Vincent Rajkumar on the Importance of Accelerated Approval for Cancer Treatment

Vincent Rajkumar, Editor in Chief of Blood Cancer Journal, shared on X:

“On Accelerated Approval

1. In life-threatening diseases like cancer the rapid approval of promising new drugs based on phase II data and/or surrogate endpoints is reasonable because waiting for full approval may result in more lives lost due to delay in approval of active drugs.
2. The mechanism is not meant to be 100% fool proof because with such a high bar sensitivity will be low and many truly active drugs will be rejected incorrectly or greatly delayed. But it needs to be as rigorous as is reasonable so we don’t approve too many ineffective drugs.
3.  Accelerated approval is predicated on confirmatory trials being done in a timely manner. Failure to do so should usually result in withdrawal of the accelerated approval. The FDA is currently being quite strict on this aspect.
4. Important: If a confirmatory trial is negative that doesn’t mean the whole accelerated approval pathway is at fault. A small proportion of confirmatory trials will be negative — that means we have followed the principles of accelerated approval (see second tweet in thread).
5. Important: If a confirmatory trial is not done in a timely manner that doesn’t mean the accelerated approval pathway is wrong. It means either regulators for a good reason have allowed a delay (or lack) of such a trial; or lacking such a reason it means they need to enforce.
6. Important: If a drug approved is priced exorbitantly it doesn’t mean the whole accelerated approval pathway is at fault. It means we have to separately reform the way drug prices in the US are set to be proportional to the added value they provide. It is a concern.
7. Important: The purpose of accelerated approval is to help patients. That is our ONE and ONLY goal.We do not want a net loss of lives because we are focused on being text-book perfect. It’s a risk benefit judgment call.
8. Accelerated approval thus balances the benefit of having access to effective drugs early versus the risks of incorrectly approving a useless or harmful drug. Done right, in a disease like myeloma with reliable biomarkers, accelerated approval saves a large number of lives. Read more about it here.
9. Disease matters. Extrapolating failures from various hard to treat cancers where response often doesn’t equate clinical benefit (either because the measurement is flawed or the benefit achievable with our current understanding is small) to all cancers is a fallacy.
10. Finally the route to accelerated approval requires expertise and experience in a given disease and the risks and benefits of interventions and the endpoints.

For more details specifically on myeloma check out this thread.

It requires collaboration of regulators, Pharma, investigators, patients, and patient organizations.”

You can also read other contributions by Vincent Rajkumar published on OncoDaily.