The “Right to Try” movement may be embedded in the Republican Party platform.
Amid the run up to the Republican National Convention that begins in Cleveland next week, the party has adopted an amendment in its draft platform that endorses a controversial stance toward obtaining treatments for fatal illnesses. Known as Right to Try, the concept is designed to allow desperately sick people to gain access to experimental medicines.
The move embraces so-called Right to Try laws that have been passed by 31 states and allow terminally ill patients to leapfrog the drug development process, which typically takes years before new treatments become available. The platform amendment was introduced on Monday by Eric Brakey, a convention delegate and Maine state senator who last year shepherded such a bill into law in his own state.
“We commend those states that have passed Right to Try legislation, allowing terminally ill patients the right to try investigational medicines not yet approved by the FDA. We urge Congress to pass similar legislation, giving all Americans with terminal illnesses the right to try,” the amendment states, according to Brakey. A final version of the party platform is expected to be completed later Tuesday, he told us.
The laws reflect rising frustration with a Food and Drug Administration program called expanded access, in which people who are seriously ill can obtain a drug under development, even though they are not enrolled in a clinical trial. Under federal law, if terminally ill patients are not eligible to obtain an experimental medicine through a clinical trial, they can apply to the FDA for what is known as compassionate use.
The agency actually approves the vast majority of these requests, which must also be approved by drug makers. But critics say the program is arbitrary and cumbersome. Two months ago, in fact, a bill was introduced in the US Senate as a companion to legislation introduced in the House last year. Neither has progressed, but these are seen by supporters as a key stepping stone toward removing the FDA from the process.
To what extent the state laws have accomplished their goal is unclear. A spokeswoman for the Goldwater Institute, a libertarian think tank that helped draft the laws, claims that 60 patients have received medicines, but she could not provide the names of the physicians or companies involved. Critics, meanwhile, say there are a host of problems with the laws that make the concept untenable.
For one thing, the frustration directed at the FDA is misplaced, they say. That’s because drug makers are the final gatekeepers and may deny requests in order to meet strict criteria needed to win FDA approval for a medicine. An unexpected patient reaction, for example, might jeopardize the chance that a clinical trial will succeed. Sometimes, a company may lack sufficient supplies of their drug.
Even so, the FDA rarely penalizes a drug maker if its product causes an adverse event prior to approval, according to a report by a working group at the New York University Langone Medical Center. One member is Ken Moch of Salutramed Group and former chief executive at Chimerix, which caused an outcry in 2014 when the company initially denied an experimental drug to a young boy who was dying of cancer.
The FDA “recognizes the inherent risk in providing investigational products to a very sick population, and it believes that the few times such action was warranted — 0.2 percent of cases over the last four decades — were due to severe, avoidable harms stemming from administration of a drug,” the working group wrote in a recently released paper that examined Right to Try laws.
One thing is clear — the number of requests submitted to the FDA has increased over the last several years. In 2014, requests rose by 92 percent, the working group found. At the same time, the number of social media campaigns has also increased — although data was not specified — and often these are directed at companies.
There are also some unknowns. Some state laws may put patient hospice coverage, home healthcare coverage, or insurance benefits at risk, according to the working group. In fact, by trying an experimental treatment while in hospice, coverage could be forfeited, the group reported.
In any event, the working group maintained that Right to Try laws do not obligate a company to provide an experimental drug to any patients asserting their right to try the medicine. And the group argued there is no evidence, to date, that anyone in a state with such a law has obtained any medicine that would have been unattainable under existing federal regulations.