Statement from the NYU Langone Health Working Group on

Compassionate Use and Pre-Approval Access

Thursday, March 15, 2018

The NYU Langone Health’s Working Group on Compassionate Use and Pre-
Approval Access (CUPA) is pleased that the House of Representatives did not
advance H.R.5247, a federal “right to try” bill. Terminally ill patients without
approved treatment or clinical trial options should have every tool at their disposal
to empower them to seek access to investigational products that have not yet been
approved by the FDA. H.R.5247 would not accomplish this goal.

As CUPA members have repeatedly argued in hundreds of op-eds, peer-reviewed

papers, and talks, no current version of right to try legislation will help patients get
experimental drugs. Right to try wrongly frames the FDA as the primary barrier to
these drugs. Data show this is not the case.

To increase patient access to investigational drugs, policymakers must 1) help

educate everyone on expanded access and on how to navigate the FDA program
and 2) find ways to make the pharmaceutical and biotechnology industries more
willing to provide access.

The Division of Medical Ethics at NYU Langone Health is an active participant in

these two endeavors. It has sponsored conferences, webinars, and talks to educate
various stakeholders on what expanded access is, how to utilize it, and how to
improve it. In partnership with Janssen Pharmaceuticals, it established
the Compassionate Use Advisory Committees (CompAC): independent, external,
expert committees that guide Janssen in providing its investigational medicines to
patients in transparent, fair, prompt, evidence-based, and patient-focused ways. In
its pilot program, the first CompAC recommended approving 180 physician
requests for individual patients to receive an investigational medicine — far more
than have been granted under any state right to try law. Since this pilot program,
the CompAC model is now being expanded within Janssen and other companies.
In the debate over H.R.5247, proponents of the bill made many inaccurate claims.
We would like to set the record straight about five of the most egregious,
deceptive, and flat out wrong claims:

Claim: The FDA’s existing Expanded Access program only benefits around 1,000
patients per year.

Fact: This claim falsely conflates the number of applications for expanded access
that the FDA receives with the number of patients that receive access to an
experimental product through expanded access. Most expanded access applications
are for individual patients, but some are for more than one patient, sometimes in
the thousands. Neither the FDA nor any other entity tracks how many patients
receive drugs through these large-scale programs.

Claim: Thousands of people in France get access to drugs through pre-approval

access but in the U.S. it’s only around 1,000 annually — and France is a much
smaller than the U.S.

Fact: It is inappropriate to compare the two countries. First, France has a national
healthcare system, and the U.S. does not. Not all Americans can afford to visit
centers of clinical excellence and/or research specialty care, where the majority of
expanded access requests in our country originate. Second, the French healthcare
system pays companies for their investigational drugs; in the U.S., patients are
generally responsible for paying for any charges. Right to try does nothing to
increase affordability of experimental medicines: in fact, the legislation
specifically states that insurers do not have to pay for investigational medical
products. Third, new drugs are approved faster in the U.S. than in France, so many
drugs patients receive through expanded access in France are approved and
available for prescription here. Finally, patients from across Europe travel to
France for experimental drugs because of financial incentives. Thus, the number of
patients receiving drugs in France includes patients from across Europe.

Claim: Drugs that have completed a Phase 1 clinical trial are generally safe.
Reality: The scientific and pharmaceutical communities agree that Phase 1 testing
is an extremely low bar for drug safety. Phase 1 trials usually enroll very small
numbers of healthy, paid volunteers. Certain types of drugs, like chemotherapies
for cancer, cannot ethically be tested on healthy volunteers and are tested on
patients. Given the small numbers of subjects involved, it is unlikely that serious
adverse events will be detected. About 75 percent of drugs that pass Phase 1
ultimately utterly fail due to serious safety issues or lack of efficacy or both.

Claim: State right to try laws are already helping patients.

Fact: Proponents of right to try have spoken only about one doctor who has used a
state law to treat patients. Although this doctor claims to have treated almost 100
cancer patients under right to try, CUPA does not consider this to be a
substantiated use of a right to try law, as the investigational product was
simultaneously available through the FDA’s expanded access program. Patients
who were treated by this doctor could have received, and did receive, this
experimental product from other doctors. This product has recently been approved
by the FDA.

Claim: There is nothing worse than death, so why not allow terminally ill patients
to try anything they want?

Fact: Patients do get to make this choice and have for decades. Both right to try
and the FDA require patients to use experimental drugs under a physician’s care.
Yet many doctors are not familiar with using experimental drugs. Only the FDA is
armed with exclusive proprietary information about these drugs and thus can offer
guidance to help patients and doctors understand which drugs may be the most
beneficial for them.

This one point bears repeating: Drug companies are not obligated by any right to
try law to provide their drugs to patients. Right to try grants no right to try a drug,
just the right to ask for a drug. Patients already have this right to ask; they have for
decades. Ethically minded drug companies are wary of providing their
investigational products to sick patients without the guidance of the FDA: thus,
removing the FDA from expanded access is likely to result in a net decrease in
patients gaining access to investigational drugs.

CUPA has several recommendations (PDF attached) that we believe will boost
pharmaceutical industry participation in expanded access while simultaneously
protecting the safety and health of patients. We urge legislators to consider them.

Terminally ill patients who have no treatment options deserve the chance to try to
save their own lives. Right to try does would not help patients and could cause
them serious harm. Now that H.R.5247 has been rejected by the House, we hope
all stakeholders will work together to develop realistic, actionable, patient-focused,
and results-oriented policies.

For more information on CUPA’s published work, proposals, and members, visit
our website.