NCATS Toolkit for Patients-Focused Therapy Development

Published by IRDiRC

Research and Drug Development Rare Disease Toolkit for Patient NCATS Toolkit Patient-Focus Therapy Development

The NCATS Toolkit for Patient-Focused Therapy Development (Toolkit) was developed through a collaboration between NCATS and rare diseases patient groups. The patient groups had expressed an interest in exchanging knowledge and sharing best practices in therapy development

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Rare Pediatric Priority Review Voucher

Published by IRDiRC

Research and Drug Development Regulatory Affairs Paediatric Medicine Marketing Authorisation Orphan Medicine Regulatory Advice Rare Paediatric Priority Voucher

This program grants a voucher for priority review from the US Food and Drug Administration (FDA), which aim to render a decision within 6 months (in contrast to 10 for a standard review). The developed drug for which the voucher is awarded must be intended for a rare disease or condition and that primarily affect individuals from 0 to 18 years. The voucher can be utilized for any other drug development program.

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FDA Pediatrics Advisory Committee

Published by IRDiRC

Regulatory Affairs Early Access Support Paediatric Medicine Paediatric Regulation Regulatory Advice Paedriatic Advisory Committee Paediatric Research Biomedical Ethics

The Pediatric Advisory Committee (PAC) is a committee of authorities knowledgeable in pediatric research, pediatric subspecialties, statistics, and/or biomedical ethics who advise and make recommendations to the FDA

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Stakeholders interactions – Patients Affairs Staff

Published by IRDiRC

Research and Drug Development Regulatory Affairs Clinical Development Early Access Support Patient Engagement Patient Advice FDA Regulatory Advice Patient Affairs Staff Patient Support

At the FDA, the Patient Affairs Staff (PAS) is devoted to supporting cross-cutting patient engagement activities across the FDA. Its mission is to coordinate and support patient engagement activities across FDA to facilitate awareness and collaboration with patients, their advocates and the FDA

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Patient-focused drug development program

Published by IRDiRC

Regulatory Affairs Early Access Support Patient Advice Patient Support Regulatory Advice Patient Focused Drug Development Programme

The Patient-focused Drug Development (PFDD) program was initiated at FDA from 2012- 2017 under Congressional Mandate (PDUFA V) to more systematically obtain the patient perspective on specific diseases and their treatments. PFDD meetings provide key stakeholders an opportunity to hear the patient’s voice.

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US Orphan Drug Designation

Published by IRDiRC

Regulatory Affairs Early Access Support Orphan Drug Designation Regulatory Advice Orphan Drug Incentive

The ODA is intended to provide incentives for Orphan Drug development and to defray the costs of qualified clinical testing expenses incurred in connection with the development of drugs for rare diseases and conditions.

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Rare Pediatric Disease Designation

Published by IRDiRC

Regulatory Affairs Early Access Support Paediatric Medicine Orphan Drug Designation Rare Paediatric Disease Designation

The FDA grants rare pediatric disease designation for diseases with serious or life- threatening conditions that affect people from birth to 18 years of age, that affect fewer than 200,000 people in the U.S.

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Right to Try Act

Published by IRDiRC

Regulatory Affairs Early Access Support Orphan Medicine Regulatory Right to Try Act Life-Threatening Disease

This law is another way for patients who have been diagnosed with life-threatening diseases or conditions who have tried all approved treatment options and who are unable to participate in a clinical trial to access certain unapproved treatments.

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US Expanded Access Program

Published by IRDiRC

Research and Drug Development Regulatory Affairs Orphan Drug Early Access Support Orphan Medicine Expanded Accesss Programme

The Program allows patients with serious rare diseases to have access to pre-market drugs and devices, if the Sponsor agrees to this under their expanded access policy

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Single Patient Expanded Access

Published by IRDiRC

Research and Drug Development Regulatory Affairs Orphan Drug Early Access Support Orphan Medicine Single Patient Expanded Accesss

This FDA program allows early access to drugs and medical devices before marketing approval, under regulatory oversight with collection of Safety data, to individual patients with serious or immediately life-threatening diseases or conditions who lack therapeutic alternatives. This includes emergency and non-emergency expanded access.

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