7 ms·

PDUFA and MDUFA (user fees) have no influence on FDA decision making. They are basically pocket change compared to the cost of bringing something on to market.

PDUFA and MDUFA (user fees) have no influence on FDA decision making. They are basically pocket change compared to the cost of bringing something on to market. Many think the fees are too low and ought to be increased. Surrogate measures are a trade off that we as a society have decided to make to reduce regulatory burden and accelerate brining new therapies to market. Maybe you disagree with it, but someone with a terrible disease waiting on a new life saving treatment might see the issue differently. In some cases it is clear that the surrogate is a good endpoint. In others maybe less so. Regulators have the tough job of balancing the risks, benefits, and science when making the call on whether to accept one. Generally FDA is doing many things to streamline and accelerate the regulatory process. This is good. We want them to innovate and take advantage of new scientific knowledge and technological breakthroughs. Finally I would say that the real precedent for this was the decision for the Duchenne Muscular Dystrophy drug (also happened on Woodcock’s watch). I kind of had this sinking feeling this was about to happen when I saw that she was acting commissioner. Say what you will about Trump, but Gottleib was a damn good appointment - at least until he got fired for going after vaping. Azar was a pussy and basically an empty suit.

forcry 5y ago
> trade off that we as a society.. >Regulators have the tough job of balancing the risks, benefits, and science when making the call on whether to accept one. I am curious. How much of the society was involved in making this decision. We can all agree that there is enormous financial pressure from the industry to make such decisions go in its favour, and virtually none to go in favor of the public. So isn't there a chance that eventually such trade offs will go in a direction that is harmful to the public? >Maybe you disagree with it, but someone with a terrible disease waiting on a new life saving treatment might see the issue differently. Sure, but we don't do things this way. You can always find someone who benefits from a community wide applied bad idea. Does not justify the bad idea sadly. This kind of short term, localised thinking might allow the regulatory process to bend heavily under the aforementioned financial pressures, in favour of the industry, and result in great public harm that might go unnoticed for a very long time..
jrochkind1 5y ago
Right, that's the opinion that supports the FDA here, that probably most readers are used to hearing. It is, however, decreasingly the universal consensus among doctors, scientists, and others, there is much more dissent these days, opinion that the FDA is approving drugs they shouldn't be -- as demonstrated by the very unusual resignations from the FDA committee. Those scientists thought it was more questionable than your story. As far as patient advocates, as many pieces point out, one of the historical things was AIDS activism for availability of drugs. I was struck by this from a recent piece about ACT UP, from one of the activists at that time: «Today, Franke-Ruta is a journalist, and she spoke to Schulman about the wider implications of some of act up’s success. “I don’t think that we realized at the time that this was part of the broader gutting of the FDA that we’ve seen since; that there was a lot of political agendas that we just happened to be in sync,” she said. After President Donald Trump touted the promise of hydroxychloroquine as a treatment for covid-19, the F.D.A. issued an emergency-use authorization for the drug, which was shown to be useless for the purpose. (The agency withdrew the authorization three months later.) “Sometimes it seems like it’s gone too far in the other direction,” Franke-Ruta went on. “But there’s a really strong pharmaceutical lobby against the FDA as well that I don’t think we were aware of.”» And from the article's author, editorializing a bit: «In retrospect, one can ask whether act up’s victories on the research front pushed the F.D.A. too far. The drugs-into-bodies approach to fast-tracking—the use of “surrogate endpoints” (like T-cell counts or viral load), for example, rather than clinical benefits in actual people—can be valuable, especially for patients who are facing death and have no good alternatives. This approach at least offers desperate patients a chance, while allowing scientists to gather meaningful data. But, today, a number of drugs, for everything from asthma to periodontitis, have won approval before benefits in human patients were established, and critics argue that drug approval is too often based solely on benefits shown in biomarkers rather than in bodies. “Right to try” legislation, meanwhile, enables the sale of drug candidates without even involving the F.D.A. When restrictions are weakened, experimental drugs—many of which end up proving useless or worse—become harder to distinguish from effective medicine.» https://www.newyorker.com/magazine/2021/06/14/how-act-up-changed-america https://www.newyorker.com/magazine/2021/06/14/how-act-up-cha...

forcry 5y ago

> trade off that we as a society.. >Regulators have the tough job of balancing the risks, benefits, and science when making the call on whether to accept one. I am curious. How much of the society was involved in making this decision. We can all agree that there is enormous financial pressure from the industry to make such decisions go in its favour, and virtually none to go in favor of the public. So isn't there a chance that eventually such trade offs will go in a direction that is harmful to the public? >Maybe you disagree with it, but someone with a terrible disease waiting on a new life saving treatment might see the issue differently. Sure, but we don't do things this way. You can always find someone who benefits from a community wide applied bad idea. Does not justify the bad idea sadly. This kind of short term, localised thinking might allow the regulatory process to bend heavily under the aforementioned financial pressures, in favour of the industry, and result in great public harm that might go unnoticed for a very long time..

jrochkind1 5y ago

Right, that's the opinion that supports the FDA here, that probably most readers are used to hearing. It is, however, decreasingly the universal consensus among doctors, scientists, and others, there is much more dissent these days, opinion that the FDA is approving drugs they shouldn't be -- as demonstrated by the very unusual resignations from the FDA committee. Those scientists thought it was more questionable than your story. As far as patient advocates, as many pieces point out, one of the historical things was AIDS activism for availability of drugs. I was struck by this from a recent piece about ACT UP, from one of the activists at that time: «Today, Franke-Ruta is a journalist, and she spoke to Schulman about the wider implications of some of act up’s success. “I don’t think that we realized at the time that this was part of the broader gutting of the FDA that we’ve seen since; that there was a lot of political agendas that we just happened to be in sync,” she said. After President Donald Trump touted the promise of hydroxychloroquine as a treatment for covid-19, the F.D.A. issued an emergency-use authorization for the drug, which was shown to be useless for the purpose. (The agency withdrew the authorization three months later.) “Sometimes it seems like it’s gone too far in the other direction,” Franke-Ruta went on. “But there’s a really strong pharmaceutical lobby against the FDA as well that I don’t think we were aware of.”» And from the article's author, editorializing a bit: «In retrospect, one can ask whether act up’s victories on the research front pushed the F.D.A. too far. The drugs-into-bodies approach to fast-tracking—the use of “surrogate endpoints” (like T-cell counts or viral load), for example, rather than clinical benefits in actual people—can be valuable, especially for patients who are facing death and have no good alternatives. This approach at least offers desperate patients a chance, while allowing scientists to gather meaningful data. But, today, a number of drugs, for everything from asthma to periodontitis, have won approval before benefits in human patients were established, and critics argue that drug approval is too often based solely on benefits shown in biomarkers rather than in bodies. “Right to try” legislation, meanwhile, enables the sale of drug candidates without even involving the F.D.A. When restrictions are weakened, experimental drugs—many of which end up proving useless or worse—become harder to distinguish from effective medicine.» https://www.newyorker.com/magazine/2021/06/14/how-act-up-changed-america https://www.newyorker.com/magazine/2021/06/14/how-act-up-cha...