Essay Example on FDA issued a draft guidance titled Clarification of Orphan Designation of Drugs and Biologics

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In December 2017 the FDA issued a draft guidance titled Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases This draft guidance pertains to the Orphan Drug Act was created by Congress in 1983 This act was authorized to encourage pharmaceutical companies to develop drugs for diseases that affect a small percentage of the population Koperniak 2017 To encourage companies to develop orphan drugs the Orphan Drug Act of 1983 included provisions designed to offset the cost of producing orphan drugs In order to qualify for Orphan Drug status a drug must be for a disease or condition with an overall prevalence of less than 200 000 people Historically the FDA allowed for drugs to receive the orphan drug designation for pediatric sub populations if the prevalence in pediatric patients was below 200 000 even if the disease is fairly common in adult populations U S Department 2017 This is became known as the pediatric subpopulation designation The FDA guidance issued in December 2017 uses the example of ulcerative colitis Ulcerative colitis is prevalent in adults however is drugs to treat ulcerative colitis have been granted pediatric subpopulation designation because in children the disease affects less than 200 000 patients The pediatric sub population designation was created to encourage the study of drugs in the pediatric population as typical clinical testing only involves adult populations Several programs have been created since the 



Orphan Drug Act that provide incentives for pediatric studies which decrease the need for the pediatric subpopulation designation The Food and Drug Modernization Act created in 1997 included a pediatric exclusivity provision giving companies an additional 6 months of market exclusivity when a sponsor submits proof of properly conducted pediatric studies U S Department 2017 This provision was reauthorized under the Best Pharmaceuticals for Children Act BPCA which was created in 2002 The Pediatric Research Equity Act PREA was created in 2003 and required marketing applications for drugs to be used in a pediatric subpopulation Manthei et al 2017 PREA required that the new marketing applications include information on any new indications dosage forms active ingredients or routes of administration in addition to safety and effectiveness making it necessary to provide evidence of pediatric research studies This was not the case with the Pediatric Subpopulation designation If a drug is granted the pediatric subpopulation designation it will be exempt from the requirements set forth in PREA Companies who have received the pediatric subpopulation designation for their drugs would also receive the incentives provided by the Orphan Drug Act should they decide to conduct pediatric studies One downside of the pediatric sub population designation is that there are no procedures in place for the FDA to mandate that these studies occur The FDA Guidance Clarification of Orphan Designation of Drugs and Biologics for

Pediatric Subpopulations of Common Diseases issued in December 2017 details the FDA s intent to no longer grant the pediatric subpopulation designation for the majority of drugs thus eliminating a loophole pharmaceutical companies have used for years to market drugs for pediatric patients without conducting pediatric research If this guidance ultimately is finalized there is the possibility for pediatric research to increase across many diseases Research on pediatric diseases is vital since children may react differently than adults to a drug Clinical studies in pediatric patients are important to identify the best dose of a drug to be used in children While drugs tested through adult clinical trials have a known dose that is safe and effective for the adult population there is no way to determine how children will tolerate this dose without pediatric studies The pharmacokinetics of a drug in children may be entirely different than that of an adult because they are still growing and developing Conducting pediatric clinical testing allows researchers to look at the absorption metabolism distribution and elimination of a drug in children These studies may also help characterize the pediatric adverse event and toxicity profile which is beneficial for physicians who will treat the patients with this drug Clinical testing in pediatric patients is also important to see if a more child friendly formulation of the drug is available Liquid or chewable formulations make it easier for pediatric patients to take the drug and additional clinical testing would be required in accordance with the requirements outlined in PREA The overall increase in pediatric research that will come as a result of the removal of the pediatric subpopulation designation will benefit pediatric healthcare as a whole Requiring sponsors to conduct clinical studies in pediatric patients will help to advance the overall knowledge of how a child's body works 



While the guidance is being finalized the FDA will not issue final decisions on requests for pediatric subpopulation designation I believe this is an important step to take and I am glad the FDA included it in the guidance This may prevent an influx of applications submitted for pediatric subpopulation designation status The Draft guidance also includes language to outline the scenarios in the future where drugs could qualify for pediatric sub population status I do believe changing the requirements surrounding the pediatric subpopulation designation may cause anger among pharmaceutical companies who rely on the designation to avoid pediatric studies while marketing their drug Overall I think the draft guidance will have a positive impact on the drug development industry and pediatric advocacy groups should be excited about the increase in pediatric research that may occur Additionally requiring pediatric clinical studies will help improve the safety of drugs available to children


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