Ways to Speed Treatments to Patients : Part 3

by Food and Drug Administration (FDA)

(Page 3 of 3)

FDA and sponsors of priority drugs may meet at the earliest stages of clinical testing to plan studies that will help develop the information necessary for a final decision on a product's approvability. Then, when a marketing application is submitted, FDA can mobilize available personnel and other resources needed to review the often large amounts of technical information contained in a priority new drug application.

In another effort to speed the review of marketing applications, the review process is becoming increasingly computerized. New drug applications that commonly run to thousands of pages are now arriving from sponsors in a form suitable for computer processing. This makes review and communication with the sponsor more efficient, saving time for both FDA and the firm.

Accelerated Approval

A highly specialized mechanism for speeding the approval of drugs or biologics that promise significant benefit over existing therapy for serious or life-threatening illnesses — so-called accelerated approval — incorporates several novel elements aimed at making sure that rapid review and approval is balanced by safeguards to protect both the public health and the integrity of the regulatory process itself.

Accelerated review, established by 1991 regulations, can be used in two very special circumstances: when approval is based on evidence of the product's effect on a "surrogate endpoint," and when FDA determines that safe use of a product depends on restricting its distribution or use.

A "surrogate endpoint" is a laboratory finding or physical sign that may not, in itself, be a direct measurement of how a patient feels, functions or survives, but nevertheless is considered likely to predict therapeutic benefit. For example, high blood pressure and elevated serum cholesterol are risk factors for heart and blood vessel disease. Drugs that control blood pressure or cholesterol can reasonably be expected to help control or prevent direct signs of disease, such as angina, congestive heart failure after a heart attack, paralysis following a stroke, and sudden death. Once a drug has been shown effective as measured against such a surrogate endpoint, FDA can grant marketing approval.

As a condition of approval, however, FDA can require the sponsor to carry out post-marketing studies to confirm that the drug does in fact produce a clinical benefit, such as increased survival time. And if further research or experience shows that a product that received accelerated approval cannot safely remain on the market, FDA can order its prompt withdrawal.

As a further safeguard, distribution of accelerated-approval drugs can be limited to institutions that have the capability to use them safely and to physicians with specialized training or experience. The agency can also require that specific medical procedures, such as blood tests, be carried out if they are deemed essential for safe and effective use of the product.

In the summer of 1994, some health professionals and consumers active in the fight against AIDS began expressing concern that drugs in accelerated approval and expanded access programs (including parallel track and Treatment IND protocols) may be made available with insufficient details about side effects and effectiveness.

FDA convened its Antiviral Drugs Advisory Committee on Sept. 12-13, 1994, as part of a continuing dialogue about expanded access to new HIV drugs.

Based in part on public testimony and committee recommendations, FDA's anti-viral drug division is expected to issue a guidance document for sponsors of AIDS drugs applying for expanded access or accelerated approval status.

The agency has reaffirmed its commitment to these ways to make new drugs available for people with serious and life-threatening diseases. Working with its advisory committees and other outside experts, FDA will continue to consider improvements to these processes, and implement them where appropriate.

It is clearly too soon to know whether efforts to make drugs and biologics more rapidly and widely available to the desperately ill are contributing to genuine advances in health care. But many thousands of patients who might otherwise be beyond hope are now able to seek help from investigational agents, and all of us stand to gain from a more efficient, more responsive system by which to bring important new agents to market.

About the Author

www.fda.gov
FDA is A United States government body that oversees medical devices, including contact lenses, intraocular lenses, excimer lasers and eyedrops. In the US, these products must be approved by the FDA before they can be marketed.