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FDA announced the availability of a draft guidance document entitled, “FDA Regulation of PET Drug Products – Questions and Answers,” which addresses nearly all aspects of the drug regulatory process for positron emission tomography (PET) drugs.  The draft guidance includes details on PET drug  application submission, review, compliance with current good manufacturing practices, inspections, registration and listing, and user fees.

Today, FDA issued a final rule entitled, “Further Amendments to General Regulations of the Food and Drug Administration to Incorporate Tobacco Products,” which amends certain general regulations to include tobacco products, ensuring that tobacco manufacturers adhere to regulations that apply to other FDA-regulated products.  The Rule also clarifies FDA’s policies for voluntary recalls of tobacco products and guarantees tobacco manufacturers the same rights as other FDA-regulated entities.

Yesterday, FDA approved Kalydeco (ivacaftor) for the treatment of a rare form of cystic fibrosis (CF) in patients ages 6 and older who have the G551D mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene.  FDA Commissioner Dr. Margaret Hamburg noted that Kalydeco is an excellent example of advances in personalized medicine, in which treatments are designed for patients with specific genetic profiles.  Kalydeco is the first available treatment which targets the defective CFTR protein, the underlying cause of CF.

Yesterday, FDA announced approval of Erivedge (vismodegib) to treat adult patients with basal cell carcinoma, the most common type of skin cancer.  The drug, manufactured by Roche’s Genentech, is the first FDA-approved drug for metastatic basal cell carcinoma and was reviewed under the agency’s priority review program.

On Friday, FDA approved Inlyta (axitinib) for treatment in patients with renal cell carcinoma who have not responded to other drugs for this cancer type.  Since 2005, seven drugs have been approved for the treatment of metastatic or advanced kidney cell cancer, which, according to Dr. Richard Pazdur, Director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research (CDER), has significantly altered the treatment paradigm for metastatic kidney cancer. 

Today, FDA announced the reopening of the comment period for the proposed rule entitled, “Direct-to-Consumer (DTC) Prescription Drug Advertisements; Presentation of the Major Statement in Television and Radio Advertisements in a Clear, Conspicuous, and Neutral Manner.”  The agency is looking for data to establish standards that would be considered in determining whether the major statement in DTC advertisements relating to side effects and contraindications of an advertised prescription drug is presented in a clear, conspicuous and neutral way.

FDA’s Center for Devices and Radiological Health (CDRH) released its 2012 strategic priorities focusing on four areas:  implementing a total product life cycle approach, enhancing its internal and external communication and transparency, strengthening its workforce, and proactively facilitating innovation.  The initiatives include a pilot program set to begin April 1, 2012 to triage premarket applications to increase review efficiency, and a proposal by December 31, 2012 to clarify the circumstances when CDRH could rely on clinical studies conducted in and for other countries.

Today, FDA announced the availability of additional draft and revised draft recommendations for the design of product-specific bioequivalence (BE) studies to support abbreviated new drug applications (ANDAs).  The new BE recommendations are published on FDA’s website, and were developed via the process described in the May 2007 draft guidance document entitled, “Bioequivalence Recommendations for Specific Products.”

Today, FDA announced the availability of a guidance document for industry entitled, “Product Name Placement, Size, and Prominence in Advertising and Promotional Labeling” intended to clarify the product naming requirements for prescription human drugs, biological drug products, and prescription animal drugs.  Specifically, the guidance document addresses the juxtaposition of the proprietary and established names in relation to certain graphic presentations, and the problems that stem from obscuring the presentation of, or minimizing disclosure of the established name.

On Friday, FDA allowed marketing of the STRATIFY JCV™ Antibody ELISA test, the first test designed to help determine the risk of a rare brain infection called progressive multifocal leukoencephalopathy (PML) in people using the drug Tysabri to treat multiple sclerosis (MS) or Crohn’s Disease (CD).  The STRATIFY JCV Antibody ELISA test, when analyzed in light of other clinical factors of the patient, can allow physicians and patients to assess the risk for developing PML, and provide better insight into the benefits of continuing Tysabri treatment in these patients.  In a separate but related action, FDA announced approval of updates to the drug label for Tysabri which identifies a positive test for anti-JCV antibodies as a risk factor for developing PML.

According to Mr. Chris Viehbacher, Chairman of the Pharmaceutical Research and Manufacturers of America (PhRMA) and CEO of Sanofi SA, the pharmaceutical industry may halt investments in medicines designed to treat primary care diseases such as diabetes or obesity, due to a lack of explicit guidelines regarding FDA’s assessment of the risk-to-benefit ratio for widely used medicines.  Companies may instead focus on specialized cancer drugs, where FDA’s willingness to accept some serious risks in exchange for potentially life-saving treatments is clearly defined.

