All Rare diseases articles
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Industry newsNew MHRA framework sets out IMA pathway for rare disease therapies
21 May saw the MHRA unveil their draft rare disease framework, which is open for consultation until 30 of July. The new framework aims to adapt the regulatory framework for rare diseases, defined as affecting 1 in 50,000 or less people in the UK, as the current development ...
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ContentsRegulatory Rapporteur April 2026 – Contents
The April issue of Regulatory Rapporteur focuses on access to medicines.
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EditorialAccess to medicines: From approval to
patient-centred deliveryThis issue focuses on access to medicines, and demonstrates how lifecycle regulation, diverse evidence, operational innovation and corss-sector collaboration all contribute to improving the timely, equitable and trusted delivery of medicines.
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JournalThe regulatory valuation of patient experience data in rare diseases: A review of FDA and EMA labelling outcomes and methodological determinants
This article summarises a review carried out to evaluate patient experience data inclusion in regulatory assessements and labelling for rare diseases in the EU and the US
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JournalFrom laboratory to patient: The role of New Substance Notifications in delivering innovative therapies in Canada
This article explores the new substance notification (NSN) processes and provides guidance on navigating this.
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Industry newsMHRA advances new reform for the treatment of rare diseases
This industry news update explores the MHRA’s upcoming framework that addresses treatments related to rare diseases.
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JournalThe importance of patient advocacy in the EMA ODD process
This article discusses the role of patient advocacy groups in the orphan drug designation process to incoporate more patient-centred approaches.
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