White Paper

Right-To-Try Or Right-to-Ask? Understanding Right-To-Try And FDA's Expanded Access Programs

Source: Rho

By Dr. David Shoemaker, Ph.D., Dr. Kevin Barber, Ph.D., Dr. Karl Whitney, Ph.D.

Patient Engagement

The Right-to-Try concept originated from the Abigail Alliance vs. von Eschenbach legal case which involved the proposed treatment a head and neck cancer patient, Abigail Burroughs, with an investigational drug in development for colorectal cancer. Abigail’s father, Frank, sued the FDA for access to Erbitux (cetuximab) to treat Abigail. The suit stated that access to investigational products by terminally ill patients after phase 1 is a constitutional right. Initially, the DC Court of Appeals ruled in favor of the Abigail Alliance. FDA requested that the Court of Appeals rehear the case, and the case was overturned at that point. Frank Burroughs appealed to the US Supreme Court which refused to hear the case, effectively leaving FDA regulations intact.

The case was based on the assumption that products that clear phase 1 are both safe and promising. Many consider this to be a fallacious assumption. Only 10-20% of drugs completing phase 1 ever reach the market either because there is no demonstrated benefit or there is an unacceptable safety risk. FDA approval is based on an analysis of the benefit to risk ratio to all subjects assessed in phases 1-3. Even then, some products are pulled from the market after approval because of safety issues that are only identified in larger patient populations. Animal safety models are rarely fully predictive of how a drug will behave in humans.

Despite these erroneous assumptions, the intent of Right-to-Try is a good one—alleviating the administrative burden for seriously ill patients to gain access to investigational products. Another important component of Right-to-Try is reducing the risk for sponsor companies in providing patients with access. Many sponsors are concerned both about their liability resulting from a negative clinical outcome and the impact of a negative clinical outcome on continued development of the product.

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