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January 15, 2003
Conceptual New Early Conditional Approval
Drugs, Biologics and Devices Intended to Treat
Life-Threatening Diseases with Unmet Needs
Overall Concept: Create a new Food and Drug Administration (“the FDA”) authority to grant
early conditional approval (“ECA”) of promising drugs, biologics and devices (“drugs”) intended
to treat life-threatening diseases with unmet needs.
Basis for the Idea: Annually, hundreds of thousands of Americans find themselves suffering
from terminal diseases with no approved drugs capable of providing a cure or control of their
disease. In most cases, at least one partially-proven investigational drug with potential to
effectively treat their disease exists, but is generally unobtainable because the drug has not been
approved by the FDA for marketing. The current regulatory system has not resulted in clinical
trials of sufficient scope and geographic distribution to provide reasonable options for the
majority of patients suffering from life-threatening diseases with unmet needs, and is not likely
to result in a clinical trials system adequate for this purpose in the foreseeable future. Privatesector participation in compassionate use and expanded access programs, although slowly
improving, has been too limited and uncertain to fill the gap. The system that governs the FDA
and our nation’s drug development and approval process fails to serve hundreds of thousands of
Americans each year, thus depriving them of their “right to live,” in this case more accurately
described as the “right to try to live.” The Abigail Alliance maintains that the United States
government has a moral and legal obligation to administer its regulation of drugs for lifethreatening and terminal diseases in a compassionate manner and in the best interests of all
Americans, including especially those who have exhausted FDA-approved and otherwise
unavailable treatment options. It is a civil and human rights issue that adversely impacts
Americans from all walks of life. The FDA is not presently inclined or empowered to act in the
best interests of all Americans afflicted with life-threatening and terminal diseases.
Conceptual Program: Create through legislation a new “Early Conditional Approval”
authority designed to allow limited marketing of drugs, biologics and medical devices for
patients that have in the judgment of their physician: (1) exhausted all approved options for
which they are reasonable candidates; (2) already failed treatment with drugs available through
open clinical trials for which they are both eligible and reasonable candidates (including
geographic considerations); and (3) been found ineligible or poor candidates (due to condition or
geographic considerations) for open, ongoing clinical trials or Treatment Investigational New
Drug protocols (expanded access programs). The new authority would be intended to
complement rather than replace the current system of clinical trials, compassionate use,
expanded access, accelerated approval, and full approval.
Conceptual Approval and Withdrawal Criteria: The FDA would grant ECA to an applicant
for limited marketing based on the results of a Phase I trial demonstrating a safety profile
sufficient to support conduct of a Phase II or Phase III clinical trial intended to further test the
Abigail Alliance for Better Access to Developmental Drugs
Conceptual New Early Conditional Approval
January 15, 2003
safety and/or efficacy of the drug and preliminary, but not necessarily conclusive evidence of
effectiveness based on case-history data from a small number of patients. Sufficient preliminary
evidence of effectiveness would, for example, consist of documented tumor regression in a very
small number of patients (e.g., two or three and certainly fewer than ten) with forms of cancer
that rarely or never regress spontaneously and the observed response cannot be readily explained
by some other factor. No statistical support would be required for ECA and the needed data
would often be generated during a Phase I clinical trial or from among the initial patients
enrolled in a Phase II clinical trial. ECA would most often be used, but would not be strictly
limited to, cases where demand for the drug outside clinical trials is likely to exceed the level of
demand that could reasonably be met through compassionate use and/or expanded access
programs. The FDA would not develop new criteria for this test, but rather would grant or deny
approval within 30 days of a request for ECA, concurrent with review of a Phase II or III clinical
trial protocol. A request for ECA would not be unreasonably denied, and the request would not
be used to delay or increase the requirements for approval of a clinical trial protocol.
Continued diligent pursuit of clinical trials and other testing required for obtaining accelerated
and/or full approval would be the primary condition of the program. Prescription and
administration of the drug would be allowed only by qualified, licensed physicians and would be
subject to informed consent from the patient and adverse event reporting by the physician. The
marketing entity would agree to provide all known information regarding safety and efficacy in
informed consent documents and would be required to review and update the informed consent
documents on a specified schedule.
Private and public health insurance providers would be required to cover the costs in the same
manner by which they cover other approved treatments for the disease being treated. Off-label
use of the drug also would be permitted, but would be covered at the option of insurance
providers. Charges for drugs marketed under ECA could be unregulated, limited by a formula
tied to similar treatments and/or direct costs of manufacture, a regulatory maximum charge per
treatment or dose, or some other mechanism.
ECA would be withdrawn if the drug receives accelerated or full approval from the FDA. ECA
could be withdrawn, after careful consideration of the impact on patients, if there is no entity
pursuing eventual full approval for the drug from the FDA. ECA could also be withdrawn if the
drug is found to be ineffective or unacceptably dangerous for the patient population(s) most
likely to be treated; except that any patient receiving the drug under limited conditional approval
would be allowed to continue receiving the drug subject to updated informed consent. When
considering withdrawal of early conditional approval, the FDA would be required to demonstrate
that the evidence regarding lack of effectiveness is compelling (e.g., almost no one can expect to
benefit from the drug), or that the dangers posed by the drug clearly outweigh the benefits (e.g.,
more patients will experience a serious life-threatening side effect known to be caused by the
drug than will be helped by the treatment).