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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NIH funded programs and resources

Published by IRDiRC

Funded Programme NIH Initiative Orphan Drug Public Research and Drug Development

NIH is the largest public funder of biomedical research in the world, investing more than $32 billion a year to enhance life, and reduce illness and disability. The tool has its best use at very early phases of drug development

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European Network of Paediatric Research at the European Medicines Agency

Published by IRDiRC

Early Access Support European Network of Paediatric Research Orphan Drug Paediatric Medicine Regulatory Affairs Research and Drug Development

The European Network of Paediatric Research at the European Medicines Agency (Enpr- EMA) is a network of research networks, investigators and centres with recognised expertise in performing clinical studies in children. Enpr-EMA enables networking and collaboration with members from within and outside the European Union (EU), including academia and the pharmaceutical industry

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EU Compassionate Use Programs

Published by IRDiRC

Clinical Development Compassionate Use Early Access Support EMA Compassionate Use European Union Orphan Drug Regulatory Affairs Research and Drug Development

Compassionate use is a treatment option that allows the use of an unauthorised medicine. Under strict conditions, products in development can be made available to groups of patients who have a disease with no satisfactory authorised therapies and who cannot enter clinical trials.

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Sakigake Designation System

Published by IRDiRC

Early Access Support Japan MHLW or PMDA Regulatory Orphan Drug Regulatory Affairs Sakigake

SAKIGAKE aims at shortening premarket review period for innovative new medical products that satisfy certain criteria, such as severity of intended indication, by designating such products during the early stages of development, and providing prioritized consultation services and premarket pharmaceutical affairs review.

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Ethic’s Committees assessment of protocols for trials with orphan medications

Published by IRDiRC

Ethic and Legal Issue Guideline Ethical Assessment Orphan Drug Protocol Assessment Regulatory Sciences

Ethical assessment of clinical trials in orphan diseases should be done by experienced committees, and should ensure that all relevant aspects of this particular type of trials is properly assessed.

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Discovering New Therapeutics Uses for Existing Molecules (NTU) program, National Center for Advancing Translational Sciences (NCATS)

Published by IRDiRC

Drug Repurposing Funding NCATS NTU-NCATS Orphan Drug Research and Drug Discovery

The NTU program aims to improve the process of developing new treatments for diseases by finding new uses for existing therapies that already have cleared several key steps along the development path. Existing or partially developed therapeutic candidates can be repurposed for use in new disease indications

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EU planned cross-border treatments

Published by IRDiRC

Early Access Support EU Planned Cross-Border Treatment Orphan Drug Patient Information Research and Drug Development

Different ways to access the health care structures and professionals of the foreign EU Country National Health System (NHS) for people insured in the EU countries.

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Orphanet database

Published by IRDiRC

Database Database or Tool Orphanet Orphan Drug Rare Disease Research and Drug Development

Orphanet is a unique public resource worldwide, gathering and improving knowledge on all rare diseases (RD), affecting less than 1 in 2’000 people in the European population.

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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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