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By Gail H. Javitt, JD, MPH
On May 22, the House of Representatives passed S. 204, the Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina Right to Try Act of 2017 (generally known as the “Right-to-Try” bill). The Senate previously passed the bill in August 2017. President Donald Trump, who had strongly advocated for the passage of the legislation, will soon sign it into law.
The Right-to-Try bill, which amends the Federal Food, Drug, and Cosmetic (FD&C) Act, removes from FDA the authority to oversee access by terminally ill patients to early stage investigational therapies. Under the new law, FDA will no longer have authority over the administration of “eligible” investigational therapies to “eligible” patients.
To be an eligible patient, an individual must:
The certifying physician must be in good standing with his or her professional licensing board and must not be compensated for providing certification by the manufacturer of the investigational drug.
To be an eligible drug, the investigational product must:
The bill also sharply constrains FDA’s ability to use information about an eligible patient’s experience with an eligible investigational drug to delay or adversely affect the agency’s review of the sponsor’s NDA. Absent a request from the sponsor to use such outcome data, a senior FDA official (Center Director or above) must make a determination that use of the eligible patient’s clinical outcome information is “critical” to determining the drug’s safety and must provide written notice that includes a public health justification for the determination to the sponsor.
The bill requires manufacturers who agree to provide investigational therapy to eligible patients to report annually to FDA the number of doses supplied, the number of patients treated, the uses for which the drug was made available, and any known serious adverse events. This information will be posted on FDA’s website.
Finally, the bill also includes provisions aimed at shielding manufacturers and physicians from liability arising from their activities related to providing the drug and, conversely, from their decision not to grant a patient’s request to obtain access to an investigational product.
Although Right-to-Try advocates cast themselves as champions of patient autonomy and self-determination in the face of an overweening federal bureaucracy, unfortunately this narrative is not in accord with the facts. Under current law, a patient who is not enrolled in a clinical trial and for whom the investigational therapy manufacturer agrees to provide access must obtain FDA authorization through the agency’s “expanded access” (also known as compassionate use) program. In practice, FDA approves expanded access requests in almost all cases, typically in a matter of days. The real rate-limiting step in expanded access is the ability and willingness of pharmaceutical companies to provide investigational therapies to patients outside the context of a clinical trial. There are a number of valid reasons that may drive such decisions. In particular, the supply of investigational product is often limited—and in the case of biologically-derived therapeutics can be challenging to manufacture. To successfully and expeditiously complete the rigorous clinical trials required for approval, manufacturers necessarily must prioritize access to current and future clinical trial participants. Furthermore, early stage companies may not have the bandwidth effectively to administer a compassionate use program. The new reporting obligations imposed by S. 204 add to this burden. Finally, manufacturers may justifiably fear accusations of bias and inequity if they grant some patients’ compassionate use requests and not others. While S. 204 provides some liability protection for sponsors and manufacturers, the law does not prevent allegations predicated on “reckless or willful misconduct, gross negligence, or an intentional tort.”
Perhaps the most pernicious aspect of S. 204 is that it perpetuates a false narrative that early-stage investigational drugs (specifically, those that have completed Phase I studies) have a high likelihood of benefiting terminally ill patients. Most drugs that complete Phase I do not make it through FDA approval. Later trials may fail to show efficacy or may reveal risks that outweigh any potential therapeutic benefits. Furthermore, advocates of Right-to-Try perpetuate the view that there are no risks too great for someone who has “nothing left to lose.” But pressure of terminally ill patients to “try everything” in the face of overwhelmingly small odds and potential additional suffering can serve as its own form of coercion that undermines the very autonomy that Right-to-Try advocates claim to promote.
The reality is that Right to Try is unlikely meaningfully to increase the number of terminally ill patients who can access investigational therapies or improve the outcomes for such patients. Those that claim otherwise, and who cast FDA as the primary obstacle between dying patients and lifesaving medicines, are peddling their own form of “snake-oil” — in the form of false hope and misdirected blame — to the most vulnerable patients and their desperate families. They also distract focus from deeper, but more difficult questions, about how best to facilitate and accelerate research and innovation to advance the current state of scientific knowledge regarding the causes and mechanisms of disease and thereby to develop new preventive and therapeutic approaches.
Gail H. Javitt is a Member of the Firm with Epstein, Becker & Green P.C. in Washington.
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