They are driving the industry forward, featuring speakers from across the entire value chain of cell and gene therapy development; forward-thinking pharma, biotech and start-up companies, researchers, clinicians, academics, HTAs, payers and regulators.
What are the general gene therapy market access insights and recommendations?
Insights:
- Gene therapy vectors often trigger immune responses, posing regulatory concerns.
- Early diagnosis through newborn screening maximises therapy benefits.
- Storage and preparation of gene therapies are complex, affecting their handling and viability.
- Different HTA outcomes for gene therapies like Yescarta and Alofisel highlight that the level of clinical benefits is a key determining factor in HTA decisions.
Recommendations:
- Highlight trial data indicating low immunogenicity or successful treatment with immunosuppressants and ensure monitoring plans post-approval.
- Engage governments early to implement cost-effective newborn screening.
- Collaborate with NHSE and specialized centres to establish necessary infrastructure for storage and administration.
- Ensure robust evidence generation and consider flexible reimbursement schemes to manage clinical uncertainties.
How does ATTC network contribute to overcoming the advanced therapy adoption challenges in the UK?
Insights:
- The ATTC Network coordinates NHS centres and organizations to establish systems for routine delivery and widespread adoption of ATMPs, positioning the UK as a leader in advanced therapy development and ensuring the NHS is ready to deliver these therapies to benefit UK patients.
Recommendations:
- Leverage ATTC network expertise for clinical trials with government funding for clinical trials.
- Pilot performance-based reimbursement models to address the uncertainty around the long-term clinical value provided by ATMPs and thus, improve misalignment between the UK HTA bodies and advanced therapy manufacturers.
- Increase targeted Investment in workforce and infrastructure delivery to support specific advanced therapies requirements.
- Apply better collation and management of the data for guiding key reimbursement and investment decisions of support long-term real-world treatment outcomes.
- Create a Government-led multi-stakeholder, cross-department advanced therapy taskforce in early 2024 to implement these recommendations through a coordinated national strategy.
What are the key takeaways of Voluntary scheme 2024?
The Voluntary scheme for branded medicines pricing, access and growth is a voluntary agreement between the DHSC, NHS England and the Association of the British Pharmaceutical Industry, designed to improve patient outcomes, manage the NHS’s budget, and support the life sciences industry. The 2024 Voluntary Scheme listed the following key points:
- NHS England commits to delivering two innovative payment model pilots, exploring the practicalities of outcomes-based agreements for ATMPs.
- Review Budget Impact Test threshold and launch a consultation on increasing the threshold to £40 million.
- Streamline medicine supply within the NHS to ensure cost-effective access to innovative treatments.
- Introduce new a PharmaScan platform in the first three years of 2024 Voluntary Scheme.
You can find out more about FIECON's approach in rare disease storytelling here: https://www.linkedin.com/pulse/patient-perspectives-rare-disease-fiecon-limited/
---
ATMP – Advanced therapy medicinal products; DHSC – Department of Health and Social Care; NHS – National Health Service; ATTC – Advanced Therapy Treatment Care; HTA - health technology assessment; R&D – Research and development; NICE – National Institute of Health and Care Excellence
