B2300 An opposing view of
Oregon’s “Right to Try” bill
Date: March 9th, 2015
To: Rep. Buehler and members of the HB 2300 working group
From: Samuel Metz, MD
HB 2300, Oregon’s “Right to Try” (RTT) bill,
would give terminally ill patients access to investigational drugs
without FDA approval. Because of the complexity of this issue, I
reviewed opinions from multiple sources and present them here. While I
conclude that Oregon should not pass this RTT bill, others who examine
this material may conclude otherwise.
This review begins with an estimate of the number of patients who might
be either harmed or helped by this bill. It then incorporates opinions
from medical ethicists, the pharmaceutical industry, state and national
medical associations, patient advocacy organizations, and economic
A. Numbers of patients who might harmed or helped
From 2010 to 2014, the Food and Drug Administration (FDA) received 6129
applications from physicians applying for “expanded access” on behalf of
their terminally ill patients. The FDA expanded access program allows
patients to receive investigational drugs that lack demonstration of
safety or efficacy. The FDA approved 6098 of these applications (99.4%)
and did not approve 33 applications.
The average number of non-approved applications each year is eight.
Applications were not approved primarily because the FDA considered the
drugs too dangerous or the FDA suggested other drugs that might be more
appropriate for that patient. In these cases, the physician withdrew the
application to pursue the suggestion.
Based on these national numbers, Oregon’s HB 2300 might allow one
patient every 12 years to receive an investigational drug who would not
otherwise receive FDA approval.
One patient advocate estimates this number might quadruple with passage
of the bill. Because of
RTT laws passed in other states, the FDA created a shorter written
physician application form (one page requiring 45 minutes to complete).
This new form may decrease the number of patients bypassing FDA
approval. The FDA continues to provide emergency approval over the
Some terminally ill patients who apply to the FDA expanded access
program may do so because they are not eligible for clinical trials.
Other patients may apply for expanded access rather than possibly
receiving a placebo rather than active drug in a clinical trial.
Regardless, all patients who receive investigational drugs either
through the expanded access program or through state RTT laws become
ineligible to participate in any clinical trial. Additionally, there are
no programs that track the outcomes of patients who do not participate
in clinical trials nor are these outcomes included in clinical reviews
of safety or efficacy.
By removing pathways that include the formal FDA data collection
process, RTT laws may decrease the number of patients participating in
clinical trials of promising investigational drugs. Thus, RTT laws might
delay the availability of these drugs to future patients suffering
Oregon’s RTT bill could potentially benefit a small number of terminally
ill patients (perhaps less than eight per century). It also could
potentially delay the availability of drugs with documented safety and
efficacy for future patients with terminal illnesses.
B. Medical ethicists and legal commentators
I found seven medical ethicists and legal commentators opposing RTT
I found none supporting them.
Medical ethicists expressed these concerns:
RTT laws, prevent terminally ill patients from participating in
clinical trials of investigational drugs already known to have a
higher probability of producing benefit.
RTT laws allow the exploitation of terminally ill patients and their
families by businesses with a financial interest in promoting
The possible side effects of investigational drugs may cause more
harm to terminally ill patients than simply an earlier death. Some
possible side effects may not affect life expectancy but could
reduce the remaining quality of life.
Neither the patient nor the attending physician has the same
extensive information available to the FDA about investigational
drugs. A risk-benefit assessment without FDA consultation is likely
to be incomplete, or based on hearsay. One review referred to
Risk comprehension of experimental drugs is low among the general
population and possibly lower still among families of terminally ill
patients. Risk-benefit ratios may not be understood in this
There is no legal or ethical precedent for exempting from FDA
supervision one set of patients (those expected to die of their
disease within 12 months) while not exempting others (e.g., those
with painful diseases, those with severe immobility, or with life
expectancies longer than 12 months).
Patient demand for non-approved drugs may place attending physicians
in the ethically challenging position of wanting to honor a
patient’s wish to try anything and the obligation to protect their
patient from harm.
C. State and national medical societies
Six states passed RTT laws. The opinions of their state medical
associations vary considerably and are not uniform.
The executive board of the Medical Society of Virginia unanimously
supported that state’s RTT bill.
