This is technical and somewhat esoteric. The background is that the FDA under RFK, Jr. has some odd leaders. One is Vinay Prasad, who first gained notoriety during the epidemic, provided some good analyses but also showed himself to be egotistical and thin-skinned. Mr. Prasad was fired shortly after he was initially appointed, because he ran afoul of a patient advocacy group by trying to derail a therapy for a rare and deadly disease. I have no idea why he was rehired. Mr. Prasad is on a jihad against innovative, but expensive therapies. He doesn’t have much concern about the needs of patients. My personal experience with him is that he can’t take anyone pointing out where he may have made an error.
He and several other FDA officials wrote an article in the Journal of the American Medical Association regarding approvals of cell therapies. Cell therapies typically use a patient’s or another persons modified immune cells to attack cancers or treat other diseases. They are expensive, but have proven to be life-saving for patients for whom other therapies have not worked. The FDA has generally been very flexible in regard to the evidence needed to approve such therapies. That is changing according to this article. (JAMA Article)
Another piece of background is that the US has a very serious health care cost problem–drug costs are a big part of that. The surest way to reduce those prices is to create competition. So what does this new proposed policy do–make it harder for there to be competition for existing cell therapies. Instead of just showing effectiveness on a stand-alone basis, applications for approval of new cell therapies will have to be tested against existing therapies, including existing cell therapies. This is just stupid and frankly, the FDA should never be requiring that a particular therapeutic is equal to or better than existing therapies in order to get approval. That is fundamentally protective of the existing therapies, the manufacturers of which can then keep prices high. This isn’t the biggest problem in US health care costs, but it is reflective of a dumb approach that fails to consider the bigger picture in regard to costs.
“the FDA should never be requiring that a particular therapeutic is equal to or better than existing therapies in order to get approval.”
I thought that was long standing policy. As I understand it, Typically a new treatment is tested against existing treatments, if such exist. There is definitely a certain logic to saying that a treatment should not be approved if it will hurt people relative to what is already available.
Actually, they are usually tested against placebo, sometimes against standard of care. Think of the statins, or blood pressure drugs, etc. There ended up being multiple statins, which lowered the price for everyone. “hurting people” is a safety issue, not an efficacy issue. My concern is mostly the suggestion from the FDA that the cost of a therapeutic would be considered in approval decisions or the evidence required for approval. That has typically been left to payers to deal with. And it is pretty obvious that the more therapeutics for a particular disease, the more competition there is and the lower prices should be. If a product truly has low efficacy, clinicians won’t prescribe it and manufacturers will stop development. So the FDA should really mostly be concerned with safety, to some extent with some showing of efficacy, but leave it to the clinicians to decide what therapies make the most sense for a patient.
Kevin, very insightful & educational!
So industry defeated FDA’s intention to regulate LDT’s, which to me is a corollary analogy (in an era of precision medicine) for unique therapeutics like you describe in your summary. Is there there potentially a groundswell by patients, advocacy groups, industry and experts to obtain a similar result for CAR T-cell innovations as was secured for LDT’s? Seems to me that not requiring to be benchmarked against FDA-approved currently commercialized therapeutics could help continued breakthrough innovation in this golden era of biologicals.
I don’t know what FDA will end up doing, it is hard to figure out the agenda of the new people in various divisions. I understand being concerned about safety and efficacy, but cost considerations have typically been dealt with by payers, especially Medicare, so FDA should not be thinking about what a therapeutic might cost in evaluating whether it should be permitted to be marketed.. It is beneficial in terms of cost to have as many therapeutics as possible aimed at the same disease. having lots of statins helped bring the cost of those down. So as long as a therapeutic has some efficicacy, it should be approved and let the clinicians and patients decide what they think is the best one for them.