issued a number of final guidance documents, including the following:

Pilot Program on Quality Information for Biotechnology Products

The FDA is announcing an extension of the deadline for submitting requests to participate in a pilot program involving the submission of quality (chemistry, manufacturing, and controls) information for biotechnology products in an Expanded Change Protocol consistent with the principles of quality-by-design and risk management in pharmaceutical manufacturing. FDA also is extending the application submission deadlines and increasing the number of applications being accepted into the pilot program. Requests to participate in the pilot program are due September 30, 2010, and investigational new drug applications and postapproval supplements are due March 31, 2011. 

FDA Issues Final Rules on Patient Access to Investigational Drugs

The Food and Drug Administration (FDA) has issued two final rules designed to further expand access to investigational drugs for treatment use and clarify when patients can be charged for investigational drugs.  The rules are effective October 13, 2009. The first rule, Expanded Access to Investigational Drugs for Treatment Use,” clarifies existing regulations and makes access to investigational drugs for treatment use easier for individual patients, including in emergencies; intermediate-size patient populations; and larger populations under a treatment protocol or treatment investigational new drug application (IND).  The FDA intends for the rule to “improve access to investigational drugs for patients with serious or immediately life-threatening diseases or conditions who lack other therapeutic options and who may benefit from such therapies.” The second final rule, “Charging for Investigational Drugs Under an Investigational New Drug Application,” clarifies the circumstances under which charging for an investigational drug in a clinical trial is appropriate; sets forth criteria for charging for an investigational drug for different population categories; and clarifies what costs can be recovered. The final rule will permit charging for a broader range of uses than was explicitly permitted previously. 

FDA Review of Post-Inspection Responses

On August 11, 2009, the FDA published a notice announcing a program to establish a deadline for inspected establishments to submit post-inspection responses to FDA 483 inspectional observations for the FDA's consideration in deciding whether to issue warning letters. According to the FDA, delayed and multiple responses to an FDA 483 have resulted in delays in the issuance of warning letters while these responses are reviewed and addressed. Under the program, the agency will not ordinarily delay the issuance of a warning letter in order to review a response to an FDA 483 that is received more than 15 business days after the FDA 483 was issued. The purpose of this program is to optimize resource utilization, facilitate the timely issuance of warning letters, and promote prompt correction of violations. FDA will assess the program after approximately 18 months to determine whether to make it permanent. The program will begin on September 15, 2009.  

FDA Targets Misconduct in Drug and Device Development

On August 7, 2009, the FDA announced its enhanced procedures for debarment and clinical investigator disqualification with, among other things, increased staffing and centralized coordination, to ensure more rapid, transparent and consistent actions. The FDA notes that in the short time these measures have been in effect, the number of debarment actions has risen considerably while the times for resolving disqualification and debarment actions have been reduced significantly. The FDA also is posting disqualification and debarment proceeding information on its website to provide clinical study sponsors with ready access to information about FDA's actions. These policy changes are in response to concerns expressed by members of Congress that the FDA has not adequately used its debarment and disqualification authorities.

Draft Guidance on Genomic Biomarkers

The FDA has published a notice announcing the availability of a draft guidance entitled E16 Genomic Biomarkers Related to Drug Response: Context, Structure, and Format of Qualification Submissions.” The FDA is requesting comments on the guidance by September 28, 2009.

NDA Reporting Requirements

On July 28, 2009, the FDA published a final rule requiring holders of new drug applications (NDAs) to submit certain information regarding authorized generic drugs in an annual report. The FDA is taking this action as part of its implementation of the Food and Drug Administration Amendments Act of 2007, which requires the FDA to provide updated listings of all authorized generic drugs. The rule is effective January 25, 2010. 

FDA Guidance Documents & FAQs

The FDA is accepting comments on the following new draft guidance documents for industry:

In addition, the FDA recently has issued a number of final guidance documents and “frequently asked question” documents, including the following:

Medicare Chemotherapy Administration Policy

The HHS Office of Inspector General (OIG) has issued a report on Medicare Part B Chemotherapy Administration: Payment and Policy.” The OIG points out that while questionable claims exceeded $60 million from 2005 to 2007, Medicare data are insufficient to determine consistently whether chemotherapy administration payments are appropriate. The OIG recommends that CMS: (1) establish a process to determine which specific drugs qualify for the chemotherapy administration payment rate, (2) instruct carriers that have not done so to consider a probe review of unmatched chemotherapy administration claims, and (3) ensure that drug administration claims are coded correctly and paid appropriately.

FTC Report on "Authorized Generic" Drugs

The Federal Trade Commission (FTC) has issued an interim report on the impact of authorized generic drugs on competition in the prescription drug marketplace. Authorized generics are drugs sold by a pharmaceutical manufacturer under both a brand-name and generic label. In short, the FTC found that retail drug prices average 4.2% lower when authorized generics are marketed against a single generic drug during the 180-day marketing exclusivity period than when they are not. Authorized generic entry during this period also reduces the revenues of a first-filer generic firm by 47% to 51% – much greater than the price decline for consumers. To prevent this loss of revenue, generic firms may agree to delay entry in return for a brand manufacturer’s agreement not to launch an authorized generic during the 180-day marketing exclusivity period. According to the report, between fiscal years 2004-08, about 25% of the final patent settlements reviewed by the FTC contained provisions related to authorized generics. 

FDA Comment Solicitation on Drug Advertising

On June 22, 2009, the FDA announced a public comment opportunity on its “Experimental Study of Presentation of Quantitative Effectiveness Information to Consumers in Direct-to-Consumer (DTC) Television and Print Advertisements for Prescription Drugs.” The study will examine: (1) various ways of communicating quantitative efficacy in DTC print ads and (2) whether the findings translate to DTC television ads. Comments will be accepted until August 21, 2009. 

