Dear readers,
At the 4th ASCERTAIN Consortium meeting, held during the European Hematology Association (EHA) Congress in Madrid, we reached an important milestone by presenting 1.5 years of progress to our international advisory board. Our discussions centered around key findings from our scoping reviews and stakeholder inputs on health technology assessments (HTA), pricing, reimbursement, and policy considerations that impact patient access to innovative therapies.
Our consortium delved deeply into the critical issue of fair pricing, exploring innovative valuation methods, especially in single-arm trials. We discussed incorporating the cost of capital and failure rates into R&D expenses and emphasized the need for transparent, adaptable pricing models. The meeting also highlighted the importance of new cost-effectiveness and budget impact models, particularly in light of the evolving EU HTA regulation.
With our policy decision-making tool, ASCERTAIN aims to promote innovation, enhance patient access, and foster sustainable healthcare planning. By testing these tools in areas like precision cancer medicine and CAR-T cell therapies, we plan to provide user-friendly, cloud-based solutions that adhere to open science principles. As we move forward, collaboration and transparency will continue to guide us in advancing equitable access to health technologies worldwide.
In this newsletter, you will find more details on our progress.
Enjoy reading this newsletter!
Your ASCERTAIN team
CONNECTING STAKEHOLDERS FOR BETTER CANCER CARE
What motivated you to start Inspire2Live, and what are its core goals?
Peter: Inspire2Live was founded in 2010 with the aim of connecting patients, researchers, and clinicians to benefit cancer patients and improve the quality of life for patients and their loved ones globally.
How do you connect patients, researchers, and clinicians to foster collaboration and improve cancer treatment and care?
Peter: We are a global network organization with approximately 100 advocates representing all stakeholders in the medical-industrial complex: patients, researchers, clinicians, industry, government, and regulators. We select them based on the topic and their expertise in the initiatives we want to start. These initiatives always contribute to the global patient community and their loved ones. We’re now represented in 42 countries, soon to be 43 with the addition of the Filipinos, including Kenya, Ghana, Nigeria, Argentina, and Brazil.
What strategies do you find most effective in advocating for patients within the healthcare system?
Peter: Cooperation in an open, fair, and equal atmosphere is definitely the most effective. We’re all equal and critical in our conversations. We are all convinced that science is the foundation, and emotions drive us with persistence.
What is your role as an ethical advisor in ASCERTAIN?
Peter: I want to contribute with the attitude: ‘If it’s about us, not without us.’ We are dealing with the interests and risks that patients face. In that situation, it is unethical not to work with patients and not to ask for their opinions. It is unethical when decisions are made without patients. I want to emphasize that and, of course, motivate that.
How do you envision the future of patient advocacy and its impact on healthcare innovation through projects like ASCERTAIN?
Peter: Advocacy, in cooperation with other stakeholders, will drive progress and make a difference. If we truly want healthcare to be patient-focused, we must work with patients and involve them in the decision-making process. ASCERTAIN is important in this process, and in my experience, it works. I sense an open, fair, and equal atmosphere in the project. That will make the difference.
For more information on Inspire2Live please visit www.inspire2live.org
CONSORTIUM MEETING IN MADRID
Throughout three days from June 12th to 14th, the ASCERTAIN Consortium convened in Madrid to advance its project goals and engage with stakeholders. The Consortium Meeting took place during the EHA Congress, which attracted over 18,000 participants from across the healthcare value chain, enhancing visibility and networking opportunities for ASCERTAIN members.
The first day of the meeting focused on exchanging knowledge and incorporating feedback from the Advisory Board members, the Ethics Advisor, and the coordinator of the Horizon Europe project, HI-PRIX. The group discussed key developments in the pharmaceutical and medical device sectors, as well as findings from scoping reviews related to pricing, cost-effectiveness, and reimbursement of innovative health technologies. The participants also explored the development of fair pricing models and structured expert elicitation in cost-effectiveness analysis.
On the second day, the consortium held work package presentations and workshops to further plan project activities. The third day featured the participation of the consortium in the EHA-Patient Joint Symposium at the EHA Congress, which included two panel discussions on unmet medical needs and fair pricing of new medicines in haematology. Consortium and Advisory Board members, including Project Coordinator Carin Uyl-de Groot, Frank Leebeek (EHA), Maximilian Salcher-Konrad (GOEG), and Maria Piggin (PNH Global Alliance), served as panellists in the second panel.
