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Expanded Access to Experimental Therapies
“A major goal of drug regulation must be to speed the journey from
laboratory to bedside of important new drugs for devastating illnesses.”
(1) One example is that under certain circumstances, the FDA allows
patients who are seriously ill or have life-threatening conditions to
access investigational drugs not yet on the market.
Treatment use of experimental drugs is generally grouped into two
categories according to the number of people treated: expanded access
and single patient use. One type of expanded access program is called
the Treatment IND (investigational new drug). It allows multiple
patients for whom there are no other approved, satisfactory treatments
available, to use investigational drugs that have shown promise in
clinical trials, but have not yet reached the market.. Patients receive
the treatments outside of the ongoing clinical trials.
Single patient use (sometimes called “compassionate use”) is an
exception granted by the FDA for individual rather than group treatment
use of experimental drugs when a patient might benefit from it, but is
ineligible for the current clinical trial. In effect, the FDA allows an
individual patient to become his or her own study.
The FDA may grant a compassionate use or Treatment IND if :
However, in no case is the sponsor legally required to provide the
experimental treatment to a terminally-ill patient. Even if the patient
has obtained FDA permission, the government cannot compel the drug
company to provide the treatment.
Should the sponsor agree to provide the drug, they would be responsible
for reporting to the FDA. However, if the commercial sponsor is
unwilling to assume responsibility for a special exception, an
investigator or the patient’s doctor may take responsibility for
treating the patient. They would then need to obtain the drug from a
manufacturer, apply directly to the FDA for an IND and report
information about the effects of the drug to the FDA. Additionally, an
Institutional Review Board must evaluate the situation and grant
approval.
“The FDA has worked diligently to balance two compelling, and sometimes
competing, factors,” says FDA Commissioner Jane E. Henney, M.D. “On one
hand, there is the need for the disciplined, systematic, scientifically
controlled studies necessary to identify treatments that may improve
patient health and that lead to the approval of new drugs. At the same
time, there is the desire of seriously ill persons, with no effective
options available, to have the earliest access to unapproved products
that could be the best therapy for them.”
Are the risks greater than the benefits when trying an experimental
medication? That depends on your viewpoint. Even if an unproven
treatment does not increase the long-term survival prognosis, the
patient and the family may feel better knowing that they have exhausted
all available options. Staff from the Office of Special Health Issues
and the Center for Drug Evaluation and Research’s drug information
branch often explain the process for getting access to an experimental
medication, but do not steer patients in one direction or the other.
Information is provided so patients, in consultation with their
physicians, can make their own informed decisions.
Differing viewpoints of Treatment INDs
In addition to benefiting individual patients, Treatment INDs may be of
benefit to many in that they generate useful information about how
specific drugs affect larger segments of the patient population than
might otherwise receive it in a clinical study. The process:
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Involves wider treatment use of unapproved agents.
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Accelerates the FDA’s new drug application review process without
compromising approval requirements for safety and effectiveness.
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Shortens the time devoted to pre-approval drug testing, eliminating
unnecessary, duplicative studies, and expediting the review of
innovative agents for the most serious or life-threatening conditions.
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Encourages FDA reviewers to help drug developers plan studies that
generate information necessary to make decisions about approvability.
The Society for Clinical Trials, however, argues that Treatment INDs
ultimately could destroy the clinical trial process, which remains “the
surest way to determine whether a drug works and is safe.” The group
argues that “Vastly broaden(ing) access to medications that have shown
only limited evidence of safety and, often, none at all of efficacy
…would devastate the clinical trial system that has fueled unparalleled
medical progress over 45 years.”
Do Treatment INDS provide meaningful access?
Since the final treatment IND
rule was published in 1987, through 2000, the FDA has made only about
forty drug or biologic investigational products available to patients
early and has approved thirty-six.
Critics of the FDA policy contend that “compassionate use” is not really
a meaningful exception for the terminally and seriously-ill because
there are few incentives in the current regulations that would encourage
companies to agree to provide experimental therapies to individuals or
groups outside of the clinical trial process. Most requests have
been refused by drug sponsors, who fear that the legal and financial
risks to their companies would outweigh the benefits.
Some solutions have been proposed. In December 2006, the FDA proposed
new rules, clarifying the existing regulations for the treatment IND and
expanding the situations where an experimental drug treatment is
available to a terminally or seriously-ill patient. The FDA would be
required to determine that providing the experimental drug “will not
interfere with the ... clinical investigations that could support the
marketing approval” of that drug.” It would also establish “clear
eligibility criteria and submission requirements, the proposed rule
would ease administrative burdens ... on sponsors willing to make
promising unapproved therapies available for treatment use.”
Other critics of the FDA policy say that the government should provide
sponsors with more protections that would “eliminate the disincentives,
including liability, cost recovery, and risks to achieving marketing
approval, that prevent drug sponsors from providing INDs for treatment
use.”
Legislative reforms have also been proposed, such as Access, Compassion,
Care, and Ethics for Seriously Ill Patients Act (S3046). Backed by the
Abigail Alliance for Better Access to Developmental Drugs, the bill
would create a new conditional approval system for drugs, biological
products, and devices that is responsive to the needs of seriously ill
patients.
However, others agree with Dr. Henney that, “Continuing to shorten
review times and work with industry to shorten development times for
drugs, biologics and medical devices is the best way to provide all
Americans with access to useful medical treatments,”
Sources:
- FDA Consumer magazine, January-February 2000, “Experimental
Treatments? Unapproved but Not Always Unavailable” by Larry Thompson.
Accessed July 29, 2008 at:
http://permanent.access.gpo.gov/lps1609/www.fda.gov/fdac/features/2000/100_exp.html
- Code of Federal Regulations 21CFR312.34, Treatment use of an
investigational new drug
- Food and Drug Administration. Protecting human study subjects (?)
- Cerino, Joanna R. The Statutory Limits of Compassion: Can Treatment INDs,
Provide Meaningful Access to Investigational Drugs for the Terminally
Ill?,
Temple Journal of Science, Technology & Environmental Law,
Food and Drug Administration. Protecting human study subjects (?),
Spring, 2008
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FDA Proposes Rules Overhaul to Expand Availability of Experimental Drugs
FDA News.Dec. 11,2006. Accessed July 29, 2008 at:
http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/2006/ucm108798.htm
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