Drugs & Clinical Trials

When Experimental Drugs May Be the Only Hope

February 01, 2008 | By Lindsey Kay, MD | Share, Save, and Bookmark | 2 Comments

While the treatment options available for fatal diseases have expanded greatly in recent years, many patients do not respond to conventional therapy and are left with no therapeutic options. As an example, despite the millions of dollars that are poured into investigational research each year, cancer remains one of the most common causes of death in the U.S.

Two advocacy groups are fighting for patient access to experimental drugs, but their appeal to the Supreme Court was recently rejected. The groups argued that denying patients who had no further treatment options access to investigational drugs was unconstitutional, depriving them of life and liberty without due process. The Court justices refused to hear the case, stating that there is no constitutional right of access to experimental drugs.

That is probably true. The Constitution does not address, directly or indirectly, this right. But even without constitutionally protected access, terminally ill patients deserve the chance to try investigational therapies, with the consent and care of their doctor. While available treatment options are effective in most patients (otherwise they would not be on the market), in every disease category there are a large number of people who receive no benefit from FDA-approved options. When these patients are terminally ill, they should have the right to at least consider the use of experimental drugs.

In December of 2006, the FDA proposed changes to the regulation of experimental drugs, widening access to include individual patients and small groups of patients who failed conventional therapy. Since that announcement, the FDA has not released any further information regarding this proposal.

Many patients have access to experimental therapies through enrollment in a clinical trial. But most patients will remain unaware of this option unless their doctor offers it to them. Patients who do not live near a major academic hospital will likewise be unable to enroll in clinical trials. The FDA’s proposal to expand access is a huge step in the right direction, but the regulatory changes must go into effect, and both patients and healthcare providers must be made aware of this option.

Hundreds of patients are told each day that they’ve run out of options, that our healthcare system has nothing left to offer them. There is no reason why these patients, given clearance by their doctor and full disclosure of the risks involved, should be denied experimental therapies and the chance, however small, of receiving some benefit from them.

References

James Vicini. U.S. court rejects appeal seeking unapproved drugs. Reuters. January 14, 2008.

FDA Proposes Rules Overhaul to Expand Availability of Experimental Drugs. FDA News. P06-19, December 11, 2006.

2 Comments

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Steven Walker
February 01, 2008 | Permalink

I found this post very encouraging because Dr. Harle clearly understands the need for access to investigational drugs and some of the barriers that exist for patients trying to access them in clinical trials. However, even if all phsyicians referred their seriously- and terminally-ill patients to clinical trials, most would remian unable to participate because of restrictive entry criteria. It is simply not true that cancer patients don’t try to get in to clinical trials for their diseases. Many do try, and the great majority find they are ineligible.

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