Bay Area Reporter - June 8, 2001
Matt Sharp, Survive AIDS Writers Pool

Activist pressure forced the Food and Drug Administration to streamline the bureaucratic drug approval process and provide new investigational AIDS drugs before they had completed rigorous clinical trials. At a time when AIDS drugs were few and far between, when people with AIDS were desperate for treatments, there needed to be a mechanism to safely access new drugs. People with AIDS simply could not wait the typical 10 to 15 years for a drug to be approved. So, activists pushed the FDA for broader access to drugs that had proven safe, if not yet effective, in order to give people a chance at slowing HIV. Thus, expanded access was born.

At least on paper, different access programs were soon developed to widen availability to people that may not qualify for studies and who desperately needed new drugs. The programs, overseen by the FDA, are called "treatment IND" (investigational new drug), "parallel track," and "open protocols." Whatever the programs are called, and whatever they have evolved to be, they are all basically used to broaden access to investigational agents before rigorous trials are completed.

Codification (official regulation) of the programs didn't happen until 1987. And even today there are misunderstandings with the programs, the time-consuming paperwork, and the reluctance of the pharmaceutical industry to participate. Activists have had to monitor each new drug on the radar screen and lobby for wider access. Drug companies don't seem to want to offer free unproved drugs on their own mostly because the programs are formidable, cost an enormous amount of money, provide little data to further the new drug application for final approval, and there are possible liabilities involved.

In the earlier days the parallel track program for d4T or Stauvidine, offered 12,000 patients free drug. The expanded access program for 3TC or Hivid, offered drug to nearly 30,000 patients. Protease inhibitor expanded access programs, however, were much more limited in scope - if they existed at all - as the companies began using the programs as a means to promote their drugs right before they became approved. Unfortunately, today's access programs are nowhere near their original intent and have become watered down, evolving into mere public relations campaigns for the companies and little help for those who need new options. However, the number of new approved drugs that have become available has reduced the desperation for investigational agents, though those who are long-term survivors still need new drugs.

One recent expanded access program is a perfect example of how drug companies have limited their programs, timing them toward a targeted approval date. Gilead opened its expanded access for tenofovir, its new antiviral, in January. But it wasn't until May that the company began admitting more people because activists reported slow enrollment from patients across the country. Showing poor timing, the company announced shortly thereafter the submission of a new drug application to the FDA, signaling that the drug was near approval. That left only a few months for the program to enroll a few people, certainly much less than if the company had been on the ball. Gilead has no doubt had a difficult time with expanded access programs in the past, and is perhaps trying to save money by offering limited access so near the date of tenofovir's approval.

But this is one case of expanded access gone bad, for the detriment of everyone concerned.

Another promising drug, T-20, written about in past B.A.R. columns, has such a dismal prospect for wider access that activists are asking the company not to even call the program "expanded access." Trimeris, in a partnership with Roche, has taken on a massive scale-up project to make just as much drug needed to get through studies and provide data. The drug is apparently extraordinarily difficult and expensive to make and not many production facilities exist to churn out even enough drug for studies. So the company has been extremely delicate with activists, forewarning them about the production difficulties. An access program for a drug in this class, or other drugs today, would not have to be the size of d4T or 3TC programs in the past. Nevertheless, this drug is probably the most promising on the horizon and Trimeris must do everything in its power to make enough drug for as many people as possible. Approval may happen before most people will be able to access the drug.

Despite the pitiful state of expanded access, at least the FDA has sped up the drug approval process for agents that affect life-threatening diseases. Drugs that are studied for these conditions can be approved much earlier than in the past. AIDS activists were instrumental in forcing the change in licensing so that it wouldn't take years to get access to a life saving therapy.

Recently the FDA announced new codification for its compassionate use programs, which is another way to access drugs. Clarification for the programs needed to happen, and now compassionate use along with expanded access may help thousands who are in the late stages of HIV disease.

With wide availability of HIV drugs today, some feel there is less need for expanded access programs as there once was. Some feel that all the drug access we've had before research is completed has led to resistance and drug toxicities in thousands who could have waited. But others think they wouldn't be here today had they not been able to string along treatments as soon as they became available. Now, there is a gap in investigational drugs, so people that are resistant and beginning to fail will need new options, and more - and therefore better - access.
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