More assertions by President Donald Trump about the Right to Try Act, a lack of regulations and the emergence of startup companies that aim to profit from the 2018 law have sparked concerns from bioethicists and patient advocates more than a year after the law was enacted.
Trump has been promoting the law as saving patients' lives — a claim not backed up by evidence, which the White House has repeatedly declined to provide.
U.S. FDA acting Commissioner Ned Sharpless, First Lady Melania Trump, President Donald Trump and HHS Secretary Alex Azar.
"A lot of people are being saved," Trump said on Sept. 11 at the White House Oval Office, with Health and Human Services Secretary Alex Azar — the nation's top healthcare official — and Acting U.S. Food and Drug Administration Commissioner Ned Sharpless seated nearby.
HHS declined to comment on Trump's remarks and Azar and Sharpless have not publicly made any rebuttal.
The Right to Try Act was adopted by Congress in May 2018. It permits critically ill U.S. patients to seek experimental treatments without going through the FDA's expanded-access program, also known as compassionate use, which has been in place for decades. The FDA said it grants about 99% of the compassionate-use requests it receives.
Trump and some lawmakers have insisted the FDA was not approving compassionate-use requests quickly enough and pushed to get the Right to Try law adopted as an additional option for patients.
Critics have said the real intent of the Right to Try Act was to weaken the FDA and have raised concerns that desperately ill patients could be exposed to "snake oil" salesmen peddling false hopes.
Under Right to Try, patients must have a life-threatening disease or condition, be unable to participate in a clinical trial and have no other comparable medical options. Physicians can request access to experimental treatments from drugmakers after a phase 1 safety study is completed and during or after a phase 2 or 3 efficacy trial.
Only two U.S. patients are known to have accessed experimental therapies under the federal Right to Try Act, though the status of those people's progress is unknown, according to bioethicists tracking the issue. The therapies tried were Brainstorm Cell Therapeutics Inc.'s NurOwn and Epitopoietic Research Corp.'s Gliovac.
An FDA spokeswoman said the agency does not currently have information on the number of patients who have accessed unapproved drugs under Right to Try to back up Trump's assertions.
The law requires drugmakers to provide an annual summary to the FDA on the use of Right to Try therapies and the agency is mandated to publicly post a consolidated summary of those reports each year. The FDA, however, has yet to propose its regulations around those requirements — rules that may take months or possibly years to finalize.
At recent political rallies, Trump has repeatedly said a patient who used an FDA-approved cancer immunotherapy for an unapproved use — a common and legal practice employed by doctors for decades and known as off-label use — did so under the Right to Try Act. Bioethicists Jeremy Snyder of Simon Fraser University, Alison Bateman-House of New York University and Patti Zettler of The Ohio State University have said the case is not one in which the 2018 law would need to be applied.
At the Republicans' Sept. 12 retreat in Baltimore, Trump said the Right to Try law had been successful at keeping terminally ill Americans from traveling overseas to access experimental treatments. He did not provide numbers or specific evidence to support that claim.
Drugmakers cannot be forced to provide their experimental treatments under the Right to Try Act and have no obligation to do so.
Last week, the Government Accountability Office reported that 13 of the 19 manufacturers they found that were willing to consider Right to Try access of their drugs said they still would require regulatory authorities to review those requests, including six that specified the FDA, just like they would under the compassionate-use program — the process the 2018 law sought to go around.
While pharmaceutical manufacturers are largely taking a cautious approach, the bioethicists pointed to two new non-traditional contract research organization startups that have emerged and are seeking to monetize the Right to Try law — both named Beacon of Hope CRO.
Beacon of Hope CRO CEO Richard Garr
The leaders of both companies told S&P Global Market Intelligence their organizations are unrelated, despite both being located in the same county in Florida, even displaying similar logos on their websites.
Richard Garr's Beacon of Hope CRO is located in Boca Raton, Fla., while the other company, run by Nancy Torres Kaufman, is an entity of Beacon Pharmaceutical. Torres Kaufman's company is relocating from New York to Jupiter, Fla., to establish a life sciences accelerator facility under an agreement with the city and the Palm Beach County Business Development Board.
Exploring the backgrounds of the two near-identical companies was like going down a rabbit hole, said Simon Fraser University's Snyder. He discovered a number of online pages linking Garr and Torres Kaufman to each other, so it remains unclear why the two company chiefs — both longtime Right to Try supporters — would give their organizations the same name, particularly if they are competing against each other.
Snyder also discovered that Beacon Pharmaceutical's venture and philanthropy arm, Beacon Capital, had designed its own blockchain technology cryptocurrency — the Beacoin — which the company said in an Aug. 11, 2018, Medium post could be used to provide access to therapeutics and treatments.
Garr is a longtime biotech industry executive and the former CEO of Neuralstem Inc.
He told S&P Global Market Intelligence his newly formed Beacon of Hope CRO has a particular client that has committed to treating between 200 and 300 patients over 12 months with its investigational drug for amyotrophic lateral sclerosis, also known as Lou Gehrig's disease.
One patient with ALS who spoke to S&P Global Market Intelligence and did not want to be identified had looked into Garr's company and was told by the CEO that his client was running an autologous bone marrow-derived stem cell harvest and transplant program.
The patient was directed to the U.S. government's clinicaltrials.gov website to the Neurologic Stem Cell Treatment Study, or NEST, program being run by MD Stem Cells.
According to MD Stem Cells' website, patients would be responsible to pay $17,000.
ALS patient advocate Cathy Collet raised concerns that the program was pay-to-play. She also called the NEST inclusion criteria and endpoints "loosey-goosey."
"I'm not sure there is anything nefarious about all of this," Snyder said. "It is just strange and opaque. When we're dealing with clinical trials, expanded access and real people with real illnesses who are desperately hoping for better health, however, transparency is needed."