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U.S. Department of Health and Human Services

For Consumers

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Speeding Access to Important New Therapies

Shortening Time to Approval

Drugs: Speeding the development and availability of drugs that treat serious diseases are in everyone's interest, especially when the drugs are the first available treatment or have advantages over existing treatments. The Food and Drug Administration (FDA) has developed four distinct and successful approaches to making such drugs available as rapidly as possible: Priority Review, Breakthrough Therapy, Accelerated Approval, and Fast Track. Because each of these approaches implies speed, there can be confusion about the specific meaning of each and the distinctions among them.

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

Breakthrough Therapies. The Food and Drug Administration Safety and Innovation Act (FDASIA) includes a provision that allows sponsors to request that their drug be designated as a Breakthrough Therapy.  FDA is in the process of developing guidance related to this designation.

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.

Medical Devices: A program for certain medical devices that demonstrate the potential to address unmet medical needs for life threatening or irreversibly debilitating diseases or conditions and are subject to premarket approval (PMA) applications is being proposed by FDA as well.  FDA believes that the "Expedited Access PMA for Unmet Medical Needs for Life Threatening or Irreversibly Debilitating Diseases or Conditions" ("Expedited Access PMA" or "EAP") program will help patients have more timely access to these medical devices by expediting their development, assessment and review, while preserving the statutory standard of reasonable assurance of safety and effectiveness for premarket approval,1 consistent with the Agency’s statutory mission to protect and promote public health.

To be eligible for participation in the program, the medical device must:
• Be intended to treat or diagnose a life-threatening or irreversibly-debilitating disease or condition
• Represent one of the following:
     1. no approved alternative treatment/diagnostic exists, or
     2. a breakthrough technology that provides a clinically meaningful advantage over existing technology, or
     3. offers a significant, clinically meaningful advantage over existing approved alternatives, or
     4. availability is in the patient’s best interest 
• Have an acceptable data development plan that has been approved by the FDA

The EAP builds on the Innovation Pathway pilot, which the FDA launched in 2011, and the FDA’s experience with expedited review programs for pharmaceuticals, including Accelerated Approval and Breakthrough Therapies. When utilizing the EAP program, the FDA will continue to apply the current approval standard of demonstrating a reasonable assurance of safety and efficacy.

In addition to the Expedited Access Program, the FDA published a separate draft guidance that outlines the agency’s current policy on when data can be collected after product approval and what actions are available to the FDA if approval conditions, such as postmarket data collection, are not met. Included in the guidance is advice on the use of surrogate or independent markers to support approval, similar to the data points used for accelerated approval of prescription drugs. 

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 (treatment use of an investigational drug) to patients with serious and life-threatening illnesses. Treatment use of an investigational drug 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 treatment use of an investigational drug to begin. FDA cannot mandate that the requested products be supplied to these programs; the agency can only review and authorize proposals to use them.

Questions and Answers about treatment of serious and life threatening illnesses, and use of investigational drugs

Rules for Expanded Access to Investigational Drugs for Treatment Use and Charging for Investigational Drugs