FTC Issues Report on Follow-On Biological Drug Competition

A lengthy new FTC report on “Follow-on Biologic Drug Competition" issued June 10, 2009 concludes that:

  • Competition between a biologic drug and a follow-on biological (FOB) is much more likely to resemble brand-to-brand competition than the dynamics of brand-generic competition under Hatch-Waxman.
  • Existing incentives that support brand-to-brand competition among biologic drugs – patent protection and market-based pricing – are likely to be sufficient to support FOB competition and biologic innovation.
  • A 12-14-year exclusivity period is unnecessary to promote innovation by pioneer biologic drug manufacturers.
  • Special procedures to resolve patent issues between pioneer and FOB drug manufacturers prior to FDA approval are unnecessary and they could undermine patent incentives and harm consumers.
  • FOB drug manufacturers are unlikely to need additional incentives to develop interchangeable FOB products.

The FTC paper will be the subject of a June 11 Energy and Commerce Health Subcommittee hearing.

Waxman Urges President Obama to Use Pathway for Biogenerics Now

On June 8, 2009, House Energy and Commerce Committee Chairman Henry Waxman wrote to President Obama asking "the Administration to consider what steps can be taken under existing authority to prepare and even begin to use a pathway for generic biologics." The letter also requests that the "Administration provide an analysis of long-term savings generated from generic biologics not only for Medicare and Medicaid, but also for businesses, insurers, and families."

Compendia Conflicts of Interest

CMS has posted a white paper on "Potential Conflict of Interest in the Production of Drug Compendia" produced by the Duke Evidence-based Practice Center under contract to the Agency for Healthcare Research and Quality (AHRQ). The report addresses, among other things: where in the compendia processes conflict of interest may arise; an "ethical framework" for evaluating potential conflicts; a review of the conflict of interest policies of the four compendia used in Medicare coverage determinations; and a discussion of the adequacy of current compendia approaches and ways to minimize conflicts of interest.

FDA Guidance on Drug Establishment Registration and Drug Listing Submissions in Electronic Format

The FDA has issued final guidance on “Providing Regulatory Submissions in Electronic Format--Drug Establishment Registration and Drug Listing.'' As of June 1, 2009, FDA will only accept electronic submissions of drug establishment registration and drug listing information, unless a waiver is granted. This document provides guidance to manufacturers, repackers, and relabelers on the types of information to include for purposes of drug establishment registration and drug listing and on how to prepare and submit the information in an electronic format that FDA can process, review, and archive.

Draft FDA Guidance on Radioactive Drug Research

The FDA is seeking comments on a draft guidance document entitled “The Radioactive Drug Research Committee: Human Research Without an Investigational New Drug Application.” This draft guidance provides information to those using radioactive drugs for certain research purposes to help determine whether research studies may be conducted under an FDA-approved radioactive drug research committee, or whether research studies must be conducted under an investigational new drug application. It also offers answers to frequently asked questions on conducting research with radioactive drugs, and provides information on the membership, functions, and reporting requirements of a radioactive drug research committee approved by FDA. Comments received on the draft by September 1, 2009 will be considered by FDA when drafting the final guidance.

Product-Specific Bioequivalence (BE) Recommendations

On June 8, the FDA announced the availability of final product-specific BE recommendations, which provide product-specific guidance on the design of BE studies to support abbreviated new drug applications. The FDA also published a separate document proposing additional draft and revised draft product-specific BE recommendations; comments on the draft recommendations will be accepted until September 8, 2009.

Generic Drugs

On June 3, 2009, the Energy and Commerce Subcommittee on Commerce, Trade, and Consumer Protection approved H.R. 1706, the “Protecting Consumer Access to Generic Drugs Act,” which would prohibit a brand name drug company from paying a generic drug company to stay off the market as part of a drug patent settlement. The Subcommittee passed the legislation on a 16-10 vote, sending it to the full Committee for further action. In a related development, the Senate Judiciary Committee has scheduled a June 11, 2009 markup of S.369, the "Preserve Access to Affordable Generics Act".

Congressional Hearings

A number of Congressional panels have held hearings recently on health policy issues, including the following:

In addition, the following hearings are scheduled this month:

Generic Drug Patent Settlement Delays -- Hearing on June 3, 2009

On June 3, 2009, the House Judiciary Courts and Competition Policy Subcommittee is holding a hearing on "Pay to Delay: Are Patent Settlements That Delay Generic Drug Market Entry Anticompetitive?"

FDA Seeks Comments on Presenting Risk Information in Drug and Device Promotion

On May 27, 2009, the Food and Drug Administration (FDA) published a notice soliciting comments on a draft guidance document entitled Presenting Risk Information in Prescription Drug and Medical Device Promotion.”   The draft guidance proposes to use a “reasonable consumer” standard, similar to the Federal Trade Commission's standard, for assessing whether advertisements are misleading, a standard FDA already applies to labels on food and dietary supplements. In the draft guidance, FDA discusses the factors the agency would consider when evaluating prescription drug and restricted device promotional materials directed at healthcare professionals and consumers. The factors include: consistent and appropriate use of language, use of signals, framing of risk information, and the hierarchy of risk information. FDA also states that the agency will review content for both quantity of risk information, as well as materiality and comprehensiveness. Finally, FDA provides extensive factors it considers when evaluating the format of promotional materials.   Comments should be submitted by August 25, 2009; for information on submitting comments, click here.

FDA Guidance on FDA-Industry Meetings

The FDA has issued final guidance regarding formal meetings between FDA and sponsors or applicants relating to the development and review of drug or biological drug products by the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research (note that the guidance does not apply to abbreviated new drug applications). Specifically, the guidance discusses the principles of good meeting management practices and describes standardized procedures for requesting, preparing, scheduling, conducting, and documenting formal meetings. The document supersedes the guidance entitled “Formal Meetings With Sponsors and Applicants for PDUFA Products” published in February 2000. 

FDA Small Entity Guide on Drug Labeling

The FDA has released a compliance guidance entitled Labeling OTC Human Drug Products; Small Entity Compliance Guide.” The document, which finalizes draft guidance published in December 2004, is intended to help small businesses better understand and comply with the agency's over- the-counter (OTC) labeling requirements and to prepare new labeling.

OIG Report on Inhalation Drugs in South Florida.

