03/13/2018 05:05 AM EDT
Congress
may be on a speedy path to lifting the hopes of terminally ill patients.
Whether it’s anything more than a feel-good exercise is an open question.
The House
of Representatives is expected to deliver the deciding vote for a right-to-try
bill Tuesday that President Donald Trump touted in his State of the Union
address and would give terminally ill patients — or those likely to die
prematurely — access to experimental medicines without the FDA’s blessing. The
Senate, which already passed its own bill, is considered likely to adopt the
changes.
The
right-to-try idea, conceived by a little-known libertarian think tank, has an
instantaneous appeal. But FDA, drug industry and health policy experts say it’s
unlikely to significantly increase the number of patients who get unapproved
drugs. And the few who do could be exposing themselves to unnecessary risks
while compromising medical research.
“I think
this is feel good legislation for legislators, rather than meaningful
legislation for people in need,” said Kenneth Moch, president and CEO of
Cognition Therapeutics, which is trying to develop treatments for neurological
conditions like Alzheimer’s disease.
Former
FDA Commissioner Robert Califf said he worries any right-to-try law will set
patients up for disappointment. “What it may do is to raise expectations of
vulnerable people and their families that there is some miracle cure that is
going to be made available.”
Even if
more companies release products under right-to-try — a prospect Califf thinks
is doubtful — the odds for helping patients are long. That’s because most
promising new drugs end up being too dangerous or simply don't work.
FDA
estimates that only five to seven of every 100 drugs that enter the first phase
of human testing eventually get approved. The rest have risks that outweigh
their stated benefit or don't deliver on those promises.
Critics
say the problem with right-to-try is it mistakenly presumes the FDA is the
barrier to patients accessing unapproved medicines outside clinical trials.
FDA’s
compassionate use program already provides experimental drugs to very sick
patients, approving 99 percent of the requests it receives each year. It’s
undergone changes in recent years to make it easier for doctors and patients to
navigate the process.
The
hurdles to accessing experimental medicines largely rest with pharmaceutical
companies that have a variety of reasons for turning patients down. New drugs
are expensive to make and companies need enough product for clinical trials —
the only way to get FDA approval. They also need patients to be incentivized to
participate in studies to get a sufficient cross-section to evaluate the
product. Obtaining an experimental drug outside of a trial carries the risk of
disrupting the costly and time-consuming process, especially if a person
outside of a trial is harmed by the medicine.
“The
right-to-try approach blames an innocent entity for access problems, and by
pushing legislation that targets the FDA, those who advocate for right-to-try
are not putting forth viable solutions to these problems. The only way forward
is to work with companies and to find out what would make them willing to
provide their experimental products to patients who wish to try them,” said
Alison Bateman House, a bioethicist at New York University.
The Trump
administration’s FDA chief raised these issues with House lawmakers in the
fall.
“There is
a perception that certain products that aren’t being offered under FDA expanded
access … will be offered under right-to-try,” Commissioner Scott Gottlieb told
the House Energy and Commerce Committee in October. “I don’t see that.”
Gottlieb,
who homed in on the barriers drug companies have to providing experimental
medicines, also emphasized that the vast majority of drugs provided to patients
through its compassionate use program turn out to be ineffective. “We know that
70 percent of all drugs that are offered under an expanded access setting are
never approved by FDA," Gottlieb said.
Right-to-try
has been opposed by mainstream medical organization and patient groups like the
American Cancer Society Cancer Action Network, the American Society of Clinical
Oncologists and the National Organization for Rare Disorders.
The drug
industry also has issues. Johnson & Johnson has already said it
will continue to incorporate FDA review in all experimental access requests,
noting the FDA may be “uniquely aware” of safety information that could affect
whether a patient should receive a drug.
This
combination of resistance — from the FDA chief, medical groups and the drug
industry — threatened to doom right-to-try in the House, even though the Senate
passed the bill by unanimous consent in August. For months, Energy and Commerce
leadership appeared disinclined to move a bill and worked with Democrats on an
alternative proposal.
But then
came Trump's State of the Union appeal and congressional Republicans' annual
retreat, where the White House aggressively pushed for passage of the measure.
“The
State of the Union targeted this policy as an administration goal, thrusting
the conversation into the national spotlight,” an Energy and Commerce
Republican aide told POLITICO. This spurred talks with members spearheading the
law, including Sen. Ron Johnson and
Reps. Andy Biggs and Brian Fitzpatrick. The committee worked with
those lawmakers and Vice President Pence to craft the consensus language.
Energy
and Commerce Chairman Greg Walden sought
assurance from FDA that tweaks to the law would ensure drug companies wouldn’t
have perverse incentives to choose the FDA compassionate use program over
right-to-try or vice versa. He also sought FDA feedback on getting the fastest
update on patients using right-to-try, an aide familiar with the negotiations
told POLITICO.
The House
bill also underwent tweaks to satisfy some concerns from FDA and outside groups
that the law put patient safety in jeopardy. It includes new requirements that
FDA be made aware when drugs are provided under right-to-try and gets reports
of any safety events with the drugs.
Those
changes have been lauded — but critics still argue they're not enough.
“I think
they’ve certainly tried to take steps to add some important provisions” like
strengthening the informed consent of patients, said Jeff Allen, president and
CEO of the Washington-based Friends of Cancer Research. But Allen said the bill
still violates one of his organization's guiding principles: “The FDA is not
the problem and taking them out would only introduce more problems,” he said.
Allen
said patients may get more access to drugs under right-to-try, but they are
unlikely to come from credible companies who he said will prefer the FDA’s
process. “Unfortunately, I fear it will be drugs they shouldn’t be receiving or
could be extremely unsafe or have flaws in the manufacturing practice or other
risks that may not be known at the time.”
Such
concerns could stoke Democratic resistance, though probably not enough to put
the bill in danger. The legislation requires the support of two-thirds of
members present and voting because it is being considered under the expedited
process known as suspension of the rules.
Energy
and Commerce ranking Democrat Frank Pallone said the bill “provides
false hope to patients and their families, who will assume they will have
access to investigational therapies even though the bill does nothing to
require manufacturers to provide these therapies to the patient.” Only one
doctor has publicly said he provided patients with drug under state
right-to-try laws.
A
Democratic aid expressed frustration with the way Republicans are “jamming” the
bill through the House, noting suspension of the rules is supposed to be
reserved for noncontroversial measures.
If the
legislation passes the House, it will need to go back to the Senate to iron out
differences such as precisely which patients would be eligible for
right-to-try. Senate leadership would not comment on its plans to take up the
legislation.
Regardless
of the outcome, the House vote marks a big victory for the libertarian
Goldwater Institute, which pushed the bills through the states and on the
federal level. The issue is the first in a broader anti-regulatory agenda — and
could lead to the roll back of other FDA laws designed to protect consumers
from unsafe, and unproven drugs.
The group
already has its next target: FDA marketing rules that keep drug companies from
promoting their products for unapproved uses.
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