Highlighting the importance of collaboration between different initiatives, Carmen Sanges, member of the Advisory Board and part of the coordination team of the T2EVOLVE Horizon Europe Project said: “The T2EVOLVE consortium is primarily focused on accelerating the development and accessibility of CAR-T therapy across Europe. What I’d really like to see from ASCERTAIN is a synergistic approach in HTA and reimbursement schemes, particularly in collaboration with T2EVOLVE and others. Now is the time for collaboration across Europe, as we aim to harmonise efforts.”
Photo: OptiMedis
CHECK OUT OUR NEW PROJECT BROCHURE
In this brochure, we highlight ASCERTAIN’s methodology for creating sustainable health technologies and its impact on key stakeholders in healthcare. Dive into the innovative approaches we are taking to revolutionize healthcare access and outcomes.
Click here to read the brochure
EXPLORING PERSPECTIVES ON FAIR PRICING OF PHARMACEUTICALS
Building on efforts reviewing the literature on pricing models for new health technologies over the past year, WP4 dove deeper into understanding the key concepts of fair pricing for pharmaceuticals. Over the past couple of months, the WP4 team conducted a series of expert interviews with payers, health care professionals, patient experts, HTA bodies, researchers, industry, and investors from across Europe. These interviews provided valuable insights into how different stakeholders perceive fair pricing, highlighting the diverse considerations to balance innovation and affordable patient access. Additionally, we consulted with the ASCERTAIN advisory board to identify key aspects of fair pricing that should be prioritised in our research. This consultation was instrumental in refining our approach and ensuring that our work remains relevant for the needs of decision makers.
Findings from literature reviews as well as early findings from expert interviews were shared and discussed at the ASCERTAIN consortium meeting in June, fostering a collaborative environment for further exploration and refinement of our ideas. Moreover, we had the opportunity to present the results of our review on fair pricing at two scientific conferences: the EHA Congress 2024 in Madrid and EuHEA 2024 in Vienna. These presentations not only showcased our progress but also facilitated valuable feedback and discussions with the wider scientific community, helping to shape the future direction of our research.
INSIGHTS FROM SITE VISITS IN NORWAY
Stakeholder feedback from the CONNECT group and the Norwegian Medical Products Agency
On April 12th, PhD student Yansi Wu at the University of Oslo (ASCERTAIN Work Package 5) presented initial findings from our Structured Expert Elicitation (SEE) project to the CONNECT group. CONNECT is a public-private consortium aimed at driving the implementations of precision cancer medicine (PCM) by addressing key obstacles (e.g., single-arm trials) and piloting novel solutions (e.g., SEE). This meeting unites representative stakeholders from the Norwegian university hospitals, pharmaceutical and technology companies (Association of Pharmaceutical Industry in Norway), patient organizations (the Norwegian Cancer Society), and the Norwegian HTA agency (the Norwegian Medical Products Agency).The study was partly funded by CONNECT and is a collaboration with Oslo Economics.
During this meeting, Yansi shared findings from ASCERTAIN’s exploration into the use ofSEE in evaluating the cost-effectiveness of selpercatinib for non-small cell lung cancer (NSCLC) patients in Norway. Given an increasing reliance on single-arm trials within PCM, SEE holds potential in offering alternative methodologies of effectiveness evaluation, an endeavor that could significantly impact decision-making processes within PCM. The presentation focused on SEE as a viable tool in the pilot case of selpercatinb for NSCLC and its potential to aid healthcare economic evaluations.
We received invaluable feedback and suggestions from the patient representatives, the industry, and the HTA agency. The patient organizations were concerned about the transparency and credibility of SEE. They were particularly interested in understanding which experts contribute to our elicitations, how these individuals are selected, and their levels of expertise. This insight encourages us to ensure that our SEE framework is both open and structured to earn trust and support from patient groups eagerly awaiting new PCMs.
Similarly, the industry and the HTA agency showed great interest in the structural integrity and validity of the model we presented. They sought clarity on the conclusions drawn from our project and were curious about SEE’s potential role in real-world applications. Their perspective is crucial as they prioritize tangible outputs that can influence healthcare decision-makings.
Overall, aligned with the overarching goal of the ASCERTAIN project, the CONNECT meeting was a cornerstone event for gaining direct input from diverse stakeholders. It offered us diverse perspectives on how to tailor further development of our project to meet the needs and expectations from the industries, HTA agencies, and patent groups.
Decision-makers, mingling and productive discussions – our site visit to DMP
Stakeholder engagement visit to the Norwegian Medical Products Agency on May 2nd, 2024
ASCERTAIN aims to meet the needs of patients, physicians, manufacturers, regulators, and payers. A recent site visit to the Norwegian Medical Products Agency (DMP – Direktoratet for medisinske produkter) provided a great opportunity for the Oslo team to exchange knowledge with health authorities and decision-making stakeholders on their needs and perspectives on our project.
