Designated Intractable Diseases (Designated Nan-byo)

Published by IRDiRC

Act on Medical Care Early Access Support MHLW or PMDA Regulatory Nan-byo Designation Rare Disease Regulatory Affairs

Definition of designated Nan-byo is provided in the law named “Act on Medical Care for Patients with Intractable/Rare Diseases”. Patients with designated Nan-byo can receive financial support for medical treatment

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EMA Scientific guidelines

Published by

Clinical Development Clinical Trial Design Guideline Regulatory Advice Regulatory Affairs

The European Medicines Agency’s Committee for Medicinal Products for Human Use prepares scientific guidelines in consultation with regulatory authorities in the European Union (EU) Member States, to help applicants prepare marketing authorisation applications for human medicines. Guidelines reflect a harmonised approach of the EU Member States and the Agency on how to interpret and apply the requirements for the demonstration of quality, safety and efficacy set out in the Community directives.

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European Paediatric Translational Research Infrastructure (EPTRI)

Published by EPTRI

Drug Development paediatric Regulatory Affairs research infrastructure Translational Research

The EPTRI Centralised Services are provided by the EPTRI CMO and dedicated experts. These services include:
*Providing access to paediatric patients’ cohorts
*Providing access to paediatric samples and related data
*Advice on in-vivo / in-silico non-clinical study design
*Advice on in-vivo / in-silico clinical study design
*Advice and document preparation for regulatory and ethics procedures
*Advice on preparing HTA reports for paediatric medicines, diagnostics, and medical devices
*Advice on data and sample sharing preparation
*Support with applications to public and private funding opportunities

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Right to Try Act

Published by IRDiRC

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

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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Single Patient Expanded Access

Published by IRDiRC

Early Access Support Orphan Drug Orphan Medicine Regulatory Affairs Research and Drug Development 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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US Expanded Access Program

Published by IRDiRC

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

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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INTERACT Meetings (Initial Targeted Engagement for Regulatory Advice on CBER products)

Published by IRDiRC

Drug Development Advice Early Access Support Regulatory Advice Regulatory Affairs Scientific Advice Targeted Engagement for Regulatory Advice

Through a CBER Initial Targeted Engagement for Regulatory Advice on CBER producTs (INTERACT) meeting, sponsors can obtain initial, nonbinding advice from FDA regarding chemistry, manufacturing and controls, pharmacology/Toxicology, and/or clinical aspects of the development program

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Patient-focused drug development program

Published by IRDiRC

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

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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Stakeholders interactions – Professional affairs and Community Engagement

Published by IRDiRC

Clinical Trial Community Engagement Early Access Support Early Stage Interaction Patient Advice Professional Affairs Regulatory Advice Regulatory Affairs Scientific Advice

Interaction of the stakeholders and multilateral education at an early stage of development will facilitate drug development (e.g. ensuring outcome measures for clinical trials are developed that are meaningful for patients, are acceptable to the regulators, and are acceptable to payers)

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FDA Pediatrics Advisory Committee

Published by IRDiRC

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

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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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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