June 20, 2018, 12:44 PM
CDT Updated on June 20, 2018, 4:59 PM CDT
A small
biotechnology company may be the first to offer dying patients unproven drugs
under a new U.S. law called Right to Try that deregulated access to such
experimental treatments.
But it
won’t be for free: Brainstorm Cell Therapeutics Inc. would charge for a
therapy it is developing for the deadly condition known as Lou Gehrig’s disease. While details are still being worked
out, the company’s chief executive officer pointed to the price of bespoke cell
therapies used to treat cancer that cost more than $300,000.
“Companies
cannot be NGOs,” the nongovernmental organizations that help provide care to
impoverished countries, Brainstorm CEO Chaim Lebovits said in a phone
interview. “We have to have an incentive.”
If it
decides to proceed, Brainstorm -- a company with no drug on the market yet and
no revenue -- would introduce a profit motive into an effort many expected to
be altruistic, adding more controversy to an already contentious debate. Small
drugmakers where much of the innovation in medicine originates can’t afford to
provide their compounds for free, and terminally ill patients with no other
options may be eager to pay for access. There would be little protection for
patients already grappling with a tumultuous time in their lives, adding
financial risk to the known medical gamble.
Health
insurers don’t typically pay for treatments that haven’t been approved by
regulators or proven to work in clinical trials. That means patients would have
to pay for the therapies out of their own pockets.
Right to
Try, signed by President Donald Trump last month, allows doctors of patients
with life-threatening diseases to work directly with drug companies for access
to experimental drugs. The law aims to simplify the process and bypass the U.S.
Food and Drug Administration almost entirely -- its chief sponsor, Republican
Senator Ron Johnson, recently put it bluntly,
saying the goal was to “diminish the FDA’s power over people’s lives, not
increase it.”
Critics
said the lack of oversight may make patients even more vulnerable as they near
death, creating opportunities for unscrupulous companies or clinics to take
advantage of them.
Brainstorm, listed in the U.S. and run from New York and Israel, is
trying to address the tension head-on. After requests for access to
Brainstorm’s NurOwn experimental drug came pouring in following the passage of
Right to Try, the company held conference calls with patients and investors to
talk through details.
The
shares closed up 2.8 percent to $4.41 on Wednesday, after rising 9.4 percent
this year as of Tuesday’s close.
Only
doctors who participated in the drug’s clinical trials would be included in the
Right to Try program under consideration at Brainstorm, CEO Lebovits said. It would
be a semicommercial enterprise with modest profits that wouldn’t exploit
patients’ desperation, he said. The company, which had operating costs of
$5 million last year, would work to obtain funding from foundations or other
charitable organizations to help pay for at least one financially strapped
patient for every two who are able to pay for it, he said.
NurOwn is
being tested for amyotrophic lateral sclerosis, or ALS. The progressive disease
erodes nerve cells, weakening the muscles throughout the body and gradually
leaving patients unable to walk, speak and breathe.
The
Brainstorm treatment is crafted for each individual in a process that includes
extracting stem cells from the bone marrow. Its price could be similar to
recently approved breakthrough cell therapies known as CAR-Ts, CEO Lebovits
said. Those two cancer treatments,
also tailored for each patient, cost about $375,000 and $475,000.
NurOwn’s
mechanism doesn’t reverse the underlying cause of ALS, meaning it’s unlikely to
cure the disease. A mid-stage study involving 48 patients found those given the
drug appeared to respond, though the benefit didn’t last for most. The final
results may not support approval.
Willing to Pay
Still,
Matt Bellina, a former Naval officer and proponent of Right to Try, is among
almost 1,000 patients hoping to get access to NurOwn. His Lou Gehrig’s disease
is so advanced that he doesn’t qualify for the late-stage study that’s
underway. So he’s willing to pay.
“We all
have to do our part,” said Bellina, who has gotten positive reports from
patients who participated in NurOwn’s studies.
https://www.bloomberg.com/news/articles/2018-06-20/the-price-to-try-a-drug-could-be-300-000-for-dying-patients?utm_campaign=KHN%3A%20First%20Edition&utm_source=hs_email&utm_medium=email&utm_content=63934055&_hsenc=p2ANqtz--9JRZC3Rkz1sD76ZK4hbBBg10rKXNSc-gv0YvOWOZsTTCF_S6eZw96o3US1gp-5b_Z94ayFcHBZgQIsl4G4JGk1WdI3g&_hsmi=63934055
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