Exclusivity pediatric drugs

The success of the various special-case areas of drug development regulation and performance, such as orphan drugs, cancer, and AIDS drugs, and the pediatric exclusivity extension could be a guide to how to approach - and what to avoid -in creating larger facilitatory approaches in the future. 

A written request is a formal communication from the FDA to the sponsor of a drug product that specifies in detail what pediatric trials are needed for a particular pediatric indication(s) to gain additional exclusivity under the FDAMA. As of March, 2002, the FDA has... 

Health Research Group (HRG). 2001. Pediatric Exclusivity Changes Needed to Assure Safety and Effectiveness of Medications for Children and More Affordable Drugs for Seniors. Washington, DC Public Citizen. 

C) MARKET EXCLUSIVITY FOR ALREADY-MARKETED DRUGS. If the Secretary determines that information relating to the use of an approved drug in the pediatric population may produce health benefits in that population and makes a written request to the holder of an approved application under section 505(b)(1) for pediatric studies (which shall include a timeframe for completing such studies), the holder agrees to the request, the studies are completed within any such timeframe, and the reports... 

Another issue facing clinical studies is the inclusion of children. The FDA Modernization Act of 1997 addresses the desirability of pediatric research before new drug approval when a drug is likely to be prescribed for children. The reward for such research is a 6-month prolongation of market exclusivity or patent life. 

Cooper KJ. Pediatric marketing exclusivity — as altered by the Best Pharmaceuticals for Children Act of 2002. Food Drug Faw J 2002 57 519h14. 

The FDA provides notice of the innovator s FDA and patent exclusivity by publishing information regarding exclusivity in the FDA Approved Drugs Product List (the Orange Book ). The Patent and Exclusivity Appendix to the list of approved products provides details regarding the expiry dates of all types of FDA exclusivity (including pediatric exclusivity) and the expiry dates of patents for each approved product. 

For drugs that are new molecular entities (NMEs), a determination should be made by the sponsor of the potential usefulness of the new drug in a pediatric population. If it is likely to generate over 100 000 prescriptions per year, this would indicate the need to develop a pediatric formulation and suitable pediatric studies. If it is likely to generate less than 50 000 prescriptions per year, the sponsor may be granted a waiver by the FDA for pediatric data, and a disclaimer statement allowed. Either way, in a children s disease, if less than 200 000 patients per year may benefit, then orphan-drug status with 7 per year exclusivity may be applicable. This would then apply only to that pediatric indication. 

Meanwhile in Europe similar efforts are underway with the EU issuing the European Draft Document for Pediatric Regulation. It proposes 10-year exclusivity for off patent drugs if required pediatric studies are done, and six months for patented medicines . This draft is currently under consideration and comment by members states before the EU Commission signs a final regulation. 

The FDA Modernization Act (FDAMA) of 1997 contains within it financial incentives for the development and marketing of drugs that could be used for pediatric patients. Some of these incentives include an extension of 6 months on market exclusivity and waiving fees for supplemental applications needed for receiving the approval of drugs for pediatric use that are already approved for adult use. In addition, the FDA published a list of drugs approved in adults for which additional pediatric data may produce health benefits for pediatric... 

---