Right to try, access, and safety.

Mar 14, 2018 | Advocacy & Policy

Earlier this week, TargetCancer Foundation joined with the National Organization of Rare Disorders (NORD) and 81 other rare advocacy organizations to express concerns with federal right-to-try legislation. The bill was considered by the U.S. House of Representatives yesterday but failed to receive the votes necessary to pass. Congressional leadership is expected to try again to pass the House bill or a similar bill which passed the Senate last year.

The safety and efficacy of investigational medicines are evaluated in clinical trials before the Food and Drug Administration (FDA) can determine whether the benefits of the medicine outweigh the risks, in which case the medicine is approved for use by patients. Right-to-try legislation eases the process under which patients can gain access to a medicine that is still in development, but reduces FDA oversight.

Our goal at TargetCancer Foundation is to support the development of lifesaving treatment protocols for rare cancers, and we understand the desire of patients who may not qualify for clinical trials to gain access to an investigational medicine outside of a trial. However, the legislation considered by the House yesterday does not fully address concerns with patient safety and would minimize the important role of FDA through its expanded access program.

As stated in the letter, “FDA’s expanded access program, though imperfect, facilitates access to investigational therapies for over a thousand patients facing serious and life-threatening conditions each year. FDA repeatedly approves over 99 percent of requests while sometimes making important dosing and safety improvements to proposed expanded use.”

The current FDA Commissioner, Scott Gottlieb, has also expressed concerns about minimizing the Agency’s role in evaluating requests from doctors for access to investigational medicines. In a Politico article published yesterday, he “emphasized that the vast majority of drugs provided to patients through its compassionate use program turn out to be ineffective. ‘We know that 70 percent of all drugs that are offered under an expanded access setting are never approved by FDA”’.

We remain committed to making our voice heard with FDA, Congress and other stakeholders to ensure that patients with rare cancer have access to medicines at the earliest opportunity that they can do so safely.