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The Right-To-Try Bill Puts Patients At Risk In The Name Of Helping Them

Congress recently approved the federal right-to-try bill after a months-long standoff in the House, and President Donald Trump signed it into law on Wednesday. The measure allows people with life-threatening diseases to bypass the Food and Drug Administration to obtain experimental drugs.

The right-to-try movement is nothing if not well-intentioned, inspired by high-profile patient cases ― mostly desperately ill children ― whose stories struck a nerve with the nation. Much of the public, enamored by the undeniable surface-level appeal of right-to-try legislation, embraced this movement. But those with a deeper understanding of the drug development process ― medical professionals, ethicists and regulators ― have been publicly skeptical.

The truth is this legislation will do almost nothing to help terminally ill patients. That’s because the entire right-to-try approach is based on a myth: that the FDA is the rate-limiting middleman preventing patients from receiving potentially life-saving drugs.

Over the last four years, right-to-try legislation has gained momentum and is already the law in 40 states. The laws grant terminally ill patients who have exhausted other treatment options the right to request access to potentially life-saving drugs that have cleared Phase I clinical trials without approval or oversight by the FDA.  

The real gatekeepers to expedited access are pharmaceutical companies.

Josh Hardy was one of the very first right-to-try patients. The 7-year-old boy received an experimental drug in 2014 that ultimately cured him of a life-threatening viral infection. The experimental antiviral treatment was in the middle of its manufacturer’s Phase 3 clinical trial at the time.

The company’s board repeatedly denied requests by Josh’s doctors and parents to use the drug on the grounds that it was in the best interest of the greatest number of patients to devote the company’s finite resources to the clinical trial ― which, if successful, would lead to FDA approval and make the drug available to thousands of more patients in need. But the company’s then-chairman, under the pressure of a fierce social media campaign, made a landmark decision to diverge from the company’s board and ultimately sent the drug to Josh in the name of compassionate use.

Josh’s story inspired a wave of well-meaning right-to-try legislation advocates who believe such laws will ensure terminally ill patients with even the slimmest of hopes of benefiting from experimental therapies have access to these drugs. 

But the FDA has already consistently demonstrated its commitment to the compassionate use of experimental therapies. Three decades ago, it launched the expanded access program that allows for compassionate use of drugs for patients with life-threatening illnesses through a case-by-case approval process. In 2015, the FDA streamlined the previously cumbersome application process, and today the form takes only about 45 minutes to complete. The agency responds in an average of four days with a 99 percent approval rate.  In emergency situations, it responds to requests within 24 hours.  

The entire right-to-try approach is based on a myth.

The reality is, the real gatekeepers to expedited access ― as Josh Hardy’s family encountered ― are pharmaceutical companies. And this new right-to-try bill does nothing to compel manufacturers to grant patients access to experimental drugs. 

In a meager attempt to nudge companies to authorize more compassionate use cases, the federal right-to-try bill offers companies immunity from legal recourse should drug-related adverse events occur. It also prohibits the FDA from considering these events during its approval process.

But drug manufacturers face other concerns, like spooking investors or inciting a PR catastrophe should unexpected adverse events occur. They may also worry that the legwork required to grant access to individual patients could slow efforts to get the drug approved for all patients in need ― an argument that holds ethical merit. Often, companies don’t have enough extra product to give away, especially while clinical trials are underway.

Put simply, right-to-try laws, though applauded by many, don’t actually give patients the “right” to try experimental therapies. They simply support a right patients already have: the right, as Josh Hardy’s parents exercised, to plead. Indeed, since Colorado in 2014 became the first state with a right-to-try law, researchers have yet to identify a single patient who has received access to an experimental drug due to a right-to-try law that would not have been attainable through the FDA’s expanded access program.

But right-to-try legislation isn’t just superfluous ― it may also create a new type of inequality among the terminally ill. To rally drug companies to provide experimental therapies, patients and their families often need to be well-connected, relatively affluent or adept at navigating social media to put drug companies under pressure. Those without the resources or know-how may never receive access to experimental therapies.

This legislation will do almost nothing to help terminally ill patients.

Plus, in discarding FDA involvement in the compassionate use process, the right-to-try approach may also be dangerous. In a recent review of expanded access proposals reviewed by the FDA, the agency recommended changes to dosing, safety monitoring or informed consent in 11 percent of cases. Bypassing this step, which involves expert review of information from early-phase clinical trials often only available to the FDA, robs patients of safety protections afforded by the agency’s guidance.   

Instead of using federal right-to-try legislation to sidestep the FDA, the medical community should focus on facilitating access to experimental drugs through the existing channel: the expanded access program. We must explore new ways to make compassionate use more feasible ― and appealing ― to drug companies and provide better education to patients and physicians when navigating the application process. And drug companies should take an active role in encouraging patients interested in experimental treatment to continue to seek FDA involvement, a precedent set publicly by Johnson & Johnson.

At a time when innovation in medical research is offering new hope to patients suffering from terminal conditions ranging from cancer to infectious diseases, there is an urgent need for fair, consistent processes to support compassionate use and guide the allocation of experimental therapies. Unfortunately, the federal right-to-try bill contributes little ― if anything ― toward this objective.

Dr. Nicole Van Groningen is an internal medicine physician at Cedars-Sinai Medical Center in Los Angeles. She writes frequently about health policy, medical ethics and general health issues, with a special focus on women’s health. She tweets at @NVanGroningenMD.


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