In meeting materials released in advance of tomorrow’s Advisory Committee for Reproductive Health Drugs meeting, FDA rejects Columbia Laboratories’ statistical analysis of their progesterone gel 8%, stating that the results lack significant proof of efficacy in reducing preterm births among US women with a short cervix.  The agency also stated that although the evidence of efficacy in populations outside of the US was stronger than within the US population, the different racial makeup of these foreign site studies does not provide enough evidence of the drug’s efficacy in the US.

Today, FDA announced the availability of a guidance document entitled, “Preparation of Investigational Device Exemptions (IDE) and Investigational New Drug (IND) Applications for Products Intended To Repair or Replace Knee Cartilage.”  This guidance document is intended to provide sponsors of IDE and IND applications with specific recommendations of what information that should be included in their submission for non-prosthesis products intended to repair or replace knee cartilage.

Christy Foreman, Director of FDA’s Office of Device Evaluation (ODE) within the Center for Devices and Radiological Health (CDRH), announced the creation of a new Chief Medical Officer position within ODE in order to add clinical support to assist with complex review issues and establish consistent clinical policies.  Dr. Randall Brockman, Medical Officer in the Division of Cardiovascular Devices, will serve as the interim Chief Medical Officer until the position is officially created and permanently filled. 

On Friday, FDA Commissioner Margaret Hamburg, MD, announced that the agency has completed their recommendations for three user fee programs, including the new Generic Drug User Fee and the Biosimilar and Interchangeable Products User Fee programs, as well as the fifth authorization of the Prescription Drug User Fee Act (PDUFA).  Dr. Hamburg stated that user fees were critical to ensuring that FDA has the necessary resources to conduct timely reviews, especially when facing greater budgetary constraint. 

FDA yesterday released minutes from multiple negotiation sessions over the 2012 user fee pact between FDA and medical device industry representatives, which indicate that the two sides have reached agreement on key points, but are still debating the amount of fees that industry would contribute.  The agreed upon draft commitment letter will form the basis for performance metrics to be included in the final user fee agreement for the fiscal years 2013 through 2017, but FDA cautioned that some of the objectives may not be possible if industry does not agree to increase the amount of fees it is willing to pay.

FDA’s Center for Drug Evaluation and Research (CDER) is creating a new Medical Policy Council (MPC) comprised of senior CDER officials to address practical and regulatory issues related to development and approval of novel therapies, as well as to discuss medical policy issues and challenges.  Dr. Janet Woodcock, Director of CDER, explained in an email to staff that MPC meetings would not be open to the public but would include a mechanism for public comment.

FDA has scheduled a meeting of the Arthritis Advisory Committee on March 12, 2012 to discuss development of anti-nerve growth factor (Anti-NGF) drugs for use as analgesics in light of safety issues.  In late 2010, the agency placed a class-wide hold on clinical trials of anti-NGF compounds following a case of avascular necrosis in one study, and reported adverse effects in other trials involving osteoarthritis patients.

FDA’s recent draft guidance document on the process for appealing decisions by the Center for Devices and Radiological Health (CDRH) is intended to clarify the options for having a decision by CDRH reviewed or reconsidered.  According to Jeffrey Shapiro, an attorney with Hyman, Phelps & McNamara, the guidance is helpful in providing a detailed description of the currently available avenues for appeal, but does not reform the process or otherwise address industry complaints including the slowness of the appeals process and potential conflicts of interest involving agency staff who review the appeals.

FDA’s new draft guidance document about the 510(k) premarket review program, issued December 27, 2011, is intended to clarify changes to the 510(k) program that occurred over the past couple of years as part of the 510(k) reform process.  However, many former FDA officials believe that this draft guidance document goes beyond the intended scope by introducing new concepts, such as “primary predicates” and “reference devices,” which may be more confusing than clarifying.

A report conducted by the Department of Health and Human Services, and set to be issued later today, states that hospital employees recognize and report only one out of seven errors, accidents, and events that harm Medicare patients.  According to the report, the amount of underreporting is due to hospital employees not recognizing what constitutes patient harm or not realizing that a particular event has harmed the patient and should be reported, and even when events were reported, hospitals rarely changed policies to prevent re-occurrence.  Medicare officials announced that they would develop a list of reportable events in order to aid hospitals and their employees in recognizing reportable events.