The FDA has a mandate to approve new drugs. Now this is a difficult process, especially as newer therapeutics are developed. Fundamentally there are three steps:
1. Does the new therapeutic cause no harms?
2. Is the new therapeutic effective. Namely does it do what it is supposed to.
3. Is the new therapeutic better than what is already available. Namely efficacy.
This may sound simple but to perform these tasks one needs a large enough statistical sample to show it is true or not. Now step 1 can be easy, relatively. But safe to whom? The normal healthy person or the ones with the disorder? Step 2 requires a large enough group of those with the disorder willing to be tested over some period of time. That can be difficult if the disease is rare. Step three is an A to B comparison. That means we have to test this perhaps for two or three years.
Now adding all these up leads to a five to ten year lag in approval. The FDA has found ways to expedite but no matter what there are always people who are harmed, often a small percent, bu always there. Then again with many of the new cancer immunotherapeutics we see 60% to 80% who are non responsive. Why? Not fully understood. Yet we utilize the therapeutics any way.
The challenge to an incoming FDA/HHS is to understand that we are in a major tectonic change in therapeutic introductions and approval. In a sense the mRNA vaccines are a now classic example. Unlike the classic smallpox vaccine, the Salk polio vaccine etc the mRNA vaccines are good at best for 6 months. The older ones are for a lifetime. Why? One would have thought that the NIH folks would have spent some time on that issue but no! Blame the head of the sub-agency I guess.
The incoming team, whoever is approved, must be open to face the challenges. As we learn more we recognize that the "unknown unknowns" start to dominate. There must be a willingness to move forward while still accepting we are dealing with a complex environment, the human body.
Posts
