Dear readers,
Six months have passed since our last newsletter, and a lot has happened.
In this second issue of the ASCERTAIN newsletter, we are pleased to present an interview with Maria Piggin, founder and chair of the PNH Global Alliance. Maria Piggin talks about the Alliance’s mission and the challenges faced by patients with rare diseases such as paroxysmal nocturnal haemoglobinuria (PNH). Their commitment to patients highlights the urgent need for affordable and sustainