As the White House continues its push for the benefits of "right to try" (RTT), the first CRO associated with the law has come onto the stage.
The RTT law, passed last year and now live under 41 states, allows patients with life-threatening and terminal conditions to get access to experimental meds without FDA approval. It's similar to the "compassionate use" exemption, but the treatments must have passed phase 1 tests.
It’s been something of a focus for President Donald Trump, who has recently lauded its use in helping patients, and a number of biopharmas have been getting in on the act to boost use of their drugs before going up at the FDA.
Now, the contract research industry is getting involved, as the first CRO dedicated to RTT launches. The so-called Beacon of Hope CRO officially came into life this month and is “the first contract research organization exclusively dedicated to providing the infrastructure for patients with life-threatening diseases seeking access to eligible investigational treatments, and the companies that are developing them,” under RTT, it said in a statement.
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The CRO said it “provides the infrastructure to bring together drug-developers, patients and physicians, seeking to make use of the Right to Try law, in a turn-key, scalable solution.”
It’s being headed up by founder Richard Garr, a biotech CEO for 17 years and formerly chief at stem cell company Neuralstem until 2016 when he stepped down. In the same year, the biotech said it was slashing jobs across all divisions after posting murky data from its experimental amyotrophic lateral sclerosis (ALS) therapy.
“As both a 17-year veteran of the biopharmaceutical industry and the father of a child who has benefited twice from investigational treatments outside of the traditional clinical trial environment, I have seen how the industry has failed such patients from both sides,” said Garr.
“In addition to creating Right to Try treatment programs for therapeutics developers, Beacon of Hope CRO is also working with patients, and supporting advocacy groups, as our presence at the ALS Hope and Help Symposium attests to.
“We seek to educate the patient community on the importance of Right to Try treatment programs and awareness of our 'opt in' Patient Database, which is how our client companies, conducting clinical trials in ALS, or other life-threatening conditions, will find them. We hope to be able to change the way very sick patients gain access to treatments, and the way therapy developers reach those patients in need.”
Under RTT rules, eligible drug developers don’t have to report patient trial data to the FDA and are also protected from future prosecution, which the CRO says means RTT data “could become a valuable source for drug developers.”
And for the patient, they only need a physician and the drugmaker to sign off on the request, although the FDA has to be informed.
Back in January, a cancer vaccine being developed by Epitopoietic Research Corp. (ERC) became the first drug to be given to a U.S. patient under RTT.
ERC said it had informed the FDA last June that it intended to make the vaccine—called Gliovac (ERC-1671)—available to a California patient who hadn’t met the eligibility criteria for enrollment in the company’s ongoing phase 2 U.S. study in brain cancer.
In 2018, Israeli biotech BrainStorm backed away from providing an experimental treatment to a patient with neurodegenerative disease ALS under RTT. In that case, the decision stemmed not from ethical, legal or practical consideration but because the company decided it wouldn’t be possible to fund treatment for all patients who wanted to try the drug.