FDA Issues Final Rule to Clarify Patient Access to Experimental Drugs

In an effort to broaden and clarify access by individual patients to experimental medicines that have not yet obtained FDA approval or haven’t been approved for a new use, the Food and Drug Administration issued two new rules on August 12, 2009.

  • The Expanded Access rule establishes how seriously ill individual patients or groups of patients can obtain investigational medicines while at the same time preserving the clinical trials process.
  • The Charging rule clarifies when a manufacturer of an investigational drug may charge for it and what costs can be recovered in those charges.

In addition, the FDA established a website for consumers to explain access to investigational drugs through clinical trials or through the expanded access process.

Expanded access — sometimes called compassionate use — makes experimental or investigational medicines available to patients with serious or life-threatening illness  who have no other comparable treatment options.

Under the Expanded Access to Investigational Drugs for Treatment Use rule, if manufacturers agree, seriously ill patients in emergencies or life-threatening situations can receive experimental medicines

  • on an individual case-by-case basis
  • as part of a medium-sized group with similar conditions without other treatment options
  • or, sometimes, as part of a large group once there is more information about safety and effectiveness from ongoing or completed clinical trials.

To obtain access as an individual:

  • The patient must have a serious or life-threatening illness for which there is no other treatment option available.
  • The patient’s doctor must apply for a single-patient Investigational New Drug (IND) for Compassionate or Emergency use with a brief clinical history and treatment plan.
  • The doctor must also obtain a statement from the drug’s manufacturer that the drug is available and will be supplied to the patient.  There is no FDA requirement that the manufacturer provide investigational medicines to individual patients.  Manufacturers can refuse or can charge for the drug.
  • An informed consent must be obtained from the patient showing that he or she understands the risks of the treatment and that it is experimental.
  • Approval from an Institutional Review Board (IRB) must be obtained by the sponsoring doctor before starting treatment.
  • Finally, the FDA needs to approve the individual IND.

Regulations are also in place to explain how a group of patients can get expanded access or a plan can be developed to provide access to a larger group of patients prior to FDA approval of the drug.

The Charging Rule allows a manufacturer to charge for an investigational drug:

  • in very limited numbers of clinical trials where only direct drug costs can be charged.
  • in most expanded access situations where both direct costs and costs of administering an expanded access program can be recovered.

Charging for Investigational Drugs Under an Investigational New Drug Application specifies what costs can be included.

American Society of Clinical Oncology President, Douglas Blayney, MD, commented,

ASCO is pleased that the FDA has issued new rules that strike a balance between meeting the immediate needs of seriously ill cancer patients today, while safeguarding the clinical trials system so it can develop new drugs to treat cancer patients in the future.

The new rules will make it clearer for doctors and patients to access experimental drugs when other treatment options have been exhausted and clinical trials are not available.

ASCO will be developing tools to ensure that oncologists understand the new rules and can help their patients benefit.

More information about access to experimental drugs and expanded access is available from CancerActionNow.Org, a project of the Marti Nelson Foundation.

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