It is the sense of the U.S. Congress that the Food and Drug Administration (FDA) should apply accelerated approval and fast track provisions set forth in a new amendment to the Federal Food, Drug, and Cosmetic Act (FDA Law). The Congress finds that accelerated approval will to a greater extent help expedite the development and availability to patients of treatments for serious or life threatening diseases or conditions while maintaining appropriate safety and effectiveness standards for such treatments. Congress has long looked to strengthen FDA, as a means to help the U.S. remain competitive, and very appropriately engage in the discussion at a time of Prescription Drug User Fee Act (PDUFA) reauthorization and the Presidents 2013 budget proposal.

What could this mean to drug development?

1. The Food and Drug Administration (FDA) serves a critical role in helping to assure that new medicines are safe and effective. Regulatory innovation is one needed element of the nation’s strategy to address serious and life-threatening diseases or conditions by promoting investment in and development of innovative treatments for unmet medical needs.

2. Over the previous two decades since the accelerated approval mechanism was established, advances in medical sciences, including genomics, molecular biology, and bioinformatics, have provided an unprecedented understanding of the underlying biological mechanism and pathogenesis of disease. A new generation of modern, targeted medicines is currently under development to treat serious and life-threatening diseases, some applying drug development strategies based on biomarkers or pharmacogenomics, predictive toxicology, clinical trial enrichment techniques, and novel clinical trial designs, such as adaptive clinical trials.

3. As a result of these remarkable scientific and medical advances, FDA should be encouraged or incentivized to implement more broadly effective processes for the expedited development and review of innovative new medicines intended to address unmet medical needs for serious or life-threatening diseases or conditions, including those for rare diseases or conditions, using a broad range of surrogate or clinical endpoints and modern scientific tools earlier in the drug development cycle when appropriate. This may result in fewer, smaller, or shorter clinical trials for the intended patient population or targeted subpopulation without compromising or altering FDA’s existing high standards for the approval of drugs. It may further positively impact sponsor costs associated with clinical development. 

4. Patients benefit from expedited access to safe and effective innovative therapies to treat unmet medical needs for serious or life-threatening diseases or conditions.

5. For these reasons, the existing statutory authority governing expedited approval of drugs or serious or life-threatening conditions should be amended in order to enhance FDA’s authority to consider appropriate scientific data, methods, and tools, and to expedite development and access to novel treatments for patients with a broad range of serious or life-threatening diseases or conditions.

FDA lacks resources

The discussion come at a time as Congress considers the Prescription Drug User Fee Act (PDUFA) reauthorization bill regarding financing the agency in the next user-fee cycle from 2012-2017. The agency lack resources following expanded authority and post-market commitments from previous PDUFA-cycle, and FDA surely advocates for increased funding to ensure and meet PDUFA deadlines, etc. However, many stakeholders worry, that both the reauthorization of the Prescription Drug User Fee Act and the newly presented 2013 budget proposal by President Barack Obama, though including higher funding for the FDA, suggests that the amount provided by the federal government would remain flat while the proportion provided by user fees would rise to 44%. The agency does not have the same flexibility with user fees as it does with federal funds, and that has some advocates worried about the ability of the agency to innovate. The user fees pay for very specific services and commitments between the sponsor and the agency, and they are really additive to what the FDA is responsible for accomplishing.

As such, the on-going discussion should be seen in a broader perspective of policymakers wishing to strengthen the FDA to its former position as the world's leader in medical product evaluation as Congress considers the Prescription Drug User Fee Act reauthorization bill.

Congress are not only trying to set the fees and add resources for the agency but also modernize it from the bottom up, including reviewing the agency's processes and creating pilot programs to bring promising therapies to patients more quickly.

By enacting above measures it has the potential to encourage the Secretary to utilize innovative approaches to the assessment of products under accelerated approval while maintaining appropriate safety and effectiveness standards for such products to the American people. Further, it provides the Secretary with a handle to instill confidence in U.S. products and technologies, stimulate economic growth and open markets overseas.

joamik@um.dk