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Compassionate Use Debate Heats Up


Pharmaceutical Executive

Patient access to critical experimental medicines continues to grab public attention, as states enact “Right-to-Try” laws and Congress eyes establishing a national policy to provide not-yet-approved therapies to terminally ill patients

Patient access to critical experimental medicines continues to grab public attention, as states enact “Right-to-Try” laws and Congress eyes establishing a national policy to provide not-yet-approved therapies to terminally ill patients. The Food and Drug Administration and biopharmaceutical companies are busy explaining how existing expanded access programs (EAPs) operate and the risks and difficulties of broader use of experimental medicines.


State compassionate use measures do little to actually provide unapproved medicines to seriously ill patients; the main thrust is to encourage physicians to seek expanded access approval from FDA. The bill approved by Arizona voters in November, for example, allows a patient’s physician to recommend an experimental therapy and permits manufacturers to make a drug available without going through FDA’s expanded access process. But it does not require biopharma companies to do so, and FDA policies are not considered an obstacle for manufacturers willing and able to provide test therapies to patients. This latest bill from Arizona is similar to others enacted in Colorado, Louisiana, Michigan and Missouri and to new ones proposed in Texas and Wyoming.


In response to state initiatives that threaten to nullify or override FDA laws and regulations, some members of Congress are proposing a national policy to facilitate early access to critical therapies. The Compassionate Use Reform and Enhancement (CURE) Act sponsored by Rep. Michael McCaul (R-Tx) would require FDA to clarify the process for patients and physicians to request early access and for manufacturers of  “covered breakthrough drugs” to inform FDA of their own expanded use programs, including company procedures for approving or denying requests. The Government Accountability Office (GAO) would analyze FDA’s EAP and a Congressionally appointed Expanded Access Task Force would develop recommendations for program improvement. FDA will incorporate all these proposals into EAP guidance.


Meanwhile, FDA officials continue to emphasize the agency’s long history with compassionate use. At the recent conference on “Advancing  Ethical Research” sponsored by PRIM&R (Public Responsibility in Medicine and Research), Richard Klein of FDA’s Office of Special Health Issues noted that FDA approves virtually all expanded access requests submitted by physicians, but acknowledged that such requests reach the agency only if the manufacturer first agrees to provide the drug. He acknowledged that EAPs can impair enrollment in clinical trials, and that treatment with some experimental products may be harmful and reduce quality of life.


Michael Rosenblatt, executive vice president and chief medical officer at Merck, emphasized his company’s commitment to meeting requests from very ill patients, even though expanded access may jeopardize drug development. Access programs can provide some safety data, but not reliably. Clinicians serving as investigators in clinical trials are first in line for access requests, he explained, followed by physicians outside trials.


The Pharmaceutical Research and Manufacturers of America (PhRMA) recently added a new chapter to its Principles on Conduct of Clinical Trials stating that appropriate use of expanded access provides an “option” for patients who are ineligible or unable to participate in clinical trials. The statement outlines how sponsors should handle requests and grant access to investigational drugs, while affirming the importance of conducting clinical trials that produce “meaningful results” to bring new medicines to patients.

“Fairness” becomes an important issue for all parties when early access for some patients interferes with development and delays treatment for a broader patient population. Compassionate use demands may be particularly difficult for small companies that lack sufficient supplies of a test product and the resources to expand production prior to approval. Europe has different policies for making experimental drugs available, which can complicate EAPs in the U.S., and a pharma company may run into legal problems when a state law calls for shipping an unapproved drug across state lines.

Although new compassionate use policies may provide false hope to seriously ill individuals, some patient advocates maintain that false hope may be better than no hope. Thus compassionate use will continue to draw public support and to raise difficult ethical issues for all parties. 

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