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FDA Opens Door a Sliver to Gain Access to Investigational Drugs: New Policy’s Success Depends on Manufacturers.
| Content Provider | Semantic Scholar |
|---|---|
| Author | Barlas, Stephen |
| Copyright Year | 2009 |
| Abstract | The FDA’s decision in mid-August to clarify its rules on when critically ill patients can gain access to investigational drugs did not make much of a news splash. Maybe that was the FDA’s intention, because announcing a “clarification” of existing rules that widens access only marginally is unlikely to become headline fodder when half the nation is on vacation. The agency’s two final rules—one setting new criteria for access to experimental drugs, the other dictating when drug companies can charge patients for those unapproved drugs—are expected to increase the number of people who will be able to obtain these drugs by just a nudge over 3,000 a year. That is the FDA’s estimate. Frank Burroughs is President of the Abigail Alliance for Better Access to Developmental Drugs, an advocacy group in Fredericksburg, Virginia, that strives to make more treatment options available to patients with life-threatening illnesses. The alliance had sued the FDA to get wider access to therapies. Mr. Burroughs had tried to obtain medications for his daughter, who died without gaining the benefit of experimental agents. He says that he is happy that the changes are in the “plus column” but also states that the FDA “missed an opportunity.” However, the FDA and even some cancer advocacy groups were worried that allowing greater access to investigational drugs outside clinical trials would sabotage the goal of increased enrollment in the trials. Janet Woodcock, MD, Director of the FDA’s Center for Drug Evaluation and Research, says: We understand that some patients have run out of options and want to try something that is not fully tested, and we want to support them in these situations without exposing them to undue risks. But we also need to make sure that ultimately all patients get a treatment that has been shown to work. The clinical trial process gives everyone the full picture on the safety and effectiveness of a drug before it is used in the population at large. Linah Lubin, spokeswoman for the National Coalition for Cancer Survivorship (NCCS), says much the same thing: We believe that it is critical to enforce and implement the standards of expanded access programs rather than simply making investigational drugs more widely available. We hope that the recent FDA ruling will better ensure that the needs of cancer patients are met and [that] accrual to ongoing trials is not impaired. The concern about hurting clinical trial enrollment motivated the FDA to clear up a few ambiguities to make it a bit easier for some physicians to gain access to these novel agents outside clinical trials and for pharmaceutical companies to get paid for providing them. This change was especially pertinent for physicians treating patients with conditions other than cancer, such as Lou Gehrig’s disease (ALS) and Parkinson’s disease, and for doctors treating largely minority populations. The FDA policy ostensibly improves access for these physicians and patients by establishing two new formal categories of patients who can have access—individuals and small groups—and by setting formal criteria that patients in those categories must meet. The final rule also sets clearer criteria for already approved large groups (organized by the pharmaceutical companies) of patients who have applied for and have been approved to receive treatment with an Investigational New Drug (IND). In the past, patients who had fit into either of the two newly endorsed categories could sometimes receive investigational drugs if their physicians knew how to work the system (which most don’t). Establishing formal criteria for the two groups might make little difference. There might be a better chance of gaining access when a drug company files a Treatment IND application. Theoretically, this filing would allow many people to receive an unapproved drug if they are not enrolled in a clinical trial. Because this is where the big opportunity lies, the FDA has set the strictest criteria. The FDA has mandated that before a company can set up an expanded access program as part of a Treatment IND, it must (1) assure the FDA that a co-occurring clinical trial has sufficient enrollment, (2) provide evidence of adequate progress in the development of the drug for marketing approval, and (3) satisfy the agency on some other scores. The larger question: will pharmaceutical companies create more expanded access programs? The answer probably depends in part on whether the companies find the FDA’s “fee-charging” rule suitable. If a pharmaceutical company meets the Treatment IND requirements, it can charge patients according to its “direct” costs, as specified by the FDA. When the FDA published the proposed rule on fees in 2006, the Pharmaceutical Researchers and Manufacturers of America (PhRMA), the brand-name industry trade group, criticized the direct-cost formula and instead asked the FDA to allow companies to charge based on “administrative” costs. In the final rule, the FDA rejected that request. Asked about the final rule, a PhRMA spokesman provided a noncommittal statement from Ken Johnson, a senior vice president at PhRMA, and essentially said that individual companies would make their own decisions. Meanwhile, Frank Burroughs and other members of the Abigail Alliance met with FDA Commissioner Margaret Hamburg, MD, and her top deputy, Joshua Sharfstein, MD, in September. The two final rules concerning expanded drug access were shaped by the FDA during the recent Bush administration. Mr. Burroughs hopes that he and others can convince the Obama FDA to widen access even more. He notes that Dr. Sharfstein, who headed the Obama FDA transition team, consulted him before the new administration took office. “We have a good relationship with them,” Mr. Burroughs says. “I’m optimistic.” |
| Starting Page | 528 |
| Ending Page | 529 |
| Page Count | 2 |
| File Format | PDF HTM / HTML |
| Volume Number | 34 |
| Alternate Webpage(s) | https://www.ptcommunity.com/system/files/pdf/ptj3410528.pdf |
| Language | English |
| Access Restriction | Open |
| Content Type | Text |
| Resource Type | Article |
