Anticipating issues, tackling challenges: Next steps with the JCA
By AmerisourceBergen
During a recent panel discussion that took place at the World Evidence, Pricing and Access Congress 2024 in Amsterdam, Herbert Altmann, Vice President, Pan European Market Access and Healthcare Consulting, Cencora, discussed with market access experts the impact that the JCA will have on companies, national HTA procedures and, crucially, on patient access. Separately, Cencora’s new EU HTA lead, Ruairi O’Donnell, has also weighed in on the JCA process, foreseeable challenges and mitigating likely and potential issues.
Implementing acts: Delays and outcomes
The draft of the first of six implementing acts defined in the Health Technology Assessment Regulation (HTAR) has finally been published. The published draft of the act – Joint clinical assessment for medicinal products – is largely in line with expectations, with a few surprises. During the panel discussion, Neil Grubert, an independent Global Market Access consultant, pointed to the potentially onerous volume of data requirements.
Among the requirements Grubert noted are:
Degree of certainty of the relative effectiveness and relative safety with regard to the PICOs (patient population, intervention, comparison, and outcomes).
- Epidemiological data for relevant EEA states, including “any profound differences between these states.”
- For medical conditions that result in disability and/or a need for a family caregiver, and for treatments that result in major changes to healthcare systems, details of the organizational and societal impact of the condition and its treatment.
- Information on substantial variations in clinical pathways between EEA states (including European clinical guidelines).
- Details of early access/compassionate use programs.
- HTA reports from EEA states, Australia, Canada, the United Kingdom, and the United States.
He also highlighted the tight deadlines outlined within the implementing act that could become even more challenging alongside the shortened marketing authorization timelines proposed under the forthcoming revision of the EU general pharmaceutical legislation (GPL).
Casper Paardekooper, Partner, Vintura, part of Cencora, reiterated concerns over the increased amount of data that will need to be submitted. For example, epidemiological data for all countries, clinical pathways for all countries, and HTA reports from other countries all present an additional burden for industry. Getting the epidemiological data will be particularly challenging for smaller companies that likely won’t have a local affiliate in every European country, Paardekooper added.
O’Donnell’s analysis of the first implementing act shows that time constraints are a huge challenge for industry, both in terms of preparedness for the JCA process, as well as dossier submission timeline – just 90 days after the release of the PICO frameworks.
“This 90-day window creates immense time pressure within the entire system, emphasizing the urgency for early preparation and timely action,” O’Donnell said. He underscored that manufacturers will have to clearly identify any PICOs for which results were not submitted and explain the reasons for their omission.
The remaining five implementing acts have yet to be published, and the most recent implementation update from the European Commission indicates the last two acts are expected by the fourth quarter of 2024i. Grubert said these delays add to concerns about being ready for the JCA by January 2025.
Ideally, according to Paardekooper, the commission should take on board more expertise from industry when developing the implementing acts, which industry representatives have previously noted is integral to achieving successful implementation of the EU HTAii.
Challenges and opportunities for industry
During the summit panel discussion, Mihai Rotaru, Director, Market Access, The European Federation of Pharmaceutical Industries and Associations (EFPIA), said collaboration between industry and HTA bodies had, so far, been very limited. While flagging concerns for industry, Rotaru welcomed the feedback opportunities on the implementation acts. However, he questioned whether stakeholders would have a similar avenue to provide feedback on the methodologies and detailed procedures coming from the Member State Coordination Group on HTA (HTACG)iii.
Some positives Paardekooper pointed out include increased involvement of the health technology developer (HTD) in the JCA process and an elevated role for experts and stakeholders in the process. “Inclusion of the HTD in the process might result in some input during scoping, if invited,” he said, however he added that invitation-only involvement of HTDs during the actual JCA process is disappointing.
