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Current February 2008

Speeding Access to Important Therapeutic Agents

Shortening Time to Approval

Fast Track, Accelerated Approval, and Priority Review. A brief overview of the major three regulatory approaches to expediting drug approval.

The FDA Speeds Medical Treatments for Serious Diseases. An FDA Consumer Magazine article about three distinct approaches developed by the Food and Drug Administration intended to make important treatments available more rapidly to those who desperately need them: "fast track" designation of drugs, "accelerated approval," and "priority review."

Critical Path. The Critical Path Initiative is FDA's effort to stimulate and facilitate a national effort to modernize the scientific process through which a potential human drug, biological product, or medical device is transformed from a discovery or "proof of concept" into a medical product.

Use of Investigational Agents Prior to Marketing Approval

Access Outside of Clinical Trials

FDA allows access to investigational drugs, in certain circumstances, for patients with serious or immediately life-threatening diseases or conditions who lack other therapeutic options and may benefit from such therapies. It is not always possible for all patients who want access to investigational drugs to enroll in clinical trials. Patients may not meet eligibility criteria or may be geographically isolated from a study site. In these situations, FDA believes that it is appropriate to help make certain promising, but as yet unproven, products available outside of a clinical trial (non-protocol) to patients with serious and life-threatening illnesses. Non-protocol investigational therapy should be offered in a way that does not pose an unreasonable risk to the patient or an unreasonable risk of losing valuable information about the effect of the drug. For these reasons, although treatment is focused on the individual patient, a study plan (protocol) may be written to ensure that the treatment is administered appropriately and that patients are monitored for toxicity. It is important to note that a pharmaceutical manufacturer must first agree to provide the requested product for a non-protocol investigational therapy to begin. FDA cannot mandate that the requested products be supplied to these programs; the agency can only review and approve proposals to use them.

In an effort to enable more patients who lack satisfactory alternatives to have access to unapproved medicines, while balancing the need to safeguard the individual patient and ensure the continued integrity of the scientific process that brings safe and effective drugs to the market, on December 11, 2006, FDA proposed significant regulatory changes to make investigational drugs more widely and easily available to seriously ill patients, including those with HIV/AIDS, who have no other treatment options. The proposed changes also seek to clarify the specific circumstances and the types of costs for which a manufacturer can charge for an investigational drug made available for the purpose of treatment.

FDA would like to increase awareness in the healthcare community of the range of options available for obtaining investigational drugs for seriously ill patients, encourage companies to make such drugs available, and reduce barriers to obtaining them.

The proposed rule defines three categories of patients to whom investigational drugs could be made available for the purpose treatment outside of a clinical trial through expanded access, when there is no satisfactory alternative therapy, and defines requirements and safeguards for each. They are

FDA has allowed many types of access to investigational therapies since the 1970's. Some of the larger programs, including those under the treatment IND (Investigational New Drug) regulations, enabled tens of thousands of patients with HIV/AIDS, cancer and cardiovascular diseases to receive promising therapies before the products were approved for marketing. However, the existing regulations do not adequately describe the full range of programs available, explicitly recognizing only emergency use for individual patients and widespread treatment use access for large groups of patients.  The proposed changes are meant to clearly reflect the full range of treatment use programs available, and ensure broad and equitable access to investigational drugs for treatment use.

The current regulations describing when it is appropriate to charge for an investigational drug need revisions because they fail to account for the full range of circumstances in which charging should be permissible and because they have proven difficult to interpret in practice, resulting in confusion over what costs could be recovered by sponsors making drug products available through expanded access programs.

The proposed rules are described in detail at www.fda.gov/cder/regulatory/applications/IND_PR.htm.

The most significant proposed changes would:

(1) Modernize applicable regulations to include all circumstances under which access to investigational drugs is permitted, including:

(2) Make investigational drugs more widely available in appropriate situations by establishing criteria that link the level of evidence needed to support the use of an investigational drug to the seriousness of the disease and the number of patients likely to be treated with the drug in an expanded access program;

(3) Revise the current regulation regarding manufacturers' recovery of the costs of an investigational drug to:

The proposal also would simplify the cost recovery calculation by making clear that charges for an investigational drug used in a clinical trial may include only direct costs associated with the drug's development, and that charges for investigational drugs for treatment use may also include administrative costs of making the drug available for intermediate patient populations and under large scale treatment INDs.

HIV/AIDS Specific Resources

Expanded Access and Expedited Approval of New Therapies Related to HIV/AIDS

October 16, 2000 Antiviral Drugs Advisory Committee Meeting

The committee discussed the use of surrogate markers in early development of immunomodulatory agents for the treatment of patients with HIV

July 14, 1998 Antiviral Drugs Advisory Committee Meeting--Transcript (PDF- 4 MB)

The committee discussed regulatory procedures and policies, including an overview of the Food and Drug Administration Modernization Act of 1997 and efforts to expedite drug development.

Cancer Specific Resources

Accelerated Approval

Summary description of how the FDA would apply the accelerated approval rule to new cancer treatments (October 2005 Memorandum to Oncologic Drugs Advisory Committee)

March 8, 2005 Oncologic Drugs Advisory Committee Meeting

The committee discussed accelerated approval of drugs to treat cancer. The meeting was intended to 1) Review the status of phase IV clinical studies; (2) identify difficulties and (3) provide advice to sponsors to assist in the planning and execution of postmarketing commitments of newly approved drugs associated with completion of phase IV commitments.

March 12/13, 2003 Oncologic Drugs Advisory Committee Meeting

The committee discussed accelerated approval of cancer therapies

Expedited Drug Development

Guidance for Industry: IND Exemptions for Studies of Lawfully Marketed Drug or Biological Products for the Treatment of Cancer

Expanded Access

Report to Congress: Patient Access to New Therapeutic Agents for Pediatric Cancer (December 2003)

June 7/8, 2001 Oncologic Drugs Advisory Committee Meeting

The committee discussed single patient use of non-approved oncology drugs and biologics

PDF documents may be read with a free copy of the Adobe Acrobat Reader.

Microsoft Word files can be viewed with that application or with a document viewer available free from the Microsoft Web Site.

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