According to a new OIG review, Medicare spent an average of five times more per beneficiary on inhalation drugs in South Florida compared to the rest of the country, with the greatest spending differences attributable to the more expensive brand name drugs levalbuterol and budesonide. In addition, three-fourths of South Florida beneficiaries receiving budesonide frequently exceeded coverage guidelines set forth in local coverage policy. The OIG recommends that CMS ensure that its contractors are enforcing the coverage guidelines for inhalation drugs, eliminate Medicare’s vulnerability to potentially fraudulent or excessive inhalation drug claims in South Florida, and review and act on cases where the DME supplier appears to be fraudulently billing Medicare for inhalation drugs.

Summary Bioequivalence Data for Abbreviated New Drug Applications (ANDA)

The Food and Drug Administration (FDA) has released draft guidance for industry entitled “Submission of Summary Bioequivalence Data for ANDAs.” The draft guidance is intended to assist ANDA applicants in complying with new requirements in a January 2009 final rule that requires ANDA applicants to submit data from all bioequivalence studies (BE studies) the applicant conducts on a drug product formulation submitted for approval. The guidance is applicable to BE studies conducted during both preapproval and postapproval periods. FDA is accepting comments on the draft guidance until July 16, 2009.  

Budget Resolutions Advance with Health Reform Funding

On April 2, 2009, the House and Senate approved separate budget resolutions (H.Con.Res. 85  and S.Con.Res. 13, respectively) that establish nonbinding spending and revenue frameworks for the Congressional committees for fiscal year (FY) 2010. Both bills include deficit-neutral “reserve funds” authorizing committees to adopt health reform measures if offsetting revenues are specified. Such reforms could include, among other things, provisions to make health coverage more affordable, expand access to insurance, improve quality, reduce health care costs, and preserve choice of providers and health plans. In a notable difference, the House bill would allow the Senate to use a procedure called reconciliation to approve health reform legislation by a simple majority, effectively blocking the minority’s ability to force Senate leaders to muster 60 votes in favor of a health reform bill. The House bill also would require the Committees on Ways and Means and Energy and Commerce each to identify $1 billion in health care savings over five years. The Senate adopted an amendment that would prohibit adoption of President Obama’s proposal to change the tax treatment of charitable contributions to pay for health reform. In addition, the House and Senate differ in their approach to fixing the Medicare physician fee schedule formula, which now would trigger an across-the-board payment cut of approximately 21% in 2010. Specifically, the Senate would require that any change to the physician fee schedule be done on a deficit-neutral basis, while the House allocates approximately $87 million over five years/$285 billion over 10 years to reform the formula. The Senate also calls for the importation of prescription drugs approved by the Food and Drug Administration (FDA) from a specified list of countries, and it would establish a deficit-neutral reserve fund to address Medicare and Medicaid reimbursement inequities that lead to access problems in rural areas. Both the House and Senate resolutions also provide up to $311 million for the Health Care Fraud and Abuse Control program for FY 2010. Lawmakers will work to iron out differences between the two measures when Congress returns from recess on April 20, 2009.

Other Congressional Hearings

A number of Congressional panels have held hearings recently on health policy issues, including the following:

Generic Animal Drug User Fees

On April 7, 2009, the FDA announced the rates and payment procedures for generic new animal drug user fees for FY 2009. The notice is available here.

Behind-the-Counter Drug Class

The GAO has issued a report entitled  Nonprescription Drugs: Considerations Regarding a Behind-the-Counter Drug Class.” This report examines the pros and cons of establishing an additional class of nonprescription drugs that would be held behind the counter (BTC) but would require the intervention of a pharmacist before being dispensed, and issues that must be addressed before establishing such a drug class.

House Hearing on Generic Drug Access Legislation (March 31)

On March 31, the House Energy and Commerce Subcommittee on Commerce, Trade, and Consumer Protection is holding a hearing on H.R. 1706, the "Protecting Consumer Access to Generic Drugs Act of 2009," which would prohibit brand name drug companies from compensating generic drug companies to delay the entry of a generic drug into the market.

Obama Budget Proposal

On February 26, 2009, the Obama Administration released its proposed federal budget for fiscal year (FY) 2010. Most significantly in terms of health policy, the proposal would establish a reserve fund of $633.8 billion over 10 years to finance health reform. While half of the reserve funds would come from tax increases on higher-income individuals, the rest would come from health system savings impacting a wide range of providers, health plans, and manufacturers. While additional details are expected to be released in the coming weeks, the following are highlights of the information released to date: 

  • Medicare Advantage (MA) Payments. The budget would replace the current mechanism for establishing MA rates with a competitive system in which Medicare payments would be based upon an average of plans’ bids. The Administration estimates a savings of more than $175 billion over 10 years from this provision – approximately half of the health care savings in the budget proposal.
  • Reducing Drug PricesThe Administration proposes establishing a regulatory pathway for approval of follow-on biologicals. Additionally, brand biologic manufacturers would be prohibited from reformulating existing products into new products to restart the exclusivity process. The Administration also would prevent drug companies from blocking generic drugs from consumers by prohibiting anticompetitive agreements between brand name and generic drug manufacturers intended to keep generic drugs off the market. The budget also would increase the Medicaid drug rebate for brand-name drugs from 15.1% to 22.1% of the average manufacturer price (AMP), apply the additional rebate to new drug formulations, and allow states to collect rebates on drugs provided through Medicaid managed care organizations. The budget also supports the Food and Drug Administration’s (FDA) efforts to allow Americans to buy drugs from other countries.
  • Medicare and Medicaid Payment Accuracy/Program Integrity. The budget would expand CMS’s capacity to identify excessive payments and correct problems, such as through use of National Correct Coding Initiative edits for Medicaid claims. The budget also proposes to dedicate additional resources for oversight and program integrity activities related to the Medicare prescription drug program, MA, and Medicaid.
  • Hospital/Post-Acute Care Bundling, Reduced Hospital Readmission Rates. The budget calls for bundling payments to hospitals and certain post-acute providers for services provided within 30 days after discharge from the hospital. In addition, hospitals with high rates of readmission would be paid less if patients are re-admitted to the hospital within the same 30-day period.
  • Hospital Quality Improvement. The budget would link a portion of Medicare payments for acute inpatient hospital services to hospital performance on specific quality measures.
  • Physician Payment System Reforms. The Administration supports “comprehensive, but fiscally responsible” reforms to the physician fee schedule formula.
  • Cancer Research.  The budget includes over $6 billion in funding for the National Institutes of Health (NIH) to support cancer research.

