Cancer Advocate Fears ‘Right to Try’ Law
Richard Plotkin, who used experience with his grandson’s cancer diagnosis to become one of the nation’s leading advocates for children with cancer, says that the “right-to-try” legislation being considered by Congress should be rejected because it would leave patients without FDA protection. Writing in a blog for The Hill, Plotkin says the proposed legislation is intended to help desperate patients who are unable to join clinical trials and for whom approved medicines offer no help. “But these patients already have a route by which to get access to experimental treatments outside of clinical trials: FDA’s expanded access program,” he declares.
The post notes that FDA approved 1,256 of 1,262 expanded access requests received from 10/1/14 through 9/30/15. “These were not ‘rubber stamp’ approvals,” Plotkin writes. “A recent study of 150 randomly-selected cases found that 11% of the cases that received FDA approval had alterations in the treatment plan due to input from FDA, input that improved the safety and efficacy of the procedures.” He says that such feedback would not be available under the proposed legislation.
He says FDA involvement is particularly important in pediatric cases because drugs that have progressed through Phase 1 and 2 trials in adults may not have the same results when given to children. “Review by FDA gives those seeking compassionate use waivers a degree of ‘cover’ from lawsuits that may later be filed against them,” he writes. “Incredibly, the right-to-try bills create a pathway by which experimental drugs can be issued to patients with terminal illnesses without any oversight by FDA. The bills specifically state that the federal government shall not take any action to prohibit or restrict the use of such drugs.”
As a retired attorney, Plotkin notes that under the proposed federal law certain parties are not liable. But doctors, hospitals, institutional review boards, parents of minor children, the Goldwater Institute (which is promoting the legislation), and perhaps others not covered in the bills are not in the protected group and thus could be sued in state and federal courts.
His final point is that the legislation provides that negative outcomes of the use of experimental medical products under the legislation cannot be used by a federal agency such as FDA in deciding whether to approve the product for public use. “This is absurd,” Plotkin says. “What this means is that if FDA is bypassed and an experimental medical product is given to a patient who suffers severe adverse effects as a result, the doctor and the drug company cannot provide this information to FDA and, if FDA learns of the situation, it must remain silent and take no action.”