US Orphan Drug Designation

Published by IRDiRC

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

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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Product specific- pre approval advisory committee

Published by IRDiRC

Advisory Commitee Approval Advisory Committee Early Access Support Regulatory Affairs Scientific Advice Technical Advice

Advisory committees (AC) provide independent advice and recommendations the US FDA on scientific and technical matters related to the development and evaluation of products regulated by the FDA

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FDA Expedited Program for serious conditions – Breakthrough Therapy Designation

Published by IRDiRC

Breakthrough Therapy Designation Early Access Support Expedited Programme Regulatory Advice Regulatory Affairs

Process designed to facilitate the development and expedite the review of drugs intended to treat a serious condition AND preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).

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FDA Expedited Program for serious conditions – Priority Review Designation

Published by IRDiRC

Accelerated Aproval Early Access Support Regulatory Advice Regulatory Affairs

FDA-AA is designed to support the development of drugs that treats serious or life- threatening conditions AND generally provides a meaningful advantage over available

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Published by IRDiRC

Biologics License Application Marketing Authorisation New Drug Application Priority Review Designation Priority Review Voucher Regulatory Advice Regulatory Affairs

A Priority Review designation results in an abbreviated PDUFA mandated goal date for a Biologics License Application (BLA) or a New Drug Application (NDA), from 10 months (under standard procedure) to 6 months.

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FDA Type C, A meetings

Published by IRDiRC

Early Access Support Product Development Programme Regulatory Affairs Scientific Advice Type A Meeting Type C Meeting

“Type A meetings are those that are necessary for an otherwise stalled product development program to proceed or to address an important Safety issue

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FDA Milestones meetings – Type B

Published by IRDiRC

Early Access Support End of Phase 2 Meeting Milestone Meeting Pre-NDA Meeting Regulatory Advice Regulatory Affairs Scientific Advice

The default drug development program includes three milestones, or Type B meetings. The earliest is the Pre-IND Meeting, the second is the End of Phase 2 (EOP2) Meeting, and the third milestone is the Pre-NDA Meeting.

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Pre Investigational New Drug (IND) interactions

Published by IRDiRC

Early Access Support Milestone Meeting Pre Investigational New Drug Interaction Pre-NDA Meeting Regulatory Affairs Scientific Advice

A pre-IND meeting is an FDA milestone meeting also known as Type B meeting . In these meetings the drug sponsor seeks FDA feedback about the quality, non- clinical and clinical development plan. The Pre-IND meetings are not mandatory.

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Regenerative Medicine Advanced Therapy (RMAT) Designation

Published by IRDiRC

Advanced Therapy Early Access Support Regenerative Medicine Advanced Therapy Designation Regulatory Affairs

The RMAT Designation is analogous to the Breakthrough Designation designed for traditional drug candidates but applies to regenerative medicine treatments, and allows companies to interact with the FDA more frequently during the clinical development of the therapy. An RMAT-designated therapy is eligible for priority review and accelerated approval.

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Neglected and Tropical Diseases Priority Review Voucher

Published by IRDiRC

Early Access Support Neglected and Tropical Disease Priority Review Voucher Regulatory Advice Regulatory Affairs Scientific Advice

This program grants a priority review voucher (PRV) from the US Food and Drug Administration (FDA) to sponsors of certain tropical disease product application

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The Innovation Management Toolbox was originally developed through funding from the EJP RD project, which received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No. 825575. The continued development and maintenance of the Toolbox are now supported by the ERDERA partnership, funded under the European Union’s Horizon Europe Research & Innovation programme, grant agreement No. 101156595.

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