Other Medicare/Medicaid health policy line-items identified in the budget charts include the following, among others: 

  • Establishing survey and certification revisit and recertification user fees;
  • Enabling physicians to form voluntary groups that coordinate care for Medicare beneficiaries and to receive performance-based payments for coordinated care;
  • Addressing financial conflicts of interest in physician-owned specialty hospitals;
  • Requiring the use of radiology benefit managers for Medicare imaging services;
  • Aligning Medicare home health payments with costs; and
  • Imposing higher Medicare drug benefit premiums on certain higher-income beneficiaries.

Note that many provisions of the proposed budget would require Congressional approval to implement. To that end, a number of Congressional committees have scheduled hearings on the budget proposal, including a March 10 Senate Finance Committee hearing focusing on the budget’s health care provisions.

Maximum FDA Civil Money Penalty (CMP) Amounts

The FDA has published a rule confirming the March 27, 2009 effective date for its November 12, 2008 direct final rule regarding CMPs prescribed by the Food and Drug Administration Amendments Act of 2007.

Draft FDA Guidance Documents

The FDA is soliciting comments on the following draft guidance documents:

Software for Proprietary Drug and Biological Names

The FDA has announced the availability of the source code and supporting technical documentation for the Phonetic Orthographic Computer Analysis (POCA) software program. POCA is an analytic tool designed to help identify drug and biologic names and medical terminology that are phonetically and orthographically similar to one another, which FDA uses to review proposed proprietary drug and biologic names. 

MedPAC Report to Congress -- Medicare Payment/Transparency Provisions

On February 27, 2009, MedPAC released its March 2009 Report to the Congress: Medicare Payment Policy. The report includes a series of recommendations for Medicare payments designed to assure beneficiaries’ access to care and preserve Medicare’s long-term sustainability, particularly through reductions in payment updates for 2010. The report also includes recommendations to increase transparency of physician financial relationships. A listing of key recommendations follows after the jump. 

Hospitals

  • The Congress should increase payment rates for the acute inpatient and outpatient prospective payment systems in 2010 by the projected rate of increase in the hospital market basket index, concurrent with implementation of a quality incentive payment program.
  • The Congress should reduce the indirect medical education adjustment (IME) in 2010 by 1 percentage point to 4.5 percent per 10 percent increment in the resident-to-bed ratio. The funds obtained by reducing the IME adjustment should be used to fund a quality incentive payment program.

Physicians and Ambulatory Surgical Centers

  • The Congress should update payments for physician services in 2010 by 1.1 percent.
  • The Congress should establish a budget-neutral payment adjustment for primary care services billed under the physician fee schedule and furnished by primary-care-focused practitioners. Primary-care-focused practitioners are those whose specialty designation is defined as primary care and/or those whose pattern of claims meets a minimum threshold of furnishing primary care services. The Secretary would use rulemaking to establish criteria for determining a primary-care-focused practitioner.
  • The Congress should direct the Secretary to increase the equipment use standard for expensive imaging machines from 25 to 45 hours per week. This change should redistribute RVUs from expensive imaging to other physician services.
  • The Congress should increase payments for ambulatory surgical centers (ASC) services in calendar year 2010 by 0.6 percent. In addition, the Congress should require ASCs to submit to the Secretary cost data and quality data that will allow for an effective evaluation of the adequacy of ASC payment rates.

Dialysis Services

  • The Congress should maintain current law and update the composite rate in calendar year 2010 by 1 percent.

Skilled Nursing Facility Services

  • The Congress should eliminate the update to payment rates for skilled nursing facility services for fiscal year 2010.
  • The Congress should require the Secretary to revise the skilled nursing facility (SNF) prospective payment system by: adding a separate nontherapy ancillary (NTA) component, replacing the therapy component with one that establishes payments based on predicted patient care needs, and adopting an outlier policy.
  • The Secretary should direct SNFs to report more accurate diagnostic and service-use information by requiring that: claims include detailed diagnosis information and dates of service, services furnished since admission to the SNF be recorded separately in the patient assessment, and SNFs report their nursing costs in the Medicare cost report.
  • The Congress should establish a quality incentive payment policy for SNFs in Medicare and to improve quality measurement for SNFs, the Secretary should: add the risk-adjusted rates of potentially avoidable rehospitalizations and community discharge to its publicly reported post-acute care quality measures; revise the pain, pressure ulcer, and delirium measures currently reported on CMS’s Nursing Home Compare website; and require SNFs to conduct patient assessments at admission and discharge.

Home Health Services

  • The Congress should eliminate the market basket increase for 2010 and advance the planned reductions for coding adjustments in 2011 to 2010, so that payments in 2010 are reduced by 5.5 percent from 2009 levels.
  • The Congress should direct the Secretary to re-base rates for home health care services in 2011 to reflect the average cost of providing care.
  • The Congress should direct the Secretary to assess payment measures that protect the quality of care and ensure incentives for the efficient delivery of home health care. The study should include alternative payment strategies such as blended payments and risk corridors and outcome-based quality incentives.

Inpatient Rehabilitation Facilities

  • The update to the payment rates for inpatient rehabilitation services should be eliminated for fiscal year 2010.

Long-Term Care Hospitals

  • The Secretary should update payment rates for long-term care hospitals for fiscal year 2010 by the projected rate of increase in the rehabilitation, psychiatric and long-term care hospital (RPL) market basket index less the Commission’s adjustment for productivity growth.

Recommendations on Medicare Advantage Payments

  • The Congress should: Eliminate the stabilization fund for regional PPOs. Remove the effect of payments for indirect medical education from the MA plan benchmarks. Set the benchmarks that CMS uses to evaluate MA plan bids at 100 percent of FFS costs. Pay-for-performance should apply in MA to reward plans that provide higher quality care. Clarify that regional plans should submit bids that are standardized for the region’s MA-eligible population.
  • The Secretary should calculate clinical measures for the FFS program that would permit CMS to compare the FFS program with MA plans.

