Latest CIRS activities on rare diseases
In recognition of Rare Disease Day today, we wanted to share what CIRS has been up to recently to help advance regulatory and HTA policies and processes in the [...]
CIRS participates in new RDI-Lancet Commission on Rare Diseases
CIRS is participating in the Rare Diseases International-Lancet Commission on Rare Diseases (RDI-LCRD), which aims to catalyse global recognition and action for people living with a rare disease, driving systemic change to [...]
CIRS RD Briefing 96 – Review of HTA outcomes and timelines in Australia, Canada and the UK 2019-2023
This R&D Briefing presents data from HTADock, an ongoing metrics study that collects publicly available data on new active substances (NASs) appraised by key international HTA agencies. It focuses [...]
Regulatory processes for rare disease drugs in the US and EU: National Academies study supported by CIRS
A new report from the NASEM Health and Medicine Division offers recommendations for enhancing rare disease drug development. CIRS is delighted to have been able to collaborate with NASEM [...]
CIRS RD Briefing 95 – Review of HTA outcomes and timelines in Australia, Canada, Europe and the UK 2019-2023
This R&D Briefing presents data from HTADock, an ongoing metrics study that collects publicly available data on new active substances (NASs) appraised by key international HTA agencies, each with [...]
Rare Disease Product Approvals: The Changing Regulatory And HTA Landscape Between 2018-2022
Background Globally, 7,000 rare diseases affecting 300 million people pose development challenges with small patient populations. Developing medicines for rare diseases requires innovation. Despite regulatory incentives, challenges for HTA [...]
Incentives for rare disease products
The Centre for Innovation in Regulatory Science (CIRS) held a multi-stakeholder workshop that examined current regulatory and reimbursement frameworks for rare disease products (orphan drugs), reviewing how these can [...]
Latest CIRS research posters
So far 2024 has been a busy year conference wise for CIRS! CIRS staff have presented work and/or moderated sessions at several conferences including DIA Europe, RAPS Euro Convergence, [...]
CIRS RD Briefing 93 – New drug approvals by six major authorities 2014-2023
This R&D Briefing presents the results from the Centre for Innovation in Regulatory Science (CIRS) annual analysis of new active substance (NAS) approvals by six major regulatory agencies: the [...]
2023 Workshop report – Review and reimbursement frameworks for rare disease products
There are an estimated 300 million people across the world affected by around 7000 known rare diseases. Challenges in bringing treatments to market for these conditions include small patient [...]