By Amanda Conschafter, Blog Editor
Should seriously ill patients who are running out of time – and have exhausted available medical treatments – have the right to try drugs not yet approved by the FDA? The debate over expanded access, dubbed “compassionate use,” prompted three United States senators to request the FDA’s data on the program last month. Their inquiry came on the heels of Colorado’s new “right to try” law, marking the third state this year to allow its citizens to access investigational medications without the FDA’s permission. Meanwhile, social media and high-profile news coverage continue to propel stories of compassionate use into the public eye.
But expanded access represents an intersection of competing needs and tough ethical considerations – posing hard questions for physicians, patients, legislators and drug manufacturers.
For physicians, expanded access begins as a question of suitability. Has the patient exhausted all other available therapies? Does the probable risk of disease progression overshadow the inherent risk of taking an unproven medication? Does the patient understand such risks, and is he or she prepared to sustain them?
If the physician and patient opt to move forward, the physician must then submit a request to the FDA on the patient’s behalf. The FDA has final say, and data suggests that the agency seldom denies a request. But the FDA cannot compel a pharmaceutical company to expand access to one of its medications. And for pharmaceutical companies, the issue takes on many dimensions.
To maintain a compassionate use program, a company needs personnel to process and answer medication requests, research new conditions, communicate with the FDA, and collect safety data from compassionate use patients. When limited resources require these staff to be pulled from existing drug development teams, a product’s long-term success can be impacted. As Pfizer CEO Ian Read recently explained, the pharmaceutical industry continues to work with the FDA and other stakeholders to “improve and to simplify the access of people to compassionate use, balancing it with the ethical needs to run clinical trials that will produce the evidence that will allow a larger population to use those medicines.”
In addition, limited quantities of investigational drugs, particularly biologics, sometimes pose the challenge of equitable distribution, requiring companies to pick winners and losers. Many manufacturers hesitate to take on such painstaking decisions.
Policymakers will continue considering how to balance these issues moving forward. Meantime, physicians must approach expanded access on a patient-by-patient basis, evaluating need, risk, medical history and the value of compassionate use for their patients.