Will SUSTAIN-HTA bring clarity to the pan-EU JCA methodologies and priorities?

Potential outlook of the JCA development

With less than a year to go until the launch of the Joint Clinical Assessment (JCA) in January 2025, its successful implementation relies on the readiness of each EU market and their willingness to incorporate the JCA into current HTA processes. The SUSTAIN-HTA project aims to bridge the gap between individual markets, the JCA and wider EU HTA updates, facilitating the adoption of new HTA methodologies across markets and ultimately taking a step towards a unified pan-EU HTA assessment.

Pan-EU Joint Clinical Assessment Overview

The JCA will be introduced as part of the recent Health Technology Assessment Regulation (HTAR), with a mission to accelerate patients’ access to medical products by strengthening HTA quality and increasing overall efficiency through process harmonization and minimizing effort duplication. The JCA will first be applied to products with challenging assessments, such as advanced therapy medicinal products (ATMPs) that often have data based off single arm trials and difficulties with determining durability, and oncology products, where comparators and guidelines are constantly evolving.

Recently, the guidelines for the JCA have been disclosed, defining the proposed procedures and timelines, and providing templates for the required dossiers, JCA reports, and summary reports. Two more recent documents also recommend against Member States specifying outcome measures and provide guidance on how to assess immature / short-term outcome data, PROs, and surrogate outcomes.[1] Despite the additional clarity on the JCA and outcomes, the extent to which individual countries will utilize the JCA remains uncertain, given that countries will still maintain full autonomy regarding market access and reimbursement decisions.[2] Therefore, to encourage uptake of the JCA within individual markets and ensure the long-term viability of this standardized approach to HTA assessments, the SUSTAIN-HTA project was initiated.

What is SUSTAIN-HTA?

SUSTAIN-HTA stands for Support the Utilisation of Sustainable and Tailored Innovative methods for HTA and was funded through a €5 million Horizon Europe grant.[3] Launched in January 2024 and set to continue until at least 2027, SUSTAIN-HTA supports the implementation of the new HTA methods, through initiating research to understand the needs of individual HTA bodies, then building tools to address those needs.[4,5]

SUSTAIN-HTA represents a collaborative effort among different stakeholders of the JCA reform. The project is directed by Utrecht University, in close collaboration with HTA bodies and several universities, SMEs and NGOs from the Netherlands, Spain, Italy, and Hungary. [4,5] Interestingly, Norway (NOMA) and the UK (NICE) take seats as partners despite not being EU members, with NICE playing a leading role in prioritising areas of methods development. [6,7]

Tools and Functions of SUSTAIN-HTA

The approach of SUSTAIN-HTA is comprehensive, spanning from early research through to implementation and ongoing support, which is reflected through the tools in development, including: [4]

Overall, the objectives are in line with Professor Wim Goettsch’s (Utrecht University) summary of the work of SUSTAIN-HTA: [8]
“We aim to be an expert, neutral platform that provides balanced information about these new methods while also being a safe harbour to test the new methods in practice and provide training to support widespread implementation.”

Implications for manufacturers

For manufacturers, the most pressing question is how the JCA reform will affect the preparation of HTA dossiers. To what extent will countries leverage the JCA, and how influential will it be in HTA outcomes?

The JCA’s new evidence requirements and structure will require manufacturers to invest additional time preparing their HTA submissions. However, given it is uncertain how countries will leverage the JCA for their HTAs, there remains the risk of further effort duplication and wasted time due to a more demanding and/or separate dossier preparation process. European pharmaceutical bodies have criticised the short timeline for manufacturer dossier submission (previously 90 days and will be extending to 100 days) for being unworkable and for the risk of limiting the quality of evidence.9 Manufacturers must focus their efforts on developing tailored dossiers for the JCA, where understanding its methods and requirements is key to ensure readiness. However, for the time being, manufacturers should also be prepared to undergo the scrutiny of market specific HTA in addition to the JCA. Closely following the developments of SUSTAIN-HTA and the JCA will be valuable to plan a successful launch.

SUSTAIN-HTA’s work can serve as an additional source of information to inform developments of the JCA due to involvement from a wide range of voices in addition to the EMA. With this in mind, SUSTAIN-HTA will be important to provide a communication channel between bodies, supporting the integration of the JCA and ensuring the system is future-proof. Therefore, the few EU markets that are directly involved in SUSTAIN-HTA may benefit from smoother integration of the JCA into their HTA processes, compared to others, due to higher willingness to engage. Nonetheless, some other markets (e.g. FR, DE) are key drivers of JCA development, though not directly involved in SUSTAIN-HTA. NICE’s involvement in SUSTAIN-HTA and the methodology development for the JCA further informs the Member States’ shift towards cost-effectiveness as a key consideration in their HTAs.

