The increasing complexity of bringing innovative therapies to market across various jurisdictions presents a significant challenge in the pharmaceutical industry. This is particularly prominent in areas such as Advanced Therapy Medicinal Products (ATMPs), which face significant pricing and reimbursement challenges. To date, only a limited number of ATMPs have received marketing authorisation in Europe, with just nine approved so far. While some ATMPs have been reimbursed in a limited number of countries, recently, two specific therapies, Glybera and Zalmoxis, have been withdrawn from the market.1
Several factors contribute to the challenges in gaining market access for ATMPs, including manufacturing processes, clinical trial design, and the availability of clinical evidence. Furthermore, many health technology assessment bodies (HTAb) and payers have not yet established distinct assessment pathways for ATMPs.1 Notably, many of the challenges faced by ATMPs are also applicable to broader categories, such as oncology and orphan drugs.2,3 Given these hurdles, early scientific advice is pivotal for manufacturers to understand and address potential challenges.
Joint Scientific Consultations (JSCs) are a core component of the new EU Health Technology Assessment (HTA) Regulation (Regulation (EU) 2021/2282), which took effect in January 2025.5 JSC is an EU-level scientific advice process that enables Health Technology Developers (HTDs) to exchange information on their development plans for a medicinal product or medical device and obtain guidance on the information, data, analyses and other evidence that are likely to be required from clinical studies for the Joint Clinical Assessment (JCA) of those products.4 JSCs aim to streamline evidence generation and ultimately facilitate faster patient access to effective innovations. Importantly, JSCs can be conducted in parallel with the European Medicines Agency (EMA)’s scientific advice process, allowing for joint EMA-HTA feedback in one unified interaction.5
The article aims to clearly explain when and how to engage in JSCs and how to maximise their value.
When should manufacturers consider a JSC?
Engaging in a JSC during clinical development can be highly beneficial. Ideally, JSCs should be sought during the development of the Phase 3 (pivotal) clinical trial protocol. This allows manufacturers to make relevant adjustments (e.g., to primary endpoints, comparator choices and statistical powering) to their clinical development plans without derailing internal timelines. Recent data showed that in 57% of cases, manufacturers modified their clinical development plans in response to HTAb List of Issues.6 This statistic underscores that JSC advice can meaningfully impact development decisions.
There are two defined request submission periods for manufacturers to apply for a JSC (Figure 1):5
Note: First window: 3 February – 3 March 2025, was reserved for medicinal products only; Second window: 2 June – 30 June 2025, open to both medicinal products and certain medical devices
Source: European Commission, 2025.5
The HTA Coordination Group (HTACG) has indicated that approximately ten JSC slots will be available in 2025. This includes five to seven slots for medicinal products and up to 3 slots for high-risk medical devices. Developers must therefore meet the following specific eligibility criteria and demonstrate high priority to secure one of these limited slots:7
- Unmet medical need
- First-in-class
- Potential impact on patients, public health, or healthcare systems
- Significant cross-border dimension
- Major Union-wide added value
- Union clinical research priorities
Since the 12th of January 2025, all new oncology therapies and ATMPs (including gene therapies, chimeric antigen receptor T-cell therapies, and regenerative medicines) will face mandatory JCAs. JCAs will become mandatory for orphan drugs from 2028 and for all drugs going through the EMA process from 13th January 2030. Therefore, engaging in JSC during development can be highly strategic for manufacturers to ensure that new medicines meet requirements and avoid delays in market access.
Benefits of JSCs – Why is it important for manufacturers?
A key benefit of engaging in a JSC is the opportunity to obtain harmonised, multinational feedback. JSCs ensure that manufacturers receive a unified perspective on core evidence requirements and can resolve potential discrepancies between countries early, such as differing expectations relevant comparators. Designing clinical trials for oncology therapies and ATMPs comes with unique challenges, as these trials often involve multiple endpoints (e.g., survival rates, quality of life, and surrogate markers, while also keeping pace with evolving standards of care.1,3
JSCs provides a platform to discuss suitable trial comparators, the durability of treatment effects, and the long-term outcomes required. This reduces the risk that a company conducts a trial acceptable to EMA but inadequate for the JCA and local HTA (a scenario which has historically led to delays in patient access). It can also address how to manage uncertainties in evidence. JSCs can guide manufacturers in designing comprehensive follow-up studies or registry data collection to meet HTA concerns about long-term value. This approach maximises the likelihood of demonstrating added clinical benefits for HTAb and can save resources by avoiding redundant or misdirected studies.
