Dear RS Community and other AAPS Community members:
FDA is initiating ARC program and asking comments to understand the difficulties and issues developing drugs for rare diseases. This is an area of Regulatory Innovation. We should be very proud of the FDA that is taking steps to encourage drug development in this area which very financially challenging if normal drug development regulatory path is followed.
I strongly encourage our community members, if you are working in rare disease area, post your comments here and/or directly to FDA site. We like to see that our voices are heard by the FDA so they can accommodate any regulatory innovation if possible.
if you are encountering issues in the following areas. please reflect them in reply to this post.
- Nonclinical and clinical pharmacology considerations
- Clinical trial design and interpretation
- Regulatory considerations for rare disease drug development
I see myself finding patients to enroll in the US alone is not going work well for rare diseases. Can we do rolling type of NDA where we get conditional approval for the product when benefit seems to outweigh risk and get final approval when we can reach final analysis point?
I am sure there are truck loads of issues/difficulties developing medicines in this area. Please take few minutes and express your concerns; our member ship like to see what you have to share.
Accelerating Rare disease Cures (ARC) Program
| U.S. Food and Drug Administration |
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| Accelerating Rare disease Cures (ARC) Program |
| The mission of CDER's ARC Program is to drive scientific and regulatory innovation and engagement to accelerate the availability of treatments for patients with rare diseases. |
| View this on U.S. Food and Drug Administration > |
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-Raja Velagapudi
RS Community Chair.
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Raja Velagapudi Ph.D.
Sr. Director, Clinical Development
Tolmar Inc
Monmouth Junction NJ
[email protected]Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
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