The afternoon started with mingling followed by an introductory round of all participants from DMP, the Norwegian Hospital Procurement Trust (NHPT), and the ASCERTAIN team from the University of Oslo. Afterwards, DMP and NHPT presented their organizational structure and gave a brief spotlight on some of their recent work with clinical subgroups in assessments. Eline Aas then gave a general introduction to the ASCERTAIN project and to the specific tasks conducted by the Oslo team. She introduced the concept of use cases, which was then illustrated in presentations by two of ASCERTAIN’s PhD students in Oslo. First, Pia Henkel presented her project on next-generation sequencing-based diagnostics in oncology. Subsequently, Yansi Wu presented his project on structured expert elicitation and single-arm trials applied to non-small cell lung cancer. Especially the timing of the expert elicitation assessment sparked a lively discussion on the challenges related to single-arm trials in health technology assessment. Our presentations were rounded off by Emily Burger, who introduced our quality assurance framework for ASCERTAIN cost-effectiveness modeling. Based on previously sent-out questions, the audience then shared their thoughts on the needs and challenges of assessing innovative health technologies, and on the concept of global models.
Our two main takeaways from this discussion are as follows. First, improving access, one of ASCERTAIN’s main objectives, was not always understood as access to products, but also as access to high-quality decisions. Second, the idea of global models was perceived positively, especially when enabling decision-makers to work with data inputs rather than finished models. Overall, we received both critical and encouraging feedback on our work. The productive discussions helped us to better understand the perspective of healthcare decision-makers and payers involved in health technology assessment. As such, we aim to conduct more of these visits and discussions with decision-makers in other countries, and continuously implement their insights into our work.
Photo: Emily Burger
DEALING WITH DATA MANAGEMENT AND COMPLIANCE IN ASCERTAIN: ETHICAL AND SECURE DATA USE IN HEALTH RESEARCH
Effective data management is a cornerstone of modern health research, especially when addressing privacy and compliance with ethical standards. In the digital health ecosystem, where data flows rapidly and innovations are constantly emerging, ASCERTAIN is committed to ensuring that data handling is both ethical and secure. Our mission to improve the affordability and accessibility of innovative health technologies in Europe is underpinned by rigorous data management practices that adhere to EU Ethics and Data Protection guidelines, including the GDPR (General Data Protection Regulation), which harmonizes data privacy laws across Europe.
The Importance of Ethical Data Management
Ethical data management is not just a regulatory requirement but a moral obligation. It ensures that patients’ sensitive health information is treated with the utmost respect and confidentiality. Within ASCERTAIN, we prioritize the dignity and rights of individuals by implementing strict protocols for data collection, storage, and analysis. These protocols are designed to safeguard personal data, ensuring that it is used solely for the intended research purposes and that individuals’ privacy is never compromised.
Adhering to EU Data Protection Regulation
The GDPR, officially known as REGULATION (EU) 2016/679 of the European Parliament and of the Council of 27 April 2016 on the protection of natural persons with regard to the processing of personal data and on the free movement, has been applicable across all EU member states since May 25, 2018, and sets the standard for data protection in Europe. At ASCERTAIN, compliance with GDPR is integral to our operations. We ensure that all personal data is processed lawfully, transparently, and for a specific purpose. Consent from data subjects is obtained whenever required, and they are informed about how their data will be used, stored, and shared.
Our legal consortium partners provide continuous assistance to ensure that the collection and further processing of personal data for the purposes of the research and innovation action remain at all times compliant with the provisions of the GDPR and applicable national data protection laws, especially with regard to data storage and transmission, automated processing, de-identification methods, access restrictions, and technical and organizational security measures.
Data Sources:
- Interviews and questionnaires
- Cancer registries
- Medical records
- Literature reviews
To protect individuals’ privacy, data used in the project is de-identified and aggregated.
Ensuring Secure Data Sharing and Analysis
Data sharing is vital for advancing health research but must be done securely. ASCERTAIN employs state-of-the-art encryption methods to protect data during transmission and storage. Our data management systems are designed with multiple layers of security to prevent unauthorized access and ensure data integrity.
When sharing data with research partners or stakeholders, we use secure channels and ensure that all parties adhere to the same stringent data protection standards. Data sharing agreements clearly outline the responsibilities of each party and the protective measures to be taken.
Fostering a Culture of Responsibility
Beyond technical measures, fostering a culture of responsibility among the team members is crucial. This ensures that everyone involved in the project understands their role in maintaining data security and upholding ethical standards.