The Louisiana State Medical Society submitted a “green card” in
support of its state RTT bill during a legislative hearing, but
offered no other opinion. The Governmental Affairs officer said the
card was submitted for “purely political reasons” to show support
for the legislator sponsoring the bill.
The Society did insist on providing legal immunity to participating
The Arizona Medical Association offered no opinion on Arizona’s
ballot measure. The association’s Vice President of Operations
called the measure, “rice cake,” claiming the law provided nothing
of substance to patients. She regarded the ballot measure as a
political tactic to defeat an incumbent Arizona legislator by his
challenger, the wife of an official at the Goldwater Institute.
The Missouri, Michigan, and Colorado medical associations took no
position on their state’s RTT laws.
California Medical Association spokeswoman expressed reservations
about that state’s proposed RTT bill:
“While the FDA process can be slow, CMA feels it is the appropriate
way to address patient safety concerns as part of the risk/benefit
evaluation when treating illness. Sick, vulnerable and trusting
patients deserve the highest quality of care and safeguards from
false hopes and potentially fraudulent schemes.” 
In 2007, the U.S. Court of
Appeals heard Abigail Alliance v. von Eschenbach
495 F.3d 695 (D.C. Cir. 2007); this court ultimately ruled
that terminally ill patients do not have a constitutional right to use
medications without FDA approval.
The American Society of Clinical Oncology and the
Association of American Medical Colleges signed amicus briefs in support
of the FDA.
On appeal, the Supreme Court declined to hear this case.
In a previous Supreme Court case,
United States v. Rutherford,
442 U.S. 544 (1979),
Justice Thurgood Marshall noted,“Nothing
in the history of the [Food, Drug, and Cosmetic Act] suggests that Congress
intended protections only for persons suffering from curable diseases.”
The American Medical Association made this comment about the
Abigail ruling: “AMA
policy supports expanded access to promising developmental drugs, as
long as any changes do not compromise the clinical trials process that
gives doctors the necessary efficacy and safety information about a
D. Pharmaceutical Industry
Two national organizations representing the pharmaceutical and
biotech industry issued comments opposing RTT laws.
Sascha Haverfield, vice president of scientific and regulatory
affairs at the Pharmaceutical Research and Manufacturers of America (PhRMA),
expressed the concern noted above that RTT laws may hamper clinical
trials and thus delay the availability of proven drugs to future
The Biotech Industry Organization
(BIO) Board Standing Committee on Bioethics has similar concerns and
added these :
“A patient's right
to treatment based on his or her autonomous decision-making ability
does not supersede a company's ethical responsibility to develop and
market safe and effective products as fast as possible.”
“A patient suffering from a life-threatening illness may not be able
to provide consent that is truly informed when receiving a product
under an early access program.”
Joan Koerber-Walker, president and CEO of the Arizona Bioindustry
Association, does not support RTT laws.
Richard Garr, CEO of Neuralstem, a Maryland company which
manufactures stem cells to treat neurologic diseases, supports RTT
E. Patient Advocacy Organizations
I made contact with three patient advocacy organizations. All were
deeply distressed by the failure of the FDA to rapidly investigate and
approve investigational drugs to treat terminal or rare diseases. They
differed in their opinions about RTT laws. Statements from these
organizations are attached and are summarized here. One additional
comment from another patient organization follows.
Frank Burroughs of
the Abigail Alliance was the plaintiff in
Abigail Alliance v. von Eschenbach.
He feels passionately that patients will benefit from RTT laws; he
was instrumental in the passage of Virginia’s RTT law.
Terry Kalley of
Freedom of Access to Medicines adamantly supports RTT laws. He also
regards RTT laws as a means to compel FDA reform and was
instrumental in Michigan’s passage of a RTT law. In his opinion, the
ultimate goal is not to pass RTT laws in every state, but to
pressure the FDA to create more expeditious means to test, approve,
and make available beneficial drugs.
Associate Director of State Policy,
National Organization for Rare Disorders, does not support
RTT laws. Here is an excerpt from his attached statement:
“While NORD is encouraged by state efforts
to ensure patients with life-threatening illnesses have access to
potentially life-saving therapies, NORD does not believe that
"right-to-try" laws are the answer.”
The National Coalition of Cancer Survivorship signed the amicus brief in
the Abigail case which
supported FDA supervision.