The policy landscape for JSCs is being shaped by ongoing feedback from stakeholders. A Stakeholder Network has been established to facilitate open dialogue, regular consultations, and information sessions.5 We may see adjustments in the future, such as an increase in the number of JSC slots, based on demand and feedback. By engaging constructively and sharing their experiences, manufacturers and consultants can help create a more streamlined HTA system that benefits patients, payers, and innovators.
What should manufacturers do to enable effective engagement in JSC
Manufacturers need a BRAVE approach to ensure optimal advice is received:
B – BE BOLD WITH QUESTIONS
Ask challenging, forward-thinking questions to gain tailored insights
R – RECOGNISE RED LINES
Know your non-negotiables and areas where flexibility is required
A – ALIGN INTERNALLY
Ensure cross-functional teams are invested in the goal of seeking advice
V – VALIDATE YOUR ARGUMENTS
Use data and expert input to support strategic narrative and company positions
E – ENGAGE PROACTIVELY
Clarify and follow-up on feedback, approaching the advice as a two-way dialogue
By adopting a Brave approach, manufacturers can better establish an internal process for the JSC while ensuring cross-functional alignment. Internal teams should collaboratively identify the key questions for the JSC and agree on potential concessions they are willing to make (e.g., adding an active comparator arm). This collaborative effort will enhance preparation, leading to more productive discussions.
Additionally, breaking down silos is essential. Regulatory and market access teams must collaborate closely to ensure that the final trial design meets both approval and reimbursement goals. Some companies conduct internal “mock HTA” boards or advisory sessions with external HTA experts in advance. This proactive strategy allows manufacturers to anticipate potential concerns and address them effectively in the submission dossier.
Conclusion
In summary, the HTA landscape in 2025 is characterised by transition and adaptation. JSCs have the potential to create harmonisation, which can increase the consistency of the evidence package generated to better inform JCAs and potentially prevent costly post-launch studies or negative HTA opinions. The JSC process is operational, but stakeholders from all sides are learning through experience. Manufacturers should adopt a BRAVE approach when engaging in JSCs and remain attentive to policy updates and new developments.
Remap Consulting offers strategic guidance and practical support to manufacturers seeking early HTA advice. We provide tailored strategic input and planning assistance to help navigate this complex process.
Watch our on-demand webinar, “Navigating Early Scientific Advice for Pricing and Market Access Success.” In this session, we introduce Remap’s proven BRAVE framework and share valuable insights on how to effectively utilise early scientific advice in HTA processes. Access the recording here.
References
- van Overbeeke E, Michelsen S, Toumi M, et al. Market access of gene therapies across Europe, USA, and Canada: challenges, trends, and solutions. Drug Discovery Today. 2021/02/01/ 2021;26(2):399-415. doi:https://doi.org/10.1016/j.drudis.2020.11.024
- Fontrier AM. Market access for medicines treating rare diseases: Association between specialised processes for orphan medicines and funding recommendations. Soc Sci Med. Aug 2022;306:115119. doi:10.1016/j.socscimed.2022.115119
- Chapman S, Paris V, Lopert R. Challenges in access to oncology medicines. Policies and practices across the OECD and the EU. Organisation for Economic Co-operation and Development (OECD). Accessed 14/05/2025, https://www.oecd.org/en/publications/challenges-in-access-to-oncology-medicines_4b2e9cb9-en.html
- Safety D-GfHaF. Factsheet – Joint Scientific Consultation for Medicinal Products (January 2025). European Commission. Accessed 14/05/2025, https://health.ec.europa.eu/publications/factsheet-joint-scientific-consultation-medicinal-products-january-2025_en
- Commission E. Joint Scientific Consultations. European Commission. Accessed 14/05/2025, https://health.ec.europa.eu/health-technology-assessment/implementation-regulation-health-technology-assessment/joint-scientific-consultations_en
- Ibargoyen-Roteta N, Galnares-Cordero L, Benguria-Arrate G, et al. A systematic review of the early dialogue frameworks used within health technology assessment and their actual adoption from HTA agencies. Systematic Review. Frontiers in Public Health. 2022-October-06 2022;Volume 10 – 2022doi:10.3389/fpubh.2022.942230
- Bean S. What are Joint Scientific Consultations and why they are important for manufacturers to consider? Remap Consulting Accessed 14/05/2025, https://remapconsulting.com/hta/what-are-joint-scientific-consultations-and-why-they-are-important-for-manufacturers-to-consider/
- Commission E. Health Technology Assessment: First joint clinical assessments begin. European Commission Accessed 14/05/2025, https://ec.europa.eu/newsroom/sante/newsletter-archives/62551