Tag: insights
Challenges and Concerns Raised by EFPIA on Joint Clinical Assessments
In the healthcare regulation sphere, proposed EU-level joint clinical assessments (JCAs) have sparked debate in the pharmaceutical industry. A recent EFPIA report highlights concerns about the rigidity of the guidelines, particularly in oncology, and suggests revisions to ensure a more efficient and adaptable evaluation process that accommodates diverse stakeholder needs across Europe.
In the realm of healthcare regulation, the proposed methods for conducting EU-level joint clinical assessments (JCAs) have stirred significant debate and concern within the pharmaceutical industry. A recent report commissioned by the European Federation of Pharmaceutical Industries and Associations (EFPIA) sheds light on the potential pitfalls and challenges companies may face under the forthcoming Health Technology Assessment Regulation ((EU) 2021/2282).
The guidelines put forth for implementing these JCAs, as highlighted by EFPIA, lack the requisite flexibility and pragmatism necessary to navigate the complexities of rapidly evolving disease areas, particularly oncology. A recent study, published on March 28, scrutinized the methodology proposed by EUnetHTA21 by applying it to three anonymized oncology products with EU regulatory approval.
One of the key findings of the report reveals that the scoping process outlined in the proposed methodology results in an overwhelming number of Population, Intervention, Comparator, and Outcome (PICO) elements that companies must address. For instance, one drug subjected to analysis had a staggering 57 PICOs initially, even after consolidation, the number remained at 23. This imposes a significant burden on companies to gather and present data for each PICO, often necessitating complex analytical methods or real-world evidence from disparate sources.
Furthermore, the report underscores the challenge posed by the wide range of comparator therapies mandated by the proposed guidelines. In the context of oncology, where treatment options vary widely across EU member states, identifying appropriate comparators becomes particularly daunting. This issue is compounded by the inclusion of off-label drug use as comparators, a practice that diverges from regulatory standards and complicates the assessment process.
JCA is not the cause of differing and partly even conflicting evidence requirements across Europe. These differences existed for decades. So far, industry coped with these requirements by planning their trials for key countries (most notably the US and Japan) and then trying to meet the evidence requirements of all other countries one after another according to their position in the (European) launch sequence. What JCA changes, is that all EU requirements have to be accounted for at once within a very restricted timeframe. This puts the timeline of life-saving innovations in jeopardy and should raise serious concerns among industry, patients, physicians, regulators as well as politicians. Doing this in oncology – a field of extreme unmet need where lives of patients are at stake and where treatment options as well as evidence requirements differ strongly across the EU – seems to be a questionable choice.
Another critical concern raised by EFPIA relates to the requirement for mature Overall Survival (OS) data, which may not be available at the time of regulatory approval for many oncology submissions. The report suggests a reconsideration of this requirement, advocating for the inclusion of intermediate outcomes such as Progression-Free Survival (PFS) in JCAs. Additionally, it highlights discrepancies between the proposed guidelines and the endpoints commonly used in national HTA assessments, further complicating the evaluation process.
EFPIA’s apprehensions regarding the JCA process echo broader industry concerns, with organizations like the Alliance for Regenerative Medicine (ARM) also cautioning against potential impediments to the implementation of the HTA regulation at a national level.
As discussions surrounding the implementation of EU-level JCAs continue, it is evident that addressing the outlined challenges and refining the proposed guidelines will be crucial to ensuring a streamlined and effective assessment process that aligns with the diverse needs of stakeholders across the European healthcare landscape.
Joint Clinical Assessment (EU HTA) Explained
A Comprehensive Exploration of Competitive Intelligence, Knowledge-Sharing, and Strategic Optimization in the Evolving Landscape of EU Health Technology Assessments.
The first companies undergoing the JCA process in 2025 will indeed be pioneers, blazing a trail for others to follow. These early adopters will be the first to gain practical experience with the JCA, providing valuable insights into the challenges and opportunities associated with the process. As they navigate the complexities of the JCA, these companies will inevitably develop strategies and best practices that contribute to their success.
