Patient groups criticize ‘right to try’ bill on experimental drugs

More than 75 patient organizations sent a letter to House leadership Monday voicing their opposition to a revised “right to try” bill on experimental drugs that is headed to the House floor for a vote on Tuesday evening.

Over the weekend, Republican leaders on the House Energy and Commerce Committee unveiled a revised version of a right to try bill, which would allow terminally ill patients to request access to drugs the Food and Drug Administration (FDA) hasn’t yet approved — and to do so without going through the FDA.

Patient advocacy groups expressed concerns with taking the FDA out of the process.

“While this version of the legislation includes patient safety improvements compared to previous versions of the legislation, we reiterate our concern with creating a secondary pathway for accessing investigational therapies outside of clinical trials that would remove Food and Drug Administration (FDA) approval and consultation, and would not increase access to promising therapies for our patients because it does not address the primary barriers to such access,” the groups wrote in the letter to Speaker Paul Ryan (R-Wis.) and House Minority Leader Nancy Pelosi (D-Calif.).

The groups on the letter include the American Cancer Society Cancer Action Network, Susan G. Komen and the National Organization for Rare Disorders.

President Trump called on Congress to pass right to try legislation in his State of the Union address in late January. The Senate passed the bill by unanimous consent over the summer, so Trump’s call put pressure on the House to act.

Energy and Commerce Chairman Greg Walden (R-Ore.) had been clear that changes to the legislation were coming, telling reporters in early February that he wanted to take into account the concerns of advocacy groups and work to make sure the measures were safe and worked.

On Sunday, Walden and the panel’s Health Subcommittee chairman, Rep. Michael Burgess (R-Texas), said in a statement that the bill “has been a long-time coming, but in striking the right balance for patients and their safety, the House is on track to deliver hopeful news for patients desperately seeking the right to try investigational treatments and therapies.”

The patient groups still aren’t satisfied. They acknowledged changes to the bill that they view as improvements, such as beefing up informed consent requirements, narrowing those who are eligible to participate, adding additional reporting to the FDA and more.  

But the groups also noted the FDA already has a compassionate use program that lets a physician ask the FDA to let a patient try an experimental drug; the agency approves more than 99 percent of those requests. They also noted that drugmakers often decide not to let a patient try a drug outside of a clinical trial.

The top Democrat on the Energy and Commerce Committee, Rep. Frank Pallone Jr. (N.J.), announced his opposition to the bill Monday. A Democratic aide expressed frustration that the House was voting for the bill under suspension of the rules, which is typically reserved for non-controversial bills. Support from two-thirds of the House will be required for passage. 

Pelosi will also oppose the bill, according to her office.

“Leader Pelosi will follow the lead of numerous patient and disease groups in opposing this legislation,” spokesman Drew Hammill wrote in an email.

Supporters of the legislation include Vice President Pence and groups backed by conservative mega-donors Charles and David Koch.

Proponents argue that those who are terminally ill should have every tool at their disposal to try a drug that could possibly help. They note that the medicines must have passed a phase 1 clinical trial and be in the FDA’s pipeline. They also say the drug approval process takes too long.