FDA Legislation and Regulations

Streamlining and Enhancing the Drug Approval Process
Recognizing the urgent need for breakthrough therapies for Parkinson’s disease, PAN strongly supports efforts to ensure FDA has at its disposal the necessary tools and resources to hasten the availability of much-needed new treatments and devices for Parkinson’s and other chronic diseases.  Central nervous system drugs, including therapies for Parkinson’s disease, are the slowest and most expensive in the drug development pipeline – it takes an average of 15-20 years for new therapies to get to market.    

Every five years, Congress must reauthorize legislation – the Prescription Drug User Fee Act (PDUFA) and the Medical Device User Fee Act (MDUFA) – enabling the FDA to collect fees from the pharmaceutical and medical device industries that partially fund the agency’s efforts to approve new drugs and therapies. In 2012, Congress passed the FDA Safety and Innovation Act (FDASIA), which included a number of patient-centered provisions supported by PAN.

Learn more about recent legislation and PAN’s policy work.

Regulatory Reforms

Modernizing FDA Regulation of Clinical Trials

In March 2012, the FDA issued a notice seeking input on how to modernize the regulation of clinical trials. For a new drug or device to be approved, it must go through rigorous clinical trials that test its safety and efficacy. The regulatory framework for clinical trials has a direct bearing on this and, as a community, we firmly believe that the FDA can, and must, do better.

In a PAN-led effort, the national Parkinson’s disease organizations – American Parkinson Disease Association, The Michael J. Fox Foundation for Parkinson’s Research, National Parkinson Foundation, PAN, The Parkinson Alliance, and Parkinson’s Disease Foundation – jointly submitted recommendations for improving regulation of clinical trials.  
Our recommendations focus on three overarching topics:

  1. Recognizing trial participants’ rights and access to information;
  2. Consideration of the impact and burden of Parkinson’s in trial design and implementation, including establishment of a structure and mechanisms for inclusion of the patient voice;  and
  3. Enhancing trial efficiency, with respect to both trial duration and cost.

The FDA continues to evaluate these and other recommendations in efforts to modernize and streamline clinical trials. Many of the patient-centric principles above were also incorporated into FDASIA in the summer of 2012.