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Portola Pharmaceuticals: What Makes A Good Drug?

Summary

Does this drug, without a doubt, provide treatment?

AAlfa reduces Anti-Factor Xa activity by a huge margin. But does this reduction directly correlate to a reduction in bleeding?

In essence, the true clinical endpoint for this study is a statistically significant reduction in bleeding.

I do research with an expectation that my findings will offer an objective review for potential FDA approval. Not just for me, but for those who might be interested in what I do. So in turn I give both sides of the coin as fairly as possible. And despite all of our earnest research, we still see promising drugs fail. I constantly ask myself what exactly the FDA wants in their drug trials. What makes a good drug?

My final conclusion/reasoning may not suffice for some of you out there, and in different shoes perhaps I would feel the same. But hopefully my thought process is clear and concise enough to leave at least a parting thought.

Before I talk about Portola Pharmaceuticals, I want to talk about study design. Here's some more reading for those interested in the topic.

It takes a good study to power results that are statistically significant. For some drugs this may require thousands of volunteers, and for others significantly less. But significance is akin to meeting the GPA requirement to apply to your favorite school. The FDA has and will continue to deny approval for things much more nuanced than statistical analysis.

Clinical endpoints drive relevance. Endpoints for research papers have quite a bit of leeway in this regard because they're meant to teach and discover; not cure. Curing a patient effectively and consistently is the only thing the FDA wants to know, and they want to know it without a doubt.

After going through these initial entry steps, we move onto things like the numbers behind the data. Sure, the drug's numbers might be statistically significant, but do they show unequivocal regard towards an actual treatment?

While reading the rest of the article, I ask you to keep in mind this one question: Does this drug, without a doubt, provide treatment?

Portola Pharmaceuticals (PTLA)

Portola's drug trial for Andexanet Alfa (AAlfa) has a very strong showing of statistical...


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