For his part, O’Donnell says lack of patient and manufacturer input in shaping the scope of the process and designing the PICOs does present concerns. “Meaningful dialogue about the product and evidence during the process is lacking, with only a few moments of technical clarification currently planned,” O’Donnell said.
Potential for external input comes from a new cross-border HTA collaboration that will support EU joint HTA. SUSTAIN-HTA -- Support the Utilisation of Sustainable and TAilored INnovative methods for HTA – is a collaboration of 15 partners across academia, HTA agencies and industryiv. It seeks to support the use of innovative methods for European HTA. Despite no longer being part of the EU, the United Kingdom’s National Institute for Health and Care Excellence (NICE) will play a lead role in reviewing existing HTA practices and prioritize areas for future tool and method innovationv.
“The lack of feet on the ground also presents a problem for local HTA discussions, which are difficult to coordinate from a global or European headquarter perspective,” Paardekooper said. “Small and medium-sized companies also have smaller teams and may struggle to absorb the significant additional workload associated with the JCA process.”
Preparing for the JCA
The emphasis for industry must be to prepare their cross-functional teams for new operating models at a global level and with affiliates and local stakeholders.
This will require local level education, particularly in the smaller countries, with advanced therapy medicinal products (ATMPs) and oncology drugs, which will be the first to be assessed by the JCA, Altmann said.
Altmann believes that, from the industry’s perspective, having market access functions drive clinical design from the early stages will help to develop the evidence needed for a successful HTA.
This reiterates his previous point emphasizing the importance of adopting best practices, such as getting internal alignment after phase II clinical data read out, before phase III clinical trial design is finished. This could allow companies to adequately prepare for the cross-functional process and avoid potential delays or setbacks in the JCA process.
In a separate analysis, O’Donnell emphasized the importance of starting the main activities for the JCA process over a year before the planned EMA submission date.
“However, with regards to PICO prediction, it's important to note that this approach may not be a one-size-fits-all solution,” he said. “For indications with multiple comparators and commercial risks related to downstream market share, PICO simulation exercises will be necessary during the preparation phase. These activities help minimize risk and maximize outcomes in such complex scenarios.
“On the other hand, if there are few comparators, or if overall access success is more dependent on other factors (e.g. pricing for a rare disease intervention), a different approach or better utilization of resources may be more appropriate than conducting a full PICO simulation.”
Impact on innovation
However, industry’s limited involvement in shaping the JCA process has dampened optimism, Grubert said. This raises questions as to whether the JCA will enhance Europe’s attractiveness as compared with emerging markets such as China and Asia-Pacific.
For his part, Altmann fears the JCA process will make innovation in the EU more challenging, as the industry doesn’t like uncertainty. He noted that the complexity around JCA, followed by the national HTA pricing and reimbursement process, may increase the focus on the United States as the primary market.
From EFPIA’s perspective, European-based pharmaceutical manufacturers are still very committed to innovation and to provide these to European patients, Rotaru said. However, there are concerns about the JCA and HTAR in general. Harmonization, particularly with regards to how evidence is developed, will be integral if those issues are to be resolved, he said.
“The one clear way where the EU can use the JCA as an opportunity to become more attractive is if they speak with one voice,” he said. With the scientific advice piece and the clinical assessment piece, if there is a real effort from 27 member states to speak with one voice in a way that could impact clinical development plans, Europe could have a huge impact, he added.
All eyes will be on the first submissions to determine how the process affects innovation, Paardekooper said. “If we indeed see a clear single voice coming through in the scoping and also a strong adoption of the JCA on a local level, it will definitely make Europe more attractive,” he said. “That is clearly needed, because the implementation of the proposed EU GPL has the risk of making Europe less so.”
This article is intended to communicate Cencora’s capabilities which are backed by the author’s expertise. However, Cencora strongly encourages readers to review the references provided with this article and all available information related to the topics mentioned herein and to rely on their own experience and expertise in making decisions related thereto as the article may contain certain marketing statements and does not constitute legal advice.
