World Orphan Drug Congress Explores Policies Impacting Development of Rare Disease Therapies

Risk-taking in the Search for New Therapies

04.19.13 | By Stephanie Fischer

While most of my colleagues were in San Diego last week for the PhRMA Annual Meeting, I attended the World Orphan Drug Congress in DC.  Industry leaders such as Freda Lewis-Hall of Pfizer, officials from the Food and Drug Administration (FDA) and National Institutes of Health (NIH) and representatives of rare disease patient groups discussed issues ranging from the current orphan drug pipeline to how public policies in the U.S. and abroad can affect the development, review and reimbursement of new therapies for rare diseases.

My favorite inspirational comments came from John Crowley, President and CEO of Amicus Therapeutics.   John is the father of two children with Pompe Disease, a rare and usually fatal disease, and author of the book Chasing Miracles (on which the movie Extraordinary Measures was based).  He told conference attendees that science and medicine were progressing at a terrific pace – but not fast enough.  We need sustained passion, he said, and risk-taking in the search for new therapies.

New Breakthrough Therapy Designation

Dr. Gayatri Rao, Acting Director of the Office of Orphan Products Development at FDA, highlighted a relatively new program, the breakthrough therapy designation, which was created by the FDA Safety and Innovation Act of 2012 (also known as FDASIA) and is only available for therapies to treat serious or life-threatening conditions.  Dr. Rao said of the 40 requests for breakthrough therapy designation received so far, 10 have been approved and 12 have been denied. The remaining 18 are pending.  She said that there is more engagement with senior officials during the review process for products that have been designated as breakthrough therapies. 

Former FDA Deputy Commissioner Scott Gottlieb addressed a few key public policy issues affecting the development of orphan drugs and subsequent patient access to them.  Dr. Gottlieb said the breakthrough therapy designation was a mandate for the FDA to try to accelerate drug development.  He said the pathway has real significance but is underappreciated, and is currently utilized inconsistently among divisions within FDA.  Dr. Gottlieb also stressed the importance of making sure health plans, particularly those created after enactment of the Affordable Care Act, cover rare diseases.

Biopharmaceutical Pipeline for Rare Diseases

As I mentioned in a previous post, a report on the biopharmaceutical pipeline released earlier this year found nearly 1,795 potential therapies for orphan diseases in development.   In order for any of those to reach patients, there must be a consistent, predictable and science-driven regulatory process. 

Prescription Drug User Fee Act

In addition to creating the breakthrough therapy pathway, FDASIA reauthorized the Prescription Drug User Fee Act (PDUFA) which, if implemented properly, will give patients – including those with rare diseases - access to new medicines more effectively and efficiently.  The National Organization for Rare Disorders (NORD) said FDASIA ‘includes the most groundbreaking measures for rare disease patients and their families since the Orphan Drug Act of 1983.”

The biopharmaceutical research industry, patient groups, and other stakeholders look forward to FDA’s anticipated release of draft guidance on the accelerated approval pathways, including the breakthrough therapy designation.  The Agency was directed to issue draft guidance on breakthrough therapies no later than 18 months after the implementation of FDASIA (which was signed into law in July 2012).

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