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 area of rare diseases.

Published insights on orphan drug approvals and HTA recommendations in key markets, helping to inform company and agency strategies.

  • R&D Briefing 93 – New drug approvals by six major authorities 2014-2023 – see p9
  • R&D Briefing 95 – Review of HTA outcomes and timelines in Australia, Canada, Europe and the UK 2019-2023 – see p8-9
  • R&D Briefing 96 – Review of HTA outcomes and timelines in Australia, Canada and the UK 2019-2023 – see p10

Conducted data analyses that informed recommendations made to the FDA by a National Academies Committee on Regulatory Processes for Rare Disease Drugs in the US and EU.

The resulting report provided recommendations for enhancing and promoting rare disease drug development by improving engagement with people affected by a rare disease, advancing regulatory science, and fostering collaboration between the FDA and EMA.

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Participation in the RDI-Lancet Commission on Rare Diseases, which aims to catalyse global recognition and action for people living with a rare disease.

As one of the Commissioners, Head of CIRS Anna Somuyiwa will provide CIRS insights on the regulatory and HTA landscape for rare diseases, including the views of regulators, HTA agencies and pharmaceutical companies, into the Commission’s work.

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