Access to Medicines
UK
- National Institute for Health and Care Excellence (NICE) has approved access to cystic fibrosis modulator drugs Kaftrio, Symkevi and Orkambi, following the conclusion of a commercial agreement between the NHS and the pharmaceutical company Vertex. See NICE positive recommendation .
- NICE has recommended CSL Behring’s Hemgenix (etranacogene dezaparvovec) as the first single infusion gene therapy for adults with severe or moderately severe haemophilia B without a history of FIX inhibitors for reimbursement via a managed access agreement, recognising the treatment as cost-effective in England. This is the first time a medicine has been granted managed access through the Innovative Medicines Fund since it was launched in 2022. See press release for further information.
Artificial Intelligence
Ireland
- The Irish Department of Trade, Enterprise and Employment opened a public consultation on the national implementation of the EU AI Act in May. The consultation is intended to inform Ireland’s approach to implementing the Act and, specifically in relation to the configuration of national competent authorities required for implementation. The consultation is open until 16th July 2024. For further information on the consultation see here
Clinical Trials
EU
- The revised EU Clinical Trial Information System (CTIS) Transparency Rules have gone live. The revised Rules seek to strike a balance between transparency of information and protection of commercially sensitive confidential information. See European Medicines Agency (EMA) press release for further information.
Competitiveness Strategy
EU
- European Federation of Pharmaceutical Industries and Associations (EFPIA) has published a Competitiveness Strategy for European Life Sciences , proposing five recommendations that can help make the region a World-leader in life sciences and secure EU competitiveness. See EFPIA Press Release
Gene and Cell Therapies
Germany
- The Germany Federal Government has been handed the strategy paper for gene and cell therapies, which will serve as the basis for policymaking and give Germany a leading role around gene and cell therapies in Europe. The proposed strategy is divided into eight areas with respective strategic objectives and proposed implementation measures intended to put Germany at the forefront for gene and cell therapies. See National Strategy for Gene and Cell Therapy for further information.
Health Technology Assessment
EU
- The European Commission has published two new guidance documents for the joint clinical assessments under the EU Health Technology Assessment Regulation: i) Guidance on outcomes for joint clinical assessments ; and ii) Guidance on reporting requirements for multiplicity issues and subgroup, sensitivity and post hoc analyses in joint clinical assessments.
- The EMA published guidance on 21 June 2024 to applicants/health technology developers on how to declare in the EMA Letter of Intent (via the Pre-submission request form) whether their application falls under the scope of the Health Technology Assessment Regulation ((EU) 2021/2282 Article 7) and, therefore, is subject to JCA. See the pre-authorisation guidance for further information and news announcement
- The European Commission has launched an online public consultation on cooperation with the EMA under the Health Technology Assessment Regulation. The online consultation is open until 24th July 2024. See here for further information on the consultation.
- The European Commission has announced a half-day stakeholder event on Implementing the EU Health Technology Assessment Regulation, which will take place on 5th November 2024. For further information on the event and how to register see here
Medicines Shortages
EU
- EMA and the Heads of Medicines Agencies (HMA) have issued recommendations to tackle shortages of the glucagon-like peptide-1 (GLP-1) receptor agonists Ozempic (semaglutide), Saxenda (liraglutide), Trulicity (dulaglutide) and Victoza (liraglutide). See press release and recommendations for further information.
Regulatory
EU
- EMA’s human medicines committee (CHMP) recommended ten medicines for approval at its June 2024 meeting. See EMA press release fir further information.
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