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House Energy and Commerce Committee, Subcommittee on Health Hearing – “FDA User Fees 2012: Hearing on Issues Related to Accelerated Approval, Medical Gas, Antibiotic Development and Downstream Pharmaceutical Supply Chain”

House Energy and Commerce Committee, Subcommittee on Health Hearing – “FDA User Fees 2012: Hearing on Issues Related to Accelerated Approval, Medical Gas, Antibiotic Development and Downstream Pharmaceutical Supply Chain”

Thursday, March 8, 2012, the House Energy and Commerce Committee Subcommittee on Health held a hearing titled, “FDA User Fees 2012: Hearing on Issues Related to Accelerated Approval, Medical Gas, Antibiotic Development and Downstream Pharmaceutical Supply Chain.”  The three panel hearing convened members of government, academia, and industry to address pressing issues surrounding the reauthorization of the Prescription Drug User Fee Act as well as additional proposals to improve mechanisms for drug approval and pending legislation in the House.  The second panel featured Friends of Cancer Research (Friends) Executive Director Dr. Jeff Allen discussing the need to examine opportunities to expand the accelerated approval process, particularly for breakthrough therapies that show great benefit early in development.

Panel One:

Janet Woodcock, Director of the Center for Drug Evaluation and Research at the Food and Drug Administration

The panel commenced with the opening statements by the Chairman, Ranking Member, and other members of the Subcommittee and Full Committee.  Chairman Pitts was particularly interested in how to better utilize the accelerated approval process to speed treatment for rare diseases, while Ranking Member Pallone and Rep. Gringrey discussed legislative proposals such as the Generating Antibiotic Incentives Now (GAIN) Act that aims to incentivize companies to develop new antibiotics to treat illnesses susceptible to drug resistance.    

During her questioning, Dr. Woodcock was asked whether any of the accelerated approval or fast-track proposals being discussed on the panels show possibility in affecting positive change in how drugs are approved, Woodcock acknowledged the need for additional clarity when examining the accelerated approval process and underscored that changing any efficacy or safety standards would not be of benefit to patients.  She stated that breakthrough therapies were important in curbing the AIDS epidemic, and the disease would not be under control today if things had been business as usual.  Mainly, breakthrough therapies are about getting everyone on board and examining the most effective ways to get drugs to patients, however, Dr. Woodcock also stated that legislation is not needed but rather a designation as an “important process.”

Rep. Schakowsky questioned whether the FDA has concerns about the Faster Access to Specialized Treatments (FAST) Act lowering standards for safety and efficacy and whether the FDA needs new authorities to speed drugs to patients.  Dr. Woodcock responded by reiterating the need to clarify, rather than change, the accelerated approval process and looks forward to working with Congress on language in the bill.  She believes no new authorities are needed, but clarification would clear up many of the issues surrounding accelerated approval. 

Dr. Woodcock also answered questions about drug shortages, drug contamination, lot level versus unit level tracking of drugs, online pharmacies, and curbing prescription drug abuse.

Panel Two:

John Maraganore, PhD., Chief Executive Officer, Alnylam Pharmaceuticals; Jeff Allen, PhD., Executive Director, Friends of Cancer Research; Barry Eisenstein, MD, FACP, FIDSA, FAAM, Senior Vice President Scientific Affairs, Cubist Pharmaceuticals; John H. Powers, MD, FACP, FIDSA, Assistant Clinical Professor of Medicine, George Washington University School of Medicine; Michael D. Walsh, President, LifeGas, on behalf of the Compressed Gas Association

In his opening testimony, Friends Executive Director Dr. Jeff Allen explained how accelerated approval, priority review, and fast track mechanisms play an important role in advancing new products and therapies and explored new ways in which to enhance these mechanisms while applying them consistently, efficiently, and effectively over all therapeutic areas without sacrificing safety and efficacy. 

Rep. Pallone stated that breakthrough therapy proposals warrant serious consideration, among others, and asked Dr. Allen to expand on safety and efficacy in these processes.  Dr. Allen first restated that safety and efficacy standards in place need to be upheld.  He went on to explain that breakthrough therapies are highly targeted drugs for specific populations that can’t necessarily follow the traditional path of drug approvals, and that there may be ways to expedite those processes. 

Panel two also featured a variety of witnesses who spoke to topics ranging from the regulation of medical gases to antibiotic development.  Dr. John Powers of the George Washington University School of Medicine discussed needing to treat patients and their symptoms rather than organisms, and stated that the number of drugs approved by the FDA is not a measure of public health.  When asked by Chairman Pitts why accelerated approval has not led to gains in treatments for rare disorders, Dr. John Maraganore, CEO of Alnylam Pharmaceuticals, said it speaks to the need for clarity Dr. Woodcock talked about in panel one.  Repeating a question from earlier in the hearing, Rep. Pitts also asked if the FDA is seeking to limit the use of accelerated approval.  Dr. Maraganore echoed his previous statement about the need to clarify accelerated approval, and time and cost are also significant barriers to getting innovative medicines to market.

Panel Three:

Shawn Brown, Vice President, State Government Affairs, Generic Pharmaceutical Association; Elizabeth A. Gallenagh, JD, Vice President, Government Affairs, General Counsel, Healthcare Distribution Management Association; Tim Davis, Pharm.D., Beaver Health Mart Pharmacy, on behalf of the National Community Pharmacists Association; Allan Coukell, Director, Medical Programs, Pew Health Group, The Pew Charitable Trusts

Panel three focuses mainly on issues of supply chain safety, counterfeit drugs, prescription drug distribution, pedigree, and track and trace programs.  Allan Coukell of the Pew Health Group of the Pew Charitable Trust, described several instances of stolen and counterfeit drugs, including temperature sensitive insulin stolen and reappearing later in pharmacies and the recent incident of counterfeit Avastin making its way to U.S. clinics. 

Rep. Pitts asked all the panelists how to ensure safety in cost effective ways and whether a national model is the best model.  The panelists agreed that a national model would be the best and most cost effective way to deal with this problem, as opposed to a state-by-state patchwork that is inefficient and potentially more expensive.