Conclusion
In the fast-evolving digital health ecosystem, the ASCERTAIN project is committed to navigating the complexities of data management and compliance with diligence. By adhering to EU Ethics and Data Protection guidelines, implementing robust security measures, and fostering a culture of responsibility, we aim to ensure that health data is managed ethically and securely. This not only builds trust with patients and stakeholders but also paves the way for groundbreaking advancements in health technologies that are both accessible and affordable.
INITIATING DEVELOPMENT OF THE ASCERTAIN POLICY SUPPORT TOOL: A PATH TOWARDS INCLUSIVE AND EFFECTIVE HEALTHCARE INNOVATIONS
The ASCERTAIN project stands at the forefront of addressing critical healthcare challenges in Europe. By focusing on affordability, accessibility, and sustainability of innovative health technologies, ASCERTAIN seeks to create a balanced ecosystem that benefits patients, healthcare providers, payers, regulators, and manufacturers alike. One of the pivotal components of this project is the development of the ASCERTAIN Policy Support Tool (PST), which aims to revolutionize healthcare decision-making processes through advanced data analytics, data modelling, and user-friendly interfaces.
Setting the Stage for Development
Our journey towards creating the ASCERTAIN Policy Support Tool begins with a comprehensive requirements analysis phase. This phase is crucial as it ensures that the tool will meet the diverse needs of all stakeholders involved. Stakeholder analysis and user story development are integral parts of this phase, capturing the goals, desired outcomes, and expectations from various user groups. In our requirement analysis process, we examine several critical components to ensure a comprehensive understanding of the project. We start by assessing the functionality and features, such as login systems and data input forms, followed by evaluating the user interface (UI) and user experience (UX) design to ensure a seamless and accessible user journey. Our review includes the technological stack, focusing on both front-end and back-end technologies, and we scrutinize security measures like encryption and authentication (where required). Performance and scalability are tested to ensure the website handles traffic efficiently. Additionally, we check for compliance with industry standards such as GDPR regulations. During this initial analysis phase, we gather valuable feedback from partners within the consortium to refine our approach. Later stages and beta releases will involve external evaluators to further enhance the project’s effectiveness and user satisfaction.
Fast Prototyping for Visual Understanding
Adopting a fast prototyping principle, we aim to create visual representations of our progress and gather feedback continuously. This approach will help us refine the tool iteratively, ensuring it aligns with user expectations and technical feasibility. The first Use Case we focus on is the development of the Fair Pricing Calculator to help stakeholders calculate a fair price for medicines, considering factors like R&D costs, market competition, production costs, and therapeutic value. It will enable users to estimate medicine prices and compare them to current or negotiated prices. The tool will focus on transparency in R&D costs, innovation, and pricing mechanisms, and include options for adjusting prices by country based on purchasing power.
A Collaborative Effort
The development of the ASCERTAIN tool is a collaborative effort. Our multidisciplinary consortium, complemented by an international advisory board, will co-create the policy-supporting tools. This collaborative approach ensures that the tools developed are relevant, effective, and practical for real-world applications.
Future Steps
As we move forward, the next steps include the iterative development of the tool’s frontend and backend, rigorous evaluation of the models and functionalities, and consecutive refinement based on user feedback. The ultimate goal is to create a publicly accessible, web-based ASCERTAIN PST that supports data storage, visualization, and interactive analysis, thereby empowering stakeholders to make informed and effective healthcare decisions.
By meticulously analyzing stakeholder requirements and leveraging advanced technologies, we are on the path to creating a tool that will significantly improve decision-making in healthcare policy and practice. Stay tuned as we continue to make strides in this exciting journey towards a more affordable, accessible, and sustainable healthcare system.
Image: Nuromedia
European Public Health Week: GÖG-Colloquium
Greener pharmaceuticals: policies for an environmentally sustainable pharmaceutical system.
Erasmus University Rotterdam Interviews Carin Uyl-de Groot on ASCERTAIN
“Everyone is entitled to the best treatment, no matter where you live”
Presentation of AIM’s Fair Price Model to the Belgian Parliament
Solidaris closes its petition for fair drug prices with more than 50,000 signatures.
EAA Spring Convention: Discussions on EU Market Access
The EAA Spring Convention, themed ‘Finding the Right Balance,’ was held at Erasmus Universiteit Rotterdam.
ISPOR Europe 2024
Join global healthcare leaders as they convene at ISPOR Europe 2024, the leading global conference in Europe for Health Economics and Outcomes Research (HEOR), 17 -20 November, for discussion and dissemination of the latest trends in healthcare. This must-attend event provides you with dedicated opportunities to network with your peers, HEOR experts, and thought leaders and to discuss with a global audience how we establish, incentivize, and share value sustainable for health systems, patients, and technology developers.
17 – 20 November 2024
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