Recommendations on Public Reporting of Physician Financial Relationships

  • The Congress should require all manufacturers and distributors of drugs, biologicals, medical devices, and medical supplies (and their subsidiaries) to report to the Secretary their financial relationships with: physicians, physician groups, and other prescribers; pharmacies and pharmacists; health plans, pharmacy benefit managers, and their employees; hospitals and medical schools; organizations that sponsor continuing medical education; patient organizations; and professional organizations.
  • The Congress should direct the Secretary to post the information submitted by manufacturers on a public website in a format that is searchable by: manufacturer; recipient’s name, location, and specialty (if applicable); type of payment; name of the related drug or device (if applicable); and year.
  • The Congress should require manufacturers and distributors of drugs to report to the Secretary the following information about drug samples: each recipient’s name and business address; the name, dosage, and number of units of each sample; and the date of distribution. The Secretary should make this information available through data use agreements.
  • The Congress should require all hospitals and other entities that bill Medicare for services to annually report the ownership share of each physician who directly or indirectly owns an interest in the entity (excluding publicly traded corporations). The Secretary should post this information on a searchable public website.
  • The Congress should require the Secretary to submit a report, based on the Disclosure of Financial Relationships Report, of the types and prevalence of financial arrangements between hospitals and physicians.

Recommendations on Reforming the Hospice Benefit

  • The Congress should direct the Secretary to change the Medicare payment system for hospice to: have relatively higher payments per day at the beginning of the episode and relatively lower payments per day as the length of the episode increases; include a relatively higher payment for the costs associated with patient death at the end of the episode; and implement the payment system changes in 2013, with a brief transitional period. These payment system changes should be implemented in a budget neutral manner in the first year.
  • The Congress should direct the Secretary to: require that a hospice physician or advanced practice nurse visit the patient to determine continued eligibility prior to the 180th-day recertification and each subsequent recertification and attest that such visits took place, require that certifications and recertifications include a brief narrative describing the clinical basis for the patient’s prognosis, and require that all stays in excess of 180 days be medically reviewed for hospices for which stays exceeding 180 days make up 40 percent or more of their total cases.
  • The Secretary should direct the Office of Inspector General to investigate: the prevalence of financial relationships between hospices and long-term care facilities such as nursing facilities and assisted living facilities that may represent a conflict of interest and influence admissions to hospice, differences in patterns of nursing home referrals to hospice, the appropriateness of enrollment practices for hospices with unusual utilization patterns (e.g., high frequency of very long stays, very short stays, or enrollment of patients discharged from other hospices), and the appropriateness of hospice marketing materials and other admissions practices and potential correlations between length of stay and deficiencies in marketing or admissions practices.
  • The Secretary should collect additional data on hospice care and improve the quality of all data collected to facilitate the management of the hospice benefit. Additional data could be collected from claims as a condition of payment and from hospice cost reports.

Good Reprint Practices

The FDA published a notice on January 13, 2009 announcing a final guidance document entitled “Good Reprint Practices for the Distribution of Medical Journal Articles and Medical or Scientific Reference Publications on Unapproved New Uses of Approved Drugs and Approved or Cleared Medical Devices.” The guidance, which finalizes a February 20, 2008 draft policy, is intended to provide manufacturers with the agency's views on permissible distribution by a company's sales representatives of medical journal articles and scientific or medical reference publications that discuss unapproved new uses for FDA-approved drugs or biologics or FDA-approved or cleared medical devices to healthcare professionals. As with the 2008 draft guidance, the final version notes the need to balance the law’s prohibition on distributing or promoting “unapproved uses of approved drugs and approved or cleared medical devices” with the “important public policy” of providing information that “may even constitute a medically recognized standard of care.” The FDA concludes that the touchstone for lawful dissemination of literature about unapproved uses is that the publications “are truthful and non-misleading.” To meet this standard, the FDA final guidance lists “principles of Good Reprint Practices” that include criteria for determining the type of publication and the manner in which the publication can be distributed. Although the final guidance closely tracks the draft guidance, it has some important clarifications, including revisions to the Good Reprint Practices and a specific reference encouraging manufacturers to seek approvals and clearance for new indications and intended uses for medical products.   A Reed Smith analysis of the final guidance is available here.

Submission of Bioequivalence Data

On January 16, 2009, the FDA published a final rule requiring an abbreviated new drug application (ANDA) applicant to submit data from all bioequivalence (BE) studies the applicant conducts on a drug product formulation submitted for approval. In the past, ANDA applicants have submitted BE studies demonstrating that a generic product meets bioequivalence criteria in order for FDA to approve the ANDA, but have not typically submitted additional BE studies conducted on the same drug product formulation, such as studies that do not show that the product meets these criteria. The FDA now believes that additional BE study data may be important in determining whether the proposed formulation is bioequivalent to the reference listed drug, and will increase FDA’s understanding of how changes in components, composition, and methods of manufacture may affect product formulation performance. The rule is effective July 15, 2009.

Certifications of Clinical Trial Registry/Results Submissions; Final Guidance

The FDA has released final guidance on “Certifications To Accompany Drug, Biological Product, and Device Applications/Submissions: Compliance with Section 402(j) of The Public Health Service Act, Added By Title VIII of The Food and Drug Administration Amendments Act of 2007.” The guidance describes the FDA’s current thinking regarding the types of applications and submissions that sponsors, industry, researchers, and investigators submit to FDA and accompanying certifications under the FDAAA related to the submission of required information to the clinical trials data bank, www.ClinicalTrials.gov.

FDA Proposes Guidance for Meeting Clinical Trial Registration Requirements

The Food and Drug Administration Amendments Act of 2007 (FDAAA) expanded the public reporting requirements for “applicable clinical trials” involving certain drugs, biologicals, and devices. In December 2008, the Food and Drug Administration (FDA) issued a draft guidance document proposing, among other things, definitions that would affect who would be required to register certain clinical trials under the FDAAA. In some cases, FDA's proposal would require manufacturers who make grants to investigators to be the responsible party required to register. In addition, FDA elaborates on the circumstances under which clinical investigations designed to demonstrate bioequivalency would be subject to reporting. Additional background information is available at the clinicaltrials.gov web site. Reed Smith is reviewing the draft guidance. Please contact Areta Kupchyk at akupchyk@reedsmith.com for more information.

