The FDA does need to change its existing policies for allowing access to investigational drugs when patients have no other remaining options. Unfortunately, that was not their intent when they issued the draft regulations referenced in the article. The draft regulations were prepared in response to our lawsuit as part of the agency’s legal strategy. FDA announced their intent to draft and issue the regulations, then took three years to do so, apparently waiting for the right moment in the progression of our suit. When issued for comment, the FDA also clearly stated they were doing nothing but codifying exisiting policies and practices that had been in place for decades. In our meeting with an FDA Deputy Commissioner and three other senior FDA officials (at least two of whom who were directly involved in drafting the regulations), the agency again affirmed that intent.

In the preamble to the draft regulations the FDA admits that its existing policies have never resulted in more than a few hundred patients gaining individual access in any year, or more than a few thousand in any year through their larger programs. Compared with the more than 500,000 cancer patients (excluding all other diseases) a large percentage of whom find themselves in need of access to investigational drugs, the access programs the FDA appears to set to reserve must be considered non-functional. The agency predicted that over several years, uiisuance of tghe new regulations might result in slight increases of access to a similary non-functional level.

By their own admission, they are doing nothing but clarifying their existing, inoperative policies and cementing them into regulation, which will make them even harder to change going forward. When directly asked, they admit they aren’t changing anything, but one would never know that from their brief and very cryptic press releases on the draft regulations.

Comments to the docket for the draft regulations overwhelmingly support dramatically increased access to investigational drugs, running more than about 90 percent in favor of that goal. Only a few financially vested interests (e.g., insurance carriers who think they might be required to pay for something they don’t pay for now) commented that FDA should maintain the status quo or allow even less access.

As always, the devil is in the details, and the details in this case show that FDA should amend its draft regulations to make investigational drugs available at meaningful levels to patients who have no where else to go in pursuit of continued life. Instead, they proposed to cement failing policies into place by issuing them as promulgated regulations.

In our lawsuit, there was disagreement within the appeals court that reversed itself, and among Constitutional scholars, on the merits of our claim. Two of the appeals court judges and many scholars thought the court should have affirmed the right we asserted, but even in saying no, the majority opinion was about as unequivocal as a federal court ever gets in its call for Congress to act.

We beleive Congress must act if there is to be any change in FDA policies. The FDA’s own Science Board recently concluded that the agency is incapable of changing itself, and fixing the agency’s internal paralysis is going to take many years. Obviously, millions of Americans won’t be able to wait.

We and our constituents are greatly encouraged by physicians like Dr. Earle who are motivated to speak out on this problem, and we hope Congress and the FDA are listening.

Steven Walker
Co-Founder – Abigail Alliance for Better Access to Developmental Drugs