Exploring the PDUFA V agreement: Increase staff for rare disease drug development efforts

As you probably already know, The Pink Sheet and FDA News reported that the FDA and industry representatives from PhRMA and the BIO have reached an agreement on the reauthorization of the Prescription Drug User Fee Act (PDUFA V) - the first major milestone in the reauthorization process. The letter deals with a lot of technical issues, and we have been working to explore what these issues really mean for patients. Today, we are tackling an issue that is important to almost 25 million patients:

Increase staff for rare disease drug development efforts

Advancing development of medicines for rare or orphan diseases is important to the nearly 25 million patients who have a disease that is considered "rare". A rare disease is defined as a disorder affecting less than 200,000 people in the United States. Many rare diseases have unmet medical needs with no approved medical therapies. According to FDA, the number of product designations under the Orphan Drug Act has increased dramatically since 2002 -- from 62 designations in 2002 to 160 designations in 2009. The FDA predicts the number of rare disease marketing applications will increase in the coming years due to rapid advances in molecular biology and targeted pharmaceutical and biotechnology products. According to the September 2010 PDUFA V negotiation minutes, the FDA has only one full time staff member devoted to the development of policies and procedures to support and accelerate rare disease drug development.

According to the March 31, 2011 meeting minutes, the new PDUFA technical agreement tentatively includes a provision to advance the development of drugs for rare diseases. The proposal, which was outlined in the January 10, 2011 minutes, includes more support for guidance and policy development related to rare disease drug development, outreach to patient organizations and industry, a public meeting to discuss complex issues in clinical trials, and additional training of FDA staff. FDA will have evaluative tools to measure the success of these activities.

Timely access to new, innovative medicines is good for all patients in need. This proposed program will help meet that goal. The U.S. should be the global leader in discovering and delivering these new, lifesaving cures and treatments.