
- Pharmaceutical Executive-10-01-2016
- Volume 36
- Issue 10
Steps Toward Rare Disease Success
When lobbying tips the scales: a path to drug approval.
This month’s National Organization for Rare Disease (NORD)
Some see Exondys 51’s approval as a questionable new precedent, deprioritizing efficacy in regulatory decisions. What steps might a new player in the rare disease space want to consider? See one path below ...
FDA is in a tough position any time it has to make a decision as heated as Exondys 51’s approval. The infighting at the agency that has been widely reported is a clear indicator of the seriousness with which it took the mission.
If a precedent has been set, let’s hope it’s for leniency toward conditional approvals that it will be willing to revoke, should post-approval, real-world evidence merit. Removing a drug like Sarepta’s from the market could once again raise intense emotion and require supreme fortitude, especially if another treatment is not yet approved-or close-offering patients hope. Also, let’s hope the decision to approve conditionally properly incentivizes the investment community to continue ventures in promising science in the risky world of rare disease.
None should malign patients, the challenges they face, nor for that matter, healthcare professionals and industry members in their laudable persistence to give them an option. Additionally, it is crucial for decisions about treatment choices to fully weigh the futures such patients face (from their own testimonials), and the fact that they often are fully willing to take burden upon themselves for a chance at a better and/or prolonged life-if not for them, for future sufferers of their affliction.
But given the new demands for greater patient involvement in the process and for real-world data to be counted, how tall a challenge will FDA face to remain credible as a reliable, sometimes calculating, but rational and precise arbiter of science, especially in election seasons? If patients can sway votes, votes may precede science. So don’t forget to write your representative.
Casey McDonald is Pharm Exec’s Senior Editor. He can be reached at
Articles in this issue
almost 9 years ago
Content Marketing’s Data Fail: Information Without Insight?almost 9 years ago
The Rise of the CFO Strategist: Balancing Numbers and Know-howabout 9 years ago
‘Huge Sellers’ Herald Changes in Japan Drug Pricingabout 9 years ago
Real-World Evidence: From Volume to Valueabout 9 years ago
Pharma and the Peril of Europe’s Trade Policiesabout 9 years ago
New Challenge to IP Innovationabout 9 years ago
Major Changes Ahead for Drug User Feesabout 9 years ago
Country Report: Czech Republicabout 9 years ago
Pharmaceutical Executive, October 2016 Issue (PDF)Newsletter
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