Compassionate Use Rules are Better Than the “Right to Try” Laws

Recently “Right to Try” bills were introduced in the US Senate and Congress. Similar laws have been enacted in 33 states; however it was felt that creating a Federal law will have a stronger impact in getting companies to give experiment drugs to terminally ill patients. While the intent behind the bills is great, the execution is misplaced. A careful review of FDA’s compassionate use programs, also known as the Expanded Access programs, show that the existing programs are far more productive in helping needy patients than the proposed Right to Try law or similar laws in effect in 33 states. The main purpose of the Right to Try law is to take FDA out of the compassionate use program. So long as the physician believes an experimental drug could benefit his terminally ill patient, the drug could be used without any liability to the physician, the manufacturer, and any other parties involved in the decision to provide the said drug. Also, FDA will not be allowed to use any negative results of the experimental use during the review of the subsequent market approval application for that drug. Basically, the doctors and the manufacturers will not have to disclose any serious adverse events possibly related to the drug use to FDA. By removing the need for the compassionate use IND and FDA approval, the Right to Try law, in theory, expedites patient access to experimental therapies. But that’s where the proposed laws make a mistake. The law is based on the assumptions that FDA is someone responsible for terminally ill patients not getting life-saving experimental drugs. That’s far from truth. The reason why terminally ill patients do not get an experimental drug is almost always not because of FDA but because the manufacturers deny the request. In fact, FDA gets anywhere from 1200-2000 requests every year for compassionate use and grants practically all of them. The new law does not require a company to provide its experimental drug to a patient. Also, there is no incentive to the manufactures to comply with the request. On the other hand, current compassionate use program provides several ways companies can provide drugs to needy patients while at the same time collecting important use data supportive of the eventual market approval application. Also, by involving FDA in the decision, the process assures that FDA can review the protocol and provide important feedback for proper use. In about a third of the cases FDA suggests important changes to the protocol to the benefit of the patient. Compare that to about 40 documented uses of experimental drugs in the 33 states with Right to Try over the same period. In many ways, the Right to Try has the opposite effect than intended. Although it absolves the company of any liability, it also makes it hard for the company to use any good information from use of its drugs. Since companies are not required to report adverse effects, were they to chose presenting the positive information to FDA, they would always be suspected by the FDA reviewers of cherry picking data. Companies are not allowed to charge for the experimental drug without FDA approval under Right to Try, just like the compassionate use programs. There exist multiple mechanisms to provide experimental drugs quickly to needy patients and to expedite approval of good drugs at the same time. Right to Try looks destined to end as a feel-good law with no practical benefit to patients.