News from the FDA

http://a257.g.akamaitech.net/7/257/2422/14mar20010800/edocket.access.gpo.gov/2003/03-25306.htm.

Under the CMA pilot program, Pilot 2, certain drug and biologic products that have been designated as Fast Track are eligible to be considered for inclusion in this pilot program, which will provide for frequent scientific feedback and interactions between the FDA and applicants during the investigational phase of development. Applicants are being asked to apply to participate in the Pilot 2 program. A maximum of one Fast Track product per review division in CDER and CBER will be selected to participate. For further information, go to: http://a257.g.akamaitech.net/7/257/2422/14mar20010800/edocket.access.gpo.gov/2003/03-25305.htm.

Monitoring Minorities in Pediatric Trials

In a General Accounting Office (GAO) report issued this month, the agency called on the FDA to consistently require drug makers to report on the ethnicity of children included in pediatric clinical trials of drugs intended to treat conditions that disproportionately affect members of minority groups. The FDA is authorized under the pediatric exclusivity provision to grant drug sponsors 6 months of additional exclusive marketing rights for conducting clinical drug studies in children, but according to the terms of the Best Pharmaceuticals For Children Act of 2002, in order to authorize such extended exclusivity, the FDA must take into account the adequacy of minority representation in such studies. The GAO found, however, that overall, 14% of pediatric clinical trial participants were not identified to the FDA by race. Absent full reporting of the race of trial subjects, drug makers could jeopardize their rights to extended marketing exclusivity.

Counter-Terrorism Products Workshop

The FDA plans to hold a public workshop titled “Counter Terrorism Products Regulated by the Center for Biologics Evaluation and Research: Effective Strategies to Assist in Product Development.” The workshop, which will be held October 23 in Bethesda, MD, will provide a forum for discussing strategies to assist in the effective development of products regulated by the Center for Biologics Evaluation and Research (CBER) that may be used in counter terrorism efforts (eg. vaccines, blood and blood products including immunoglobulins, gene therapies, and human cellular and tissue-based products).

For further information, go to: http://a257.g.akamaitech.net/7/257/2422/14mar20010800/edocket.access.gpo.gov/2003/03-24303.htm.

Reporting Post-approval Safety Data

A draft guidance intended to bring international harmonization to the collection and management of post-approval product safety data, titled “E2D Postapproval Safety Data Management: Definitions and Standards for Expedited Reporting,” is now available from the FDA. The draft guidance was prepared under the auspices of the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH), and provides definitions associated with postapproval product safety information and standards for collecting and expedited reporting of safety information to the regulatory authorities.

For further information, see: http://a257.g.akamaitech.net/7/257/2422/14mar20010800/edocket.access.gpo.gov/2003/03-23508.htm.

Guidance on INDA Exemptions for Studies of Cancer Treatment Products

A recently published a guidance clarifying the FDA's policy on exemption from investigational new drug application (IND) requirements for studies of marketed cancer drug or biological products is now available. This guidance, “IND Exemptions for Studies of Lawfully Marketed Drug or Biological Products for the Treatment of Cancer,” is intended to decrease the submission of unnecessary IND exemptions. To obtain a copy, go to www.fda.gov/cder/guidance/index.htm. For further information, see: http://a257.g.akamaitech.net/7/257/2422/14mar20010800/edocket.access.gpo.gov/2003/03-23510.htm.

Guidance on Premarket Approval Applications

A guidance titled “Guidance for Industry and FDA Staff: FDA and Industry Actions on Premarket Approval Applications: Effect on FDA Review Clock and Performance Assessment” is now available. The guidance describes premarket review cycle and decision actions and goals for original Premarket Approval Applications (PMAs), original expedited PMAs, panel-track supplements and 180-day PMA supplements. Because of tight statutory deadlines, it was not feasible for the FDA to obtain comments before issuing the guidance, but in accordance with the agency's good guidance practices procedures, the FDA will accept comments on the guidance at any time.

For further information, go to:

http://a257.g.akamaitech.net/7/257/2422/14mar20010800/edocket.access.gpo.gov/2003/03-25447.htm.

Pilot Programs for Fast-Track Drugs

Two related guidances for industry have just been published as part of a series of guidance documents that the FDA agreed to draft and implement in conjunction with the June 2002 reauthorization of the Prescription Drug User Fee Act of 1992 (PDUFA). The publications are: “Continuous Marketing Applications: Pilot 1 – Reviewable Units for Fast Track Products Under PDUFA” and “Continuous Marketing Applications: Pilot 2 – Scientific Feedback and Interactions During Development of Fast Track Products under the Prescription Drug User Fee Act of 1992.”