However, as you rightly point out, not all companies will be willing to share their experiences openly. In a competitive market environment, companies may view their knowledge of the JCA process as a valuable asset and guard it closely as a trade secret. This creates a challenge for other companies seeking to understand the key success factors and optimize their approach to the JCA.
This is where competitive intelligence plays a crucial role. Competitive intelligence involves gathering, analysing, and interpreting information about competitors, market trends, and industry developments to gain a competitive advantage. In the context of the JCA, competitive intelligence can help companies learn from the experiences of others and identify strategies and tactics that contribute to success.
By skillfully gathering information from multiple sources, including public records, regulatory filings, industry publications, and market research reports, companies can gain insights into the approaches and outcomes of their competitors undergoing the JCA process. This information can help companies identify patterns, trends, and best practices that inform their own approach to the JCA.
Moreover, competitive intelligence enables companies to benchmark their performance against industry peers and identify areas for improvement. By comparing their approach to the JCA with that of their competitors, companies can identify gaps, weaknesses, and opportunities for enhancement. This allows companies to refine their strategies, optimize their resources, and increase their chances of success in the JCA process.
In addition to competitive intelligence, collaboration and knowledge-sharing among industry stakeholders are essential for navigating the complexities of the JCA. Industry associations, trade groups, and professional networks can serve as valuable forums for sharing insights, exchanging best practices, and collectively addressing challenges associated with the JCA. By fostering a culture of collaboration and knowledge-sharing, stakeholders can collectively advance their understanding of the JCA and optimize their approach to market access within the EU.
In conclusion, the introduction of the JCA represents a paradigm shift in the evaluation of medicines within the EU. As companies embark on this new journey, they face significant challenges and uncertainties. However, by leveraging competitive intelligence and fostering collaboration among industry stakeholders, companies can gain valuable insights, identify key success factors, and optimize their approach to the JCA. In doing so, they can enhance their chances of securing timely access to innovative therapies and improving patient outcomes across the EU.
Sources:
- Regulation (EU) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on health technology assessment and amending Directive 2011/24/EU. EUR-Lex. https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX:32021R2282. Retrieved Jan 30 2024.
- HTAR (HTA-Regulation): Implementation in Austria. Austrian Institute for Health Technology Assessment GmbH. https://aihta.at/page/htar-hta-verordnung-implementierung-in-oesterreich/en. Retrieved Jan 31 2024.
- D4.2 Practical Guideline on Scoping Process. EUnetHTA. https://www.eunethta.eu/wp-content/uploads/2022/09/EUnetHTA-21-D4.2-practical-guideline-on-scoping-process-v1.0.pdf. Retrieved Feb 14 2024.
- D5.1 Submission Dossier Guidance. EUnetHTA. https://www.eunethta.eu/wp-content/uploads/2022/12/EUnetHTA-21-D5.1-Submission-Dossier-Guidance-v1.0.pdf. Retrieved Feb 15, 2024.
- EUnetHTA 21 – Stakeholder Kick Off Meeting. EUnetHTA. https://www.eunethta.eu/wp-content/uploads/2021/12/EUnetHTA-21-Stakeholder-Meeting-03.12.-FOR-WEBSITE.pdf.
- Joint HTA Work. EUnetHTA. https://www.eunethta.eu/jointhtawork/. Retrieved Jan 30 2024
- EU Regulation on Health Technology Assessment (HTA). MedTech Europe. https://www.medtecheurope.org/news-and-events/press/eu-regulation-on-health-technology-assessment-hta/. Retrieved Feb 14 2024
- Proposals On EU-Wide Joint Clinical Assessments Fall Short, Warn German Industry Groups. Pink Sheet Pharma Intelligence. https://pink.pharmaintelligence.informa.com/PS147635/Proposals-On-EUWide-Joint-Clinical-Assessments-Fall-Short-Warn-German-Industry-Groups. Retrieved Feb 12 2024