The U.S. Food and Drug Administration (FDA) states its purpose in the following mission statement:
“The FDA is responsible for protecting the public health by assuring the safety, efficacy, and security of human and veterinary drugs, biological products, medical devices, our nation’s food supply, cosmetics, and products that emit radiation. The FDA is also responsible for advancing public health by helping to speed innovations that make medicines and foods more effective, safer, and more affordable; and helping the public get the accurate, science-based information they need to use medicines and foods to improve their health.”
The FDA provided a public draft guidance document to the pharmaceutical industry on the development of drugs to treat acne vulgaris or acne. The draft guidance document contains the FDA’s current thinking on the development of acne drugs and the methods that should be used.
Some guidance document information is summarized below. At the time of this writing, the FDA document had not been finalized.
Types of Acne Lesions
The two major types of acne lesions are classified as non-inflammatory and inflammatory. Non-inflammatory acne lesions are more commonly known as whiteheads and blackheads. Inflammatory acne lesions include papules and pustules and are more deeply seated in the skin than non-inflammatory lesions.
How Acne Severity Is Rated
Currently, there is no standardized method for rating the severity of acne outbreaks. Several methods have been proposed, but each has its difficulties. A sample scale for rating acne severity is summarized below, from least to most severe:
0 Clear skin with no inflammatory or non-inflammatory lesions
1 Almost clear; rare non-inflammatory lesions with no more than one small inflammatory lesion
2 Mild severity; greater than Grade 1; some non-inflammatory lesions with no more than a few inflammatory lesions (papules/pustules only, no nodular lesions)
3 Moderate severity; greater than Grade 2; up to many non-inflammatory lesions and may have some inflammatory lesions, but no more than one small nodular lesion
4* Severe; greater than Grade 3; up to many non-inflammatory and inflammatory lesions, but no more than a few nodular lesions
(* worsening beyond Grade 4 is possible).
Acne Drug Treatment Study Groups
Acne drug test groups should be large enough to be statistically significant so that any claims of success and safety can be supported. Test patients are usually recruited during their most severe acne breakouts.
Acne drug trials are recommended to be randomized, blinded, multi-center trials, and other requirements will also apply. A control or placebo group should be utilized.
Furthermore, those doing testing should recruit test populations that are representative of the age, race, gender, and geographic location of acne patients in the United States.
Determining The Success Of Acne Drug Treatments
Acne severity is rated at the beginning and end of every test. Photographic evidence and/or lesion counts may also be taken. It is proposed to measure the success of acne medications as either a success or a failure, based on one of these two methods of measuring success:
1. Success meaning a rating of clear skin or almost clear (severity rating 0 or 1) within the test period; or,
2. Success meaning an improvement of 2 severity grades within the test period. Under this definition, a successful test subject would have a severity improvement from 4 to 2 within the test period, as an example.
The Final Decision On New Acne Drugs
Several phases of testing are recommended for proposed acne drugs. Studies must be designed to account for effects such as test dropouts. Rigorous data analysis must be performed in conformance with FDA regulations. Only acne drug test data that has been validated and undergone quality assurance testing should be submitted to the FDA for approval.
Only through a rigorous and tightly-controlled testing and analysis procedure, followed by FDA approval, can new acne drugs be made available to the public.