Under the CMA pilot program, Pilot 1, applicants submitting new drug applications or biological licensing applications for products that have been designated as Fast Track drug or biological products (ie, products intended to treat a serious and/or life-threatening disease for which there is an unmet medical need) may be eligible to submit portions of their marketing applications (reviewable units) in advance of the complete marketing application. The FDA has agreed to complete reviews of reviewable units within a specified time and to provide early feedback for those pre-submissions in the form of discipline review letters. Pilot 1 is an exploratory program that will allow the FDA to evaluate the added value, costs and impact of early review and feedback on parts of applications (reviewable units) in advance of submission of the complete application. For more information, go to:

http://a257.g.akamaitech.net/7/257/2422/14mar20010800/edocket.access.gpo.gov/2003/03-25306.htm.

Under the CMA pilot program, Pilot 2, certain drug and biologic products that have been designated as Fast Track are eligible to be considered for inclusion in this pilot program, which will provide for frequent scientific feedback and interactions between the FDA and applicants during the investigational phase of development. Applicants are being asked to apply to participate in the Pilot 2 program. A maximum of one Fast Track product per review division in CDER and CBER will be selected to participate. For further information, go to: http://a257.g.akamaitech.net/7/257/2422/14mar20010800/edocket.access.gpo.gov/2003/03-25305.htm.

Monitoring Minorities in Pediatric Trials

In a General Accounting Office (GAO) report issued this month, the agency called on the FDA to consistently require drug makers to report on the ethnicity of children included in pediatric clinical trials of drugs intended to treat conditions that disproportionately affect members of minority groups. The FDA is authorized under the pediatric exclusivity provision to grant drug sponsors 6 months of additional exclusive marketing rights for conducting clinical drug studies in children, but according to the terms of the Best Pharmaceuticals For Children Act of 2002, in order to authorize such extended exclusivity, the FDA must take into account the adequacy of minority representation in such studies. The GAO found, however, that overall, 14% of pediatric clinical trial participants were not identified to the FDA by race. Absent full reporting of the race of trial subjects, drug makers could jeopardize their rights to extended marketing exclusivity.

Counter-Terrorism Products Workshop

The FDA plans to hold a public workshop titled “Counter Terrorism Products Regulated by the Center for Biologics Evaluation and Research: Effective Strategies to Assist in Product Development.” The workshop, which will be held October 23 in Bethesda, MD, will provide a forum for discussing strategies to assist in the effective development of products regulated by the Center for Biologics Evaluation and Research (CBER) that may be used in counter terrorism efforts (eg. vaccines, blood and blood products including immunoglobulins, gene therapies, and human cellular and tissue-based products).

For further information, go to: http://a257.g.akamaitech.net/7/257/2422/14mar20010800/edocket.access.gpo.gov/2003/03-24303.htm.

Reporting Post-approval Safety Data

A draft guidance intended to bring international harmonization to the collection and management of post-approval product safety data, titled “E2D Postapproval Safety Data Management: Definitions and Standards for Expedited Reporting,” is now available from the FDA. The draft guidance was prepared under the auspices of the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH), and provides definitions associated with postapproval product safety information and standards for collecting and expedited reporting of safety information to the regulatory authorities.

For further information, see: http://a257.g.akamaitech.net/7/257/2422/14mar20010800/edocket.access.gpo.gov/2003/03-23508.htm.

Guidance on INDA Exemptions for Studies of Cancer Treatment Products

A recently published a guidance clarifying the FDA's policy on exemption from investigational new drug application (IND) requirements for studies of marketed cancer drug or biological products is now available. This guidance, “IND Exemptions for Studies of Lawfully Marketed Drug or Biological Products for the Treatment of Cancer,” is intended to decrease the submission of unnecessary IND exemptions. To obtain a copy, go to www.fda.gov/cder/guidance/index.htm. For further information, see: http://a257.g.akamaitech.net/7/257/2422/14mar20010800/edocket.access.gpo.gov/2003/03-23510.htm.

Guidance on Premarket Approval Applications

A guidance titled “Guidance for Industry and FDA Staff: FDA and Industry Actions on Premarket Approval Applications: Effect on FDA Review Clock and Performance Assessment” is now available. The guidance describes premarket review cycle and decision actions and goals for original Premarket Approval Applications (PMAs), original expedited PMAs, panel-track supplements and 180-day PMA supplements. Because of tight statutory deadlines, it was not feasible for the FDA to obtain comments before issuing the guidance, but in accordance with the agency's good guidance practices procedures, the FDA will accept comments on the guidance at any time.

For further information, go to:

http://a257.g.akamaitech.net/7/257/2422/14mar20010800/edocket.access.gpo.gov/2003/03-25447.htm.