An MBA graduate from the Harvard Business School, Raj Prabhakar is a Chief Business Officer and Head of Business Operations and Corporate Strategy at Rocket Pharmaceuticals in New York City, United States. One of the areas that Raj Prabhakar is actively involved in is preclinical and clinical development-stage operations, including Rare Pediatric Disease Designated products (RPDD).
A rare pediatric disease is a life-threatening and severe condition that affects people up to 18 years from birth and primarily manifests in newborns, neonates, infants, and adolescents. However, in order for the RPDD designation to apply, it must be rare and documented in less than 200,000 prevalence in the United States.
The RPDD attends to pediatric patients with unmet need requirements and helps stimulate research and development of new medications for RPDD through financial and tax incentives to obtain approval from the Food and Drugs Administration (FDA). The criteria to meet the RPDD includes the drug intended for someone who requires prevention or treatment of a rare pediatric disease. Also, verifiable documentation and supportive data are necessary to ensure that the intended purpose is rare and that the drug is effective.
The Food and Drug Administration Safety and Administration Innovation Act added the section in 2012 with options for priority review vouchers. To access this, however, the sponsor must request the Priority Review Voucher (PRV) in the original Non Disclosure Agreement or Biologics License Applications process.
One of the primary benefits of the RPDD is the ability of a sponsor’s eligibility to receive more benefits through an incentive program. Section 529 of the FD&C Act allows the Food and Drugs Act to award a PRV after approval for the RPDD products.
A rare pediatric disease is a life-threatening and severe condition that affects people up to 18 years from birth and primarily manifests in newborns, neonates, infants, and adolescents. However, in order for the RPDD designation to apply, it must be rare and documented in less than 200,000 prevalence in the United States.
The RPDD attends to pediatric patients with unmet need requirements and helps stimulate research and development of new medications for RPDD through financial and tax incentives to obtain approval from the Food and Drugs Administration (FDA). The criteria to meet the RPDD includes the drug intended for someone who requires prevention or treatment of a rare pediatric disease. Also, verifiable documentation and supportive data are necessary to ensure that the intended purpose is rare and that the drug is effective.
The Food and Drug Administration Safety and Administration Innovation Act added the section in 2012 with options for priority review vouchers. To access this, however, the sponsor must request the Priority Review Voucher (PRV) in the original Non Disclosure Agreement or Biologics License Applications process.
One of the primary benefits of the RPDD is the ability of a sponsor’s eligibility to receive more benefits through an incentive program. Section 529 of the FD&C Act allows the Food and Drugs Act to award a PRV after approval for the RPDD products.
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