FDA Finalizes Guidance Document on OTC Drug Labeling

The FDA has issued final guidance on “Labeling OTC Human Drug Products—Questions and Answers.” This document is intended to assist manufacturers, packers, and distributors of over-the-counter (OTC) drug products in complying with the agency’s regulation on standardized content and format requirements for the labeling of OTC drug products, including the use of toll-free numbers and compliance with adverse event reporting requirements.

FDA Training Program

The FDA is inviting pharmaceutical companies to participate in the FDA’s Regulatory Project Management Site Tours and Regulatory Interaction Program, through which FDA personnel observe operations of pharmaceutical manufacturing and/or packaging facilities, pathology/toxicology laboratories, and regulatory affairs operations. The goals of the program are to provide FDA regulatory project managers with first-hand exposure to industry’s drug development processes, and to offer a venue for sharing information about project management procedures (but not drug-specific information) with industry representatives.  Interested companies may submit proposed agendas to FDA by March 6, 2009. 

FDA Notice on Electronic Submissions

FDA is soliciting comments on the paperwork burden associated with its plan to require drug establishment registration and drug listing submissions in electronic format. Comments will be accepted until February 9, 2009. 

FDA Study on DTC Ad Coupons

The Food and Drug Administration (FDA) is seeking comments on a planned "Experimental Study of the Impact of Coupons Embedded in Direct-to-Consumer Prescription Drug Print Advertisements." According to the FDA notice, the study will examine the impact of the presence of coupons offering price incentives or rebates on consumers' perceptions of product risks and benefits in direct-to-consumer (DTC) print ads. The FDA acknowledges, however, that "it does not actually regulate the dollar or other incentive amount of coupons, price incentives, or rebate offers with respect to how they affect the price of prescription drugs or biological products." Comments will be accepted until February 13, 2009.  

FDA Grants for Orphan Product Studies

The FDA Office of Orphan Product Development has announced grants to support the clinical development of products for use in rare diseases or conditions where no current therapy exists or where the proposed product will be superior to the existing therapy. Grants are available for clinical studies on safety and/or effectiveness that will either result in, or substantially contribute to, market approval of these products. Of the estimated FY 2010 funding ($14.1 million) for this program, approximately $10 million will fund noncompeting continuation awards, and approximately $4.1 million will fund 10 to 12 new awards, subject to availability of funds. 

Labeling of Nonprescription Human Drug Products and Dietary Supplements; Enforcement Date Extended to January 1, 2010

The FDA has released revised draft guidance entitled “Questions and Answers Regarding the Labeling of Nonprescription Human Drug Products Marketed Without an Approved Application as Required by the Dietary Supplement and Nonprescription Drug Consumer Protection Act: Revision 1.” This guidance is intended to assist industry in complying with the labeling requirements for nonprescription (over-the-counter (OTC)) human drugs marketed without an approved application established by the Dietary Supplement and Nonprescription Drug Consumer Protection Act. In an earlier version of the guidance issued January 2, 2008, the FDA stated that it intended to begin enforcing the labeling requirements for OTC drug products marketed without an approved application that are labeled on or after January 1, 2009; because the agency is still finalizing the guidance, the revised draft extends the enforcement date until January 1, 2010. Separate guidance regarding compliance with the labeling requirements for dietary supplements also has been released.

Orally Disintegrating Tablets

The FDA has released guidance for manufacturers of new and generic pharmaceutical products on the definition of an orally disintegrating tablet (ODT). The FDA notes that manufacturers have developed products that can be ingested simply by placing them on the tongue, eliminating the need to chew the tablet, swallow an intact tablet, or take the tablet with liquids. As new products have been developed using different technology and formulations, many of these later products exhibited wide variation in product characteristics from the initial products. Because this shift in product characteristics can affect suitability for particular uses, the agency developed this guidance for industry.

FDA Study on DTC Drug Advertising

The Food and Drug Administration (FDA) is announcing an opportunity for public comment on a planned FDA study examining consumer comprehension of inclusion of a toll-free number to report side effects in direct-to-consumer (DTC) prescription drug television advertisements. Comments will be accepted until January 26, 2009. 

Draft FDA Guidance on Patent Information for Certain Old Antibiotics

The FDA has released for public comment draft guidance entitled “Submission of Patent Information for Certain Old Antibiotics.” The document describes the agency's current thinking on the implementation of certain provisions of the Q1 Program Supplemental Funding Act (the Q1 Act) that concern old antibiotics and addresses which sponsors of new drug applications must submit patent information under the Q1 Act by December 5, 2008. Comments on the guidance will be accepted until February 2, 2009. 

Cooperative Manufacturing Arrangements for Licensed Biologics

The FDA has announced the availability of a document entitled “Guidance for Industry: Cooperative Manufacturing Arrangements for Licensed Biologics.” The guidance, which applies to biological products subject to licensure under the U.S. Public Health Service Act, describes the licensing strategies for meeting the increased need for flexible manufacturing arrangements, such as short supply arrangements, divided manufacturing arrangements, shared manufacturing arrangements, and contract manufacturing arrangements.

FDA/WebMD Partnership

On December 3, 2008, the FDA announced it is teaming with WebMD to expand consumer access to FDA safety alerts and other public health information. Among other things, the partnership includes a new online consumer health information resource on WebMD.com where consumers can access information on the safety of FDA-regulated products. 

Draft FDA Guidance: Evaluating Drug/Biological Proprietary Names

On November 24, 2008, the Food and Drug Administration (FDA) announced the availability of a draft guidance document entitled “Contents of a Complete Submission for the Evaluation of Proprietary Names.” The document is intended to help prevent medication errors by assisting industry in the submission of complete product information that will enable FDA to evaluate the safety of proposed proprietary drug and biological product names. In addition, FDA intends to use this information in the assessment of promotional aspects of proposed proprietary names. The FDA will accept comments on the draft until January 23, 2009.   

Draft FDA Guidance: Process Validation: General Principals and Practices

Separately, on November 18, 2008, the FDA announced the availability of draft guidance entitled Process Validation: General Principles and Practices.” The FDA is revising related May 1987 guidance to promote a “lifecycle approach” to process validation for the manufacture of human and animal drug and biological products, including active pharmaceutical ingredients. Comments will be accepted until January 20, 2009.

