Last month, the FDA outlined their new toxicology requirements for ultra-rare diseases in their draft guidance for industry, titled “Investigational Enzyme Replacement Therapy Products: Nonclinical Assessment.” The guidance potentially allows for only three months of chronic toxicology animal studies if there are no adverse findings, which is a significant improvement in policy. These changes are more in line with the applied European standards for entering clinical trials and is less than the traditional ICH guideline requirements for other non-native protein products. The Foundation worked to address this issue in our 2014 Scientific Workshop “Rationalizing Safety Testing to Enable Clinical Studies and Approval in the US for Rare Disease Treatments” and through our Cure The Process 2 Campaign. Longer toxicology requirements in the U.S. were preventing early-stage clinical trials from starting in the U.S., delaying access to lifesaving treatments for U.S. patients.
The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy. We can do more with the science we already have and bring life-saving treatments to millions of people suffering from rare diseases.
4th International RASopathies Symposium, Seattle WA, July 17-19, 2015
North-East Legislative Conference, Newark, NJ, July 20, 2015
West Coast Legislative Conference, San Francisco, CA, July 31, 2015
Annual Rare Disease Scientific Workshop, Washington DC, September 15th, 2015
EveryLife Foundation Community Congress Meeting, Washington DC, November 4th, 2015
Rare Voice Awards Gala, Washington DC, November 4th, 2015