Cell and Gene Therapies
Cell and gene therapies offer substantial therapeutic benefits but are expensive and often have a limited evidence base at launch. There are concerns over affordability and long-term cost-effectiveness, as well as uncertainty as to whether their value is accurately captured by current HTA methods.
Health Technology Assessment of Gene Therapies: Are Our Methods Fit for Purpose?
1 June 2022
Gene therapies represent a new era of medicine, offering the potential for truly transformational health gains, and further benefits for society and health systems.
Key Considerations for Early Access Schemes for Single-Administration (One-Time) Therapies
1 June 2021
Early access schemes enable patients in exceptional need to access therapies that are not yet available through their health system. These schemes were not designed for…
Exploring the Financial Sustainability of Gene Therapies
5 January 2021
Gene therapies represent a paradigm shift in medicine, with the potential to address the root causes of chronic diseases. They offer one-time treatment regimens and, in some cases, potentially a cure. As a result, they offer transformative value for patients, physicians, health systems and society. However, with the prospect of more gene therapy approvals there is concern in Europe that these technologies could threaten the financial sustainability of health systems.
The Promise of Gene Therapies: Are We Ready?
1 April 2021
In a webinar held on 16 March 2021, Mary Harney, Annie Hubert and Simone Boselli joined OHE’s Adrian Towse to debate the issues surrounding the adoption of gene therapies. In this interactive report we highlight our 5 key takeaways from the debate with clips, quotes and highlights.
Personalised Medicine: is it an Oil-Rush or Oil-Spill?
1 May 2020
Personalised medicine, also known as ‘precision’ or ‘stratified’ medicine, targets treatment by using genetics and/or biomarkers to identify patients or subgroups of patients who have distinct…
Early Experience with Health Technology Assessment of Gene Therapies in the United States: Pricing and Paying for Cures
1 February 2019
Gene therapies, which can provide cures for diseases, are a new area of research for ICER. Some are within two to three years of becoming available…
Gene Therapy: Understanding the Science, Assessing the Evidence, and Paying for Value
1 March 2017
This report presents an analysis of the significant clinical potential of gene therapy and the unique challenges in developing and evaluating evidence on their effectiveness and…
Exploring the Assessment and Appraisal of Regenerative Medicines and Cell Therapy Products: Is the NICE Approach Fit for Purpose?
1 February 2017
In 2016 the University of York undertook a review exercise to determine whether NICE’s existing methods and processes are appropriate for assessment of regenerative medicines. The…
Genomics, Healthcare and Public Policy
1 October 1999
This Conference on Genomics, Healthcare and Public Policy, organised by the Office of Health Economics in collaboration with the School of Public Policy, University College London,…