Final Thoughts

While details are still being unveiled on how the JCA will evolve and the specific outcomes it may bring, the picture is beginning to take shape. For example, AIFA has recently confirmed that it will adopt EU Regulations and use JCA outcomes in its HTA process. [10] Industry leaders such as Roche are already preparing some of its molecules to go through the JCA by setting up cross-functional and specialist teams, calling for more transparency and details on JCA procedures and methodologies. [11] Despite potential obstacles and imperfections in the integration of the JCA, the hopes of all stakeholders, including manufacturers, regulatory bodies, healthcare professionals and patients, are in line with Professor Goettsch’s outlook: “We hope that, gradually, we can bring everyone on board in our effort to implement new HTA methods”. [12]

How Can Windrose Help?

Windrose is helping clients to navigate the upcoming EU HTA and complex uncertainty that it brings. Through our ongoing work in market access, extensive experience working across the European markets, and commitment to staying atop of developments in this space, we endeavour to ensure Global Market Access teams are equipped with strategies that align with the criteria for success. For more information, please get in touch via email at info@windrosecg.com.

References:

Health Technology Assessment: two new guidance documents published - European Commission. Accessed June 27, 2024. https://health.ec.europa.eu/latest-updates/health-technology-assessment-two-new-guidance-documents-published-2024-06-13_en
EU HTA: Can a one-size-fits all approach really work, and how can manufacturers prepare? — WindroseCG. Accessed June 28, 2024. https://windrosecg.com/posts/eu-hta-jca
Improving the implementation of innovative methods in health technology assessment practice - News - Utrecht University. Accessed June 27, 2024. https://www.uu.nl/en/news/improving-the-implementation-of-innovative-methods-in-health-technology-assessment-practice
Sustain HTA – Support Utilisation of Sustainable and Tailored Innovative methods for HTA is a European initiative to build a supporting infrastructure. Accessed June 27, 2024. https://sustain-hta.org/
IMPLEMENTATION ROLLING PLAN 2023-2024 REGULATION (EU) 2021/2282 ON HEALTH TECHNOLOGY ASSESSMENT. Published June 2024. Accessed June 27, 2024. https://health.ec.europa.eu/document/download/397b2a2e-1793-48fd-b9f5-7b8f0b05c7dd_en
Our projects and partners | Our research work | What we do | About | NICE. Accessed June 27, 2024. https://www.nice.org.uk/about/what-we-do/our-research-work/our-projects-and-partners
SUSTAIN-HTA | Cergas. Accessed June 28, 2024. https://cergas.unibocconi.eu/international-projects/sustain-hta
SUSTAIN HTA to advance innovative HTA methods in Europe. Accessed June 27, 2024. https://www.evidencebaseonline.com/sustain-hta-launched-to-advance-and-align-innovative-hta-methods-in-europe/
Life science industry concerns over the workability of EU HTA: Europe cannot miss out on the opportunity to speed up access to innovative medicines for European patients. Accessed June 27, 2024. https://www.efpia.eu/news-events/the-efpia-view/statements-press-releases/life-science-industry-concerns-over-the-workability-of-eu-hta-europe-cannot-miss-out-on-the-opportunity-to-speed-up-access-to-innovative-medicines-for-european-patients/
AIFA-Farmindustria meeting. AIFA’s President Nisticò: “Right away a technical committee to simplify procedures and halve the time for access to medicines.” Accessed June 28, 2024. https://www.aifa.gov.it/en/-/incontro-aifa-farmindustria-il-presidente-dell-agenzia-nistic%C3%B2-subito-un-tavolo-tecnico-per-semplificare-le-procedure-e-dimezzare-i-tempi-per-l-accesso-ai-farmaci-
EU HTA Implementation: Challenges, Commitments and Collective Actions (Guest blog). Accessed June 28, 2024. https://www.efpia.eu/news-events/the-efpia-view/blog-articles/eu-hta-implementation-challenges-commitments-and-collective-actions/
Improving the implementation of innovative methods in health technology assessment practice - News - Utrecht University. Accessed June 27, 2024. https://www.uu.nl/en/news/improving-the-implementation-of-innovative-methods-in-health-technology-assessment-practice