The Terminally Ill Deserve Right-to-Try Laws
Originally published by Ron Johnson for The Wall Street Journal
The Terminally Ill Deserve Right-to-Try Laws | The Wall Street Journal
In 2014 I met a young mother of three from Waukesha, Wis. Her name was Trickett Wendler, and she had the degenerative nerve disease amyotrophic lateral sclerosis (ALS). Before her diagnosis, Trickett was living a normal life and looking forward to a bright future. ALS robbed her of her dreams and took away her body’s ability to function. She died on March 18, 2015.
Like their mother and grandfather, Trickett’s children are genetically predisposed to ALS. Trickett was prescribed the same medications her father was given in the 1990s. Her husband Tim prays that better treatments will be developed if, God forbid, one of his children must fight this horrible disease.
The Food and Drug Administration will play a key role. Bills now pending in Congress are designed to make sure the agency plays a positive, life-sustaining role by not standing in the way of terminally ill patients who are asking for a chance to try new treatments when there are no other options left.
Potential treatments for many terminal diseases are in the clinical pipeline. Some that may hold promise for ALS patients have already passed initial Phase I safety trials.
But safety is not the FDA’s only mandate. The agency also must establish a drug’s effectiveness before potential breakthroughs are allowed on the market. This risk-averse approach results in an approval process that on average takes 14 years, according to a 2014 study from Tufts University, and costs nearly $2.6 billion for a single drug.
Consider 7-year-old Jordan McLinn who has Duchenne, a 100% fatal form of muscular dystrophy. On Monday, Sept. 19, the FDA finally granted approval for a promising treatment. But this was only after forcing Jordan and boys like him to wait through years of development, clinical trials and FDA applications.
The FDA spent 14 months completing its final review of the treatment, missing multiple deadlines during that time for a drug, called eteplirsen, that qualified for so-called accelerated approval. In the interim, patients like Jordan continued to suffer debilitating muscle loss.
Why would anyone delay a safe treatment to patients like Jordan when death is the certain alternative?
A growing national movement believes terminally ill patients deserve the right to try safe but experimental drugs and treatments. Thirty-one states now have enacted laws to give terminally ill patients the legal right to access drugs and treatments that have demonstrated safety but not yet received FDA approval. At a time when national politics are often divided, right-to-try laws are passing with nearly unanimous support.
But the Obama administration’s posture on these laws is unclear. Doctors and pharmaceutical companies rightly fear professional and legal consequences if they administer experimental treatments. In my Senate committee, I’ve attempted to get the FDA to speak clearly on whether it will respect these state laws.
We heard from Dr. Ebrahim Delpassand, a Texas oncologist who is bravely treating 78 cancer patients under his state’s law—patients he is almost certain would be dead without the treatment. We also spoke with an experienced clinical researcher and a biopharmaceutical company CEO who told us that right to try would actually enhance knowledge and innovation. But these medical professionals worry that going against the FDA, even to the benefit of their patients, could jeopardize their ability to get favorable consideration from the agency in the future. It could even jeopardize their careers.
On May 10, I introduced the Trickett Wendler Right to Try Act of 2016 in Congress. It requires the federal government to abide by the right-to-try laws in the now-31 states that have adopted them. This bipartisan bill now has 42 senators signed on, and there is similar legislation in the House of Representatives. Next week I intend to go to the Senate floor to ask my colleagues to join me in supporting this effort and allow this bill to pass by unanimous consent.
No one can guarantee a miracle cure. What we can and must do is give patients and families the freedom to decide for themselves how to fight their illness. With no other options, they at least have the right to hope.
Terminal patients don’t have a decade to wait for the FDA to judge whether a drug is effective—the cost of delay might be measured in the unnecessary loss of human lives. Right-to-try laws simply establish the freedom for patients and their doctors to try safe therapies where the potential benefits far outweigh the risks terminal patients are eager to take. Families like the Wendlers, the McLinns and the Bellinas deserve hope. They deserve the right to try.
Mr. Johnson, a Republican from Wisconsin, is chairman of the Senate Homeland Security and Governmental Affairs Committee.