Right-To-Try Does Not Change Drug Company-Patient Interactions for Test Drugs
​​​[Posted on: Thursday, May 24, 2018]

Right-To-Try is all set to become the law and the only big change is the shift in the role of FDA from an approver to observer for compassionate use of investigational products. The impact on patient access is limited at best and it does help the drug companies who own such investigational products but eliminating the regulatory risk. The law does not affect how drug companies interact with patients and doctors requesting investigational products on a compassionate basis. Drug companies can still refuse to provide the drug and will have no liabilities if there are any new adverse events related to the use of their unapproved drug. FDA is specifically forbidden from using any information associated with the use of an eligible investigational drug “to delay or adversely affect the review or approval of such drug”.  This will be a cause of celebration for those looking to reduce FDA’s role as FDA has been cast as the villain in this debate, for all the wrong reasons. FDA has rarely objected to compassionate use of investigational products in patients will life-threatening diseases who have exhausted all available therapies. FDA recently even made it easier to apply for the compassionate program and promised response within days, but it did not sway the political discussion. Under the new law, the drug still needs to be in clinical trials and have completed at least Phase 1 safety clinical trials. The biggest limitation to the compassionate use program was companies unwilling to spare precious stocks of investigational products, which is unaddressed. FDA is now required to publish a list of compassionate use programs listing all drugs that were used for treatment of patients under Right-To-Try. This means that companies that provide investigational drugs to one patient will likely be the target of future similar request and if they take different decisions, may be accused of discrimination. The law tries to eliminate a common excuse by companies about the negative regulatory outcomes of the uncontrolled compassionate use. But from a practical point of view, if a drug is used on a compassionate basis and found to have serious negative consequences, it would be hard for a company to escape some penalty even if it is not direct. The FDA reviewers, for example, could still use the information to subject the drug to more vigorous review without mentioning the negative news from compassionate use. And negative news still travels. The unintentional consequence could be that even if there are no regulatory penalty, the news of an investigational drug causing harm to any patient will adversely affect the market of a given drug. On the contrary, should the drug show exceptional benefit, it could help the drug immensely. It could allow the manufacturer to seek expedited approval from FDA or even incentives such as priority review, fast-track designation, reduced clinical trials, even Breakthrough Therapy Designation. By not changing much, the law is not bad for the industry and may give some hope to patients. And hope, as they say, is the biggest medicine.