Drugs in the Pipeline and the FDA

Here's a piece at Reason online about experimental drugs for terminally ill patients and the debate concerning those patients' rights to access those drugs before they have been FDA approved.

The issues here concern the safety and efficacy of new drugs offset by urgent needs of these patients for potentially life-saving or life-extending drugs.

In the hemophilia community we're always hopeful for new factor products. We hope in the near term for factor with longer half-lives or oral or topical factor products. We hope eventually for a cure. But new pharmaceuticals take time and research and clinical trial and lots and lots of money. But does it take too much time? Can more new drugs be fast-tracked, saving time and costs that you know will end up being paid by the end-user (or rather the third party payer).

With good reason this community is cautious of product safety and is suspicious of product sales taking precedence over product safety. It’s a delicate balance to want the product improvements from the very industry that many still mistrust.

I don't know the answers. I know I want a cure for Jack as soon as possible And in lieu of that I want better and easier factor products. But at what risks?