Promising Pathways Act

[Buglaw]

Can someone explain to me how this is a good idea? It seems like the idea is to conditionally approve drugs that fail their trials? I mean, the FDA recently approved 1 drug off of one phase 2 that was barely statistically significant and another drug that failed it’s phase 3 but had great bio marker data (and does appear to be significantly more effective than the other meds). It seems like the fda is committed to approving all drugs that show themselves effective. It seems to me that the problem is that we don’t have good drugs, not that the FDA is doing a bad job. I’m not aware of any med with evidence of effectiveness fror ALs that is not being approved. Am I missing somethin?

 

[Nikki J]

I don’t think so. It is already happening I think. Qalsody is provisional contingent on ATLAS results though it did have a phase 3. The phase 3 wasn’t long enough. Someone from biogen admitted this publicly Instead of having to wait for atlas or do another trial it is provisionally approved. An unintended consequence is some insurers refuse to pay saying it is unproven

Relyvrio was approved after phase 2 so an example of what promising pathways wants. Not exactly contingent but there was pressure at the adcomm for them to promise to withdraw if the European phase 3 fails

I agree the fda isn’t the problem people think it is. Some will disagree because of Nurown but I think the fda had it right. They really don’t have the evidence yet. The briefing document laid it out. Years of hype and press releases led to unrealistic expectations in my opinion

 

[lgelb]

I agree with you both. It's sad that mugging for the cameras is considered more important than supporting clinical development (that includes R&D to better understand the mechanisms of disease) on the ground that could actually surface more safe/effective meds. And it's sad that so much P/CALS energy goes into pushing programs that are not scientifically baked.

 

[Mana]

Thank you all. I'm relieved by your responses to the push for new "miracle drugs" My husbands mantra is "more good days" . We feel that there should be more emphasis on care than on cure and more care taken with drug approval.