Off-Label Uses of Anti-Cancer Drugs

On October 24, 2008, CMS issued a transmittal updating the Medicare Benefit Policy Manual to recognize the four authoritative compendia for use in the determination of a medically-accepted indication of drugs and biologicals used off-label in an anti-cancer chemotherapeutic regimen. The transmittal also modifies requirements for contractors to identify off-label uses that are supported by clinical research.

FDA Guidance on Cellular and Gene Therapy Products

The Food and Drug Administration (FDA) has released a draft guidance document on “Potency Tests for Cellular and Gene Therapy Products.” The document is designed to provide manufacturers of cellular and gene therapy products with recommendations for developing tests to measure potency to support an Investigational New Drug Application (IND) or a Biologics License Application (BLA). FDA will accept comments on the guidance through January 7, 2009. 

AHRQ Technology Assessments for Public Comment

The AHRQ's Technology Assessment Program develops systematic reviews and health technology assessments at the request of CMS in order to inform national Medicare coverage policies. AHRQ has announced that beginning October 15, 2008, it will post draft Technology Assessment Program reports for public comment on its website.  In a related development, on October 14, 2008, AHRQ has announced that it will be releasing for public comment a draft White Paper on "Potential Conflict of Interest in the Production of Drug Compendia" on October 22, and comments will be accepted on the draft until November 5, 2008.

Final Rule on Medicaid Definition of Multiple Source Drug

On October 7, 2008, CMS pubished a final rule revising the definition of "multiple source drug" under the Medicaid program. The rule also responds to public comments received on the March 14, 2008 interim final rule with comment period. The final rule adopts the March 2008 interim final rule with the following change: in §447.205, paragraph (3)(i) of the definition of multiple source drug, the term "covered outpatient drug" is revised to read "drug product" and "listed product" respectively to reflect the statutory language. As noted in the interim final rule with comment period, to the extent that this rule may affect Medicaid reimbursement rates for retail pharmacies, it is subject to the injunction issued by the United States District Court for the District of Columbia in National Association of Chain Drug Stores et al. v. Health and Human Services. The rule is effective is effective November 6, 2008. 

FDA Authorized Generics Reporting Requirements

On September 29, 2008, the Food and Drug Administration (FDA) published a direct final rule  requiring holders of a new drug application (NDA) to submit certain information regarding authorized generic drugs in an annual report .  The rulemaking is part of the agency’s implementation of the Food and Drug Administration Amendments Act of 2007 (FDAAA), which requires that FDA publish a list of all authorized generic drugs included in an annual report since 1999, and that the agency update the list quarterly.  The agency is publishing the requirement as a direct final rulemaking because it does not expect significant adverse comment on the rule. As part of the administrative requirements for a direct final rule, the agency is concurrently issuing a proposed rule and soliciting comments and, if any significant adverse comment is received, the FDA will withdraw the direct final rule and address the comments in a subsequent rulemaking.  If there is no significant adverse comment, the direct final rule will automatically become effective February 11, 2009.  Comments will be accepted until December 15, 2008, although comments on related information collection provisions are due October 29, 2008.

Online Pharmacy, Organ Transplant Bills Clear Congress; Health Start Bill Enacted.

The Senate recently joined the House in approving H.R. 6353, the “Ryan Haight Online Pharmacy Consumer Protection Act,” which would prohibit the sale of controlled substances over the Internet without a valid prescription and subject on-line pharmacies to a series of new restrictions. Likewise, the Senate approved H.R. 6469, which would increase funding for the Organ Procurement and Transplantation Network. Both bills now await the President’s signature. In addition, President Bush has signed into law S. 1760, which reauthorizes the Healthy Start Initiative to provides grants to reduce infant mortality and improve maternal health.

Health Policy Legislation Moves Through Congress

There has been a flurry of recent legislative activity as the 110th Congress prepares to adjourn in the coming days. Congress has taken action on a wide range of health policy bills, addressing such issues as FDA drug and device approvals, internet pharmacy regulation, health care workforce issues, insurance coverage, and medical treatment, and patent protection for antibiotics.   Specifically, the following legislation has been approved by the House and Senate and now await the President’s signature:

  • H.R. 6353, the “Ryan Haight Online Pharmacy Consumer Protection Act,” which would prohibit the sale of controlled substances over the Internet without a valid prescription and subject on-line pharmacies to a series of new restrictions.
  • S. 3560, the “QI Program Supplemental Funding Act,” which would authorize an additional $45 million for the Medicare Qualifying Individuals (QI) program, which helps certain low-income individuals pay their Medicare Part B premiums. The legislation also includes provisions to modify the patent protections applicable to antibiotics and clarify the ability of generic drug companies to gain approval of and market generic antibiotics. In addition, the bill would expand the education activities under the Medicaid Integrity Program (MIP) and extend funding for the Medicare Improvement Fund to make improvements under the original Medicare program. 
  • Several measures aimed at expanding disease research and information resources, including: H.R. 1157, which would authorize grants for the development and operation of research centers for the study of environmental factors that may be related to the etiology of breast cancer; H.R. 1532, which would reauthorize the Preventive Health Services Regarding Tuberculosis program; H.R. 5265, which would promote research into the causes and treatments of various forms of Muscular Dystrophy; S. 1810, to authorize the HHS Secretary to collect and disseminate information regarding Down syndrome or other prenatally or postnatally diagnosed diseases and to coordinate the provision of support services for those who receive a diagnosis of one of those diseases; and S. 1382, which would authorize funding for the establishment of a national registry for the collection and storage of data on amyotrophic lateral sclerosis (ALS).

 

  • S. 2932, which would reauthorize the poison center national toll-free number, provide grants for poison centers, and expand poison prevention education efforts.
  • S. 1760, which would reauthorize the Healthy Start Initiative, which provides grants to reduce infant mortality and improve maternal health.
  • H.R. 1343, which would provide funding to enable health centers to serve medically underserved populations and reauthorize the National Health Service Corps Scholarship and Loan Repayment programs.

In addition, the following bills have been approved by the House but are awaiting Senate action.

