Should Patients Participating in Trials Get Free Drugs Post Approval
​[Thursday, April 18, 2019]

One of the reasons patients participate in clinical trials, particularly pivotal (Phase 3) trials, is to get access to new medication not yet approved. For many patients, particularly with life-threatening diseases who have exhausted all available therapies, a clinical trial may offer the only option available for potential benefit. And many times, it works. The patient sees a benefit from the experimental treatment. Actually, the efficacy data measured from these patients is critical for the market approval of that experimental treatment. So, should these patients get access to the same drug post-approval, free of cost? This is particularly true for rare diseases where the patient likely has no other option once the trial is over, and the approved product comes with an astronomical list price. In an interesting story on BBC, several patients who participated in the clinical trials for a new treatment for a rare genetic disease, phenylketouria (PKU), and who benefited from the experimental treatment, found that they could not afford the buy the drug post-approval, and hence went through hardships for lack of treatment. The happy ending was when the patients were able to get the drug from the sponsor probably free or at much discounted rates due to a provision of the Declaration of Helsinki, to which the UK complies.  The Declaration of Helsinki requires that sponsor provide the drug to patients who participated in their trial after the trial is over. This provision was added in 2000 to protect patient-participants from poor countries who would likely not be able to afford newly approved drugs. The US FDA abandoned Declaration of Helsinki in 2008 after strong protest from the pharmaceutical industry for obvious business reasons. So, it is not uncommon for patient-participants in the US to find out that while they play an increasingly critical role in getting drugs approved, they may not be able to afford the same drug once approved. Patients are increasingly important players in drug approval decisions particularly with the emphasis of Patient Focused Drug Development and case studies where patient activism was successfully used to influence FDA decision. But should this change? Do patients have the ethical right to the drugs they helped get to the market? These patients generally represent a very small fraction of the overall target population for the marketed drug so it cannot be too expensive for the sponsors, and it will go a long way in building good will. Some concerns have been raised about the added “incentive” for informed consent, but since there is no guarantee if a given drug in clinical trial will get approval from FDA and how long it may take to get to the market, the concerns about this inducement can be easily addressed. Should patients demand that they be given free drugs if approved? At least the more organized patient groups can attempt to push the sponsors to do the right thing. As far as making FDA accept the Declaration of Helsinki, that is a high barrier due to strong resistance from the pharmaceutical industry.