The introduction of a pan-European HTA: Shaping the landscape for future ATMP launches
The market access environment in Europe is complex and constantly evolving. While the transformative potential of advanced therapies is widely recognised, there is a growing urgency for European HTA processes to support timely patient access at prices reflecting the potential value of treatment, whilst managing uncertainty associated with the often limited data on long term benefits of treatment at the time of launch.
In 2021, a number of significant policy changes were announced that will impact the processes and methods for the evaluation of new health technologies in Europe, with implications for the assessment of clinical value and patient access across member states.
Lightning Health closely monitors the European policy landscape for developments relating to the pricing and market access for new medicines. Here, we take a look at the potential implications these policy changes may have for the launch of ATMPs in Europe.
European Joint Clinical Assessment (JCA)
A significant development in the European HTA landscape was announced in June 2021, confirming the introduction of a pan-European HTA process. The new European Joint Clinical Assessment will redefine the processes and methods for the evaluation of new medicines in the EU, potentially reducing the time and cost required to bring innovative therapies to patients.
Under the JCA process, EU member states will retain responsibility for determining the additional clinical benefit of a new medicine and making pricing and reimbursement decisions, however this will be based on a new centralised assessment of the clinical evidence including the strengths and limitations of available data and relative effects for health outcomes.
Scope for JCA
Importantly, under the JCA process, member states are likely to be permitted to perform complementary clinical analyses necessary for national level decision making, and retain autonomy for the assessment of non-clinical domains (such as pharmacoeconomic evaluation and the ethical aspects of care). Member states are also likely to be able to request the submission of additional clinical information if this has not already been submitted at the European level.1
Timelines for implementation
The JCA process will be introduced in a phased approach, starting with cancer therapies in 2024. This will then expand to include orphan drugs and ATMPs by 2027, and will be routinely used for all centrally approved medicines by 2032.
Tiemo Wölken, MEP, European Parliament Rapporteur
Proposed framework for the European HTA process
The programme will be led by the Co-ordination Group, comprised of national HTA organisations designated by member states1
Process map for the JCA report development
*E.g. relating to products outside of scope for the Joint Clinical Assessment (such as vaccines)
Implications for manufacturers
For all ATMPs predicted to launch from 2027 onwards, ensuring clinical development programmes align with JCA requirements will be critical, as the outcomes from this assessment will likely inform the clinical value assessment and pricing and reimbursement throughout EU member states. In particular, early engagement with the Joint Scientific Consultation Group will be important to ensure alignment on the appropriate pivotal trial design, endpoint selection and clinical comparator.
ATMP manufacturers should also anticipate ongoing requirements for national level submissions, potentially including requests for complementary clinical analyses to support the evaluation process, as well as data on non-clinical domains of HTA (such as pharmacoeconomic evaluation) to support pricing negotiations.
Additional European policy updates
In addition to the announcement of the JCA, several other developments have emerged in Europe with implications for the ATMP access landscape.
Ahead of the German Federal election in September 2021, the GKV-SpV has proposed several policy initiatives including a call for greater transparency on net pricing in other member states, mandatory registry data collection for new therapies with accelerated regulatory approval, and an ‘interim’ price at launch for these products based on the price of the appropriate comparator in Germany. Although any policy changes are unlikely to be announced until a new government is formed, these proposals align with ongoing initiatives across Europe promoting greater transparency in pricing between member states, and conditional reimbursement for advanced therapies based on ongoing evidence generation to inform future re-evaluation of the clinical benefit and pricing.
In July 2021, NHS England confirmed the creation of the “Innovative Medicines Fund”. This £680 million fund will support coverage with evidence development for clinically promising treatments where further data is needed to support NICE in making a final recommendation. This is likely to accelerate patient access to potentially transformative therapies in England. Furthermore, the ongoing NICE methods review has proposed ‘QALY modifiers’ that will increase with the willingness to pay for therapies that address the most severe diseases, potentially providing more flexibility within the HTA process for advanced therapies addressing indications with high clinical unmet needs.
Lightning Health can support you to navigate these complexities and to ensure robust value demonstration across healthcare systems. Contact us today to discuss how we can support your European launch and patient access strategy.
www.lightning.health +44(0) 203 488 9438
Sources: 1. Council of the European Union March 2021. Available at: https://www.consilium.europa.eu/media/48963/st07310-en21.pdf [Accessed September 2021]
Abbreviations: ATMP: Advanced Therapy Medicinal Products; GKV-SpV: GKVSpitzenverband (National Association of Statutory Health Insurance Funds); HTA: Health Technology Assessment; NICE: National Institute of Health and Care Excellence; QALY: Quality Adjusted Life Year
Prepared by Lightning Health. September 2021
Article published 28 October 2021