  • H.R. 1014, the Heart Disease Education, Analysis Research, and Treatment (HEART) for Women Act, which would require new drug, biologics, and device applications submitted to FDA to include any clinical data possessed by the sponsor that relates to safety or effectiveness by gender, age, and racial subgroups. This information would be posted on the internet. The bill also would authorize research and public health activities to reduce cardiovascular disease in women. 
  • H.R. 6908, the “HIPAA Recreational Injury Technical Correction Act,” which would require timely disclosure of limitations and restrictions on coverage under group health plans.
  • H.R. 758, which would require health insurers to cover minimum lengths of stay and secondary consultations for patients undergoing procedures to treat and diagnose breast cancer.
  • H.R. 2583, the “Physician Workforce and Graduate Medical Education Enhancement Act ,” which would authorize a loan repayment program for hospitals to start a residency training program.
  • H.R. 6568, which would expand research and educational activities related to pulmonary hypertension.
  • H.R. 2994, which would direct the Department of Health and Human Services to establish a national pain care education outreach and awareness campaign.
  • H.R. 6469, which would increase funding for the Organ Procurement and Transplantation Network.
  • H.R. 5352, the “Elder Abuse Victims Act,” which would establish specialized elder abuse prosecution and research programs and activities to aid victims of elder abuse, provide training to prosecutors and other law enforcement related to elder abuse prevention and protection.
  • H.R. 6901, which would improve treatment for methamphetamine addiction.

CGMPs for Finished Pharmaceuticals

On September 8, 2008, the Food and Drug Administration (FDA) published a final rule amending its current good manufacturing practice (CGMP) requirements for finished pharmaceuticals, effective December 8, 2008. In particular, the rule revises CGMP requirements concerning aseptic processing, verification of performance of operations by a second individual, and the use of asbestos filters. 

FDA Guidance: Bioequivalence (BE) Recommendations and Drug Clinical Trials

The FDA has announced the availability of additional draft and revised draft product-specific guidance on the design of BE studies to support abbreviated new drug applications (ANDAs). FDA will accept comments on the recommendations until December 4, 2008. Separately, the FDA has released draft guidance entitled “M3(R2) Nonclinical Safety Studies for the Conduct of Human Clinical Trials and Marketing Authorization for Pharmaceuticals.'' The document, which discusses the types of nonclinical studies and their relation to the conduct of human clinical trials and marketing authorization for pharmaceuticals, is intended to facilitate the timely conduct of clinical trials and reduce the unnecessary use of animals and other drug development resources. The FDA is requesting comments on the draft guidance by October 20, 2008.

Animal Drug User Fees

On July 16, 2008, the House Energy and Commerce Committee approved amended versions of H.R. 6432, the "Animal Drug User Fee Amendments of 2008," and H.R. 6433, the "Animal Generic Drug User Fee Act of 2008." 

FDA CGMP Rule Regarding Investigational Drugs

On July 15, 2008, the Food and Drug Administration (FDA) published a final rule amending the current good manufacturing practice (CGMP) regulations for human drugs and biological products to exempt most phase 1 investigational drugs from complying with the regulatory CGMP requirements.  FDA will continue to exercise oversight of the manufacturing of these drugs under FDA's general statutory CGMP authority and through review of the investigational new drug applications. In addition, FDA is announcing the availability of a guidance document on "CGMP for Phase 1 Investigational Drugs," which sets forth recommendations on approaches to compliance with statutory CGMP for the exempted phase 1 investigational drugs.  FDA is taking this action to focus a manufacturer's effort on applying CGMP that is appropriate and meaningful for the manufacture of the earliest stage investigational drug products intended for use in phase 1 clinical trials while ensuring safety and quality.  This action will also streamline and promote the drug development process, according to FDA.

FDA Rule/Guidance on Investigational New Drugs Intended for Use in Clinical Trials

 On July 15, 2008, FDA is publishing a final rule amending the current good manufacturing practice (CGMP) regulations for human drugs and biological products to exempt most phase 1 investigational drugs from complying with the regulatory CGMP requirements. FDA will continue to exercise oversight of the manufacturing of these drugs under FDA's general statutory CGMP authority and through review of the investigational new drug applications (IND). In addition, FDA is announcing the availability of a guidance document on "CGMP for Phase 1 Investigational Drugs," which sets forth recommendations on approaches to compliance with statutory CGMP for the exempted phase 1 investigational drugs. FDA is taking this action to focus a manufacturer's effort on applying CGMP that is appropriate and meaningful for the manufacture of the earliest stage investigational drug products intended for use in phase 1 clinical trials while ensuring safety and quality. This action also will streamline and promote the drug development process, according to FDA.

Anti-Cancer Drug Compendia

CMS has updated its list of authorized compendia that may be used in determining medically-accepted indications of drugs and biologicals used off-label in an anti-cancer chemotherapeutic regimen under Medicare Part B. Specifically, CMS will add Elsevier Gold Standard’s Clinical Pharmacology compendium to the list of Medicare anti-cancer treatment compendia. Earlier this year, CMS approved the NCCN Drugs & Biologics Compendium and Thomson Micromedex DrugDex. More information is available here.

OIG Report on FDA Generic Drug Reviews

The OIG has released a report entitled "The Food and Drug Administration's Generic Drug Review Process". The OIG found that FDA disapproved 96 percent of original Abbreviated New Drug Applications (ANDA) under review in 2006 because they did not meet FDA review standards, and that many reviews exceeded the statutory review timeframe. In response, the FDA stated that it is implementing process improvements to address these areas.

FDA Rule on Drug Label Pregnancy Information

On May 29, 2008, the Food and Drug Administration (FDA) published a proposed rule to make major revisions to drug label requirements related to the use of a drug during pregnancy and breast feeding. Among other things, the agency is proposing to require that labeling include a summary of the risks of using a drug during pregnancy and lactation and a discussion of the data supporting that summary. The labeling also would include relevant clinical information – presented in sections on fetal risks, clinical considerations, and data – designed to help health care providers make prescribing decisions and counsel women about the use of drugs during pregnancy and/or lactation. Current letter categories would be removed from the pregnancy section of drug labeling. Certain newly approved drugs would use the new pregnancy and lactation labeling format, while labeling requirements for previously approved drugs will be phased-in. The FDA is accepting comments on the proposal until August 27, 2008. Additional information is posted here.