F. Economic policy organizations
Several respected policy organizations support RTT laws. All have a
similar mission statement promoting free enterprise and small
government. Their primary, though not only, argument is that government
should not intervene in the rights of terminally ill patients with
little to lose from taking investigational drugs.
Goldwater Institute 
The American Conservative
Cascade Policy Institute
G. The FDA
The F.D.A. does not have a position on RTT laws. It encourages
patients to use the existing expanded access program.
The number of patients who may potentially gain access to
investigational drugs through an Oregon RTT law is small. We don’t know
if these terminally ill patients will be helped or harmed by taking
investigational drugs without FDA supervision.
Medical ethicists, business organizations, the American Medical
Association, and one patient advocacy organization are concerned that
RTT laws will harm future patients by reducing the number of patients
participating in clinical trials of investigational drugs. This may
delay the evaluation and release of future drugs demonstrated to be safe
Medical ethicists and the California Medical Association expressed
additional concerns that RTT laws may create a predatory environment in
which businesses exploit the desperation of terminally ill patients and
State medical associations are divided in their opinions.
One patient advocacy organization fully endorses RTT laws. Two endorsed
RTT laws as a means to compel the FDA to reform its drug approval
process. Their ultimate goal is for the FDA to provide patients with
safe, efficacious, life-saving drugs as expeditiously as possible.
Economic policy institutes support RTT laws a means to reduce government
interference in private lives.
In summary, two groups endorse RTT laws but with differing motives.
Patient advocacy organizations want to promote faster drug evaluation by
the FDA. Economic policy institutes want to promote personal liberty.
I conclude that HB 2300 may hurt more patients than it helps and
therefore recommend that Oregon not pass HB 2300.
FDA Development Approval Procoess.
personal communication, February 21, 2015
Draft Guidance for Industry
Arthur L. Caplan, Ph.D., Drs. William F. and Virginia Connolly Mitty
Professor and founding head of the Division
of Bioethics at New York
University Langone Medical Center and Alison Bateman-House,
Yoram Unguru, MD,
Johns Hopkins Berman Institute of Bioethics
Alta Charo, professor of law and bioethics at the University of
Dr. Ezekiel Emanuel, chair of the Department of Medical Ethics and
Health Policy at the University of Pennsylvania
David H. Gorski, MD,
Medical Director of the Alexander J. Walt Comprehensive Breast
Center and chairman of the board of directors for the Society
for Science-Based Medicine
George Annas, JD, MPH, William Fairfield Warren Distinguished
Professor and Chair of the Department of Health Law, Bioethics &
Human Rights of Boston University School of Public Health
Henry Greely, JD. Director of the Stanford University Center of
Law and the Biosciences
(link no longer valid)
University of Georgia School of Law
Jonathan J. Darrow, Ameet
Sarpatwari, Jerry Avorn, and Aaron S. Kesselheim.
Practical, Legal, and Ethical Issues in Expanded Access to Investigational
N Engl J Med 2015;
Senior VP, Health Policy & Planning, Medical Society of Virginia,
personal communication, February 21, 2015
Jennifer Marusak, Vice President of Governmental Affairs, Louisiana
State Medical Society, personal communication, February 24, 2015
Carol Wagner, Associate Vice President of Operations, Arizona
Medical Association, personal communication, March 5, 2015. Note:
this author attended elementary and high school in the contested
legislative district mentioned above.
Personal communications with officers of the Missouri, Michigan, and
Colorado medical societies
Peter D. Jacobson, Wendy E.
A New Era of Unapproved Drugs. The Case of Abigail Alliance v Von
Eschenbach. JAMA 2007; 297:205.
Sascha Haverfield. “State “Right-to-Try” Legislation, While
Well-Intentioned, Is Unlikely to Achieve Goal of Bringing Safe and Effective New
Medicines to Patients.” PhRMA June 14, 2014
The Biotech Industry Organization (BIO) Board
Standing Committee on Bioethics
HCR 2005 – “Right to Try” Won’t Benefit Patients
(link no longer valid)
Frank Burroughs, see attached statement
Terry Kalley, personal communication, February 26, 2015, and
Tim Boyd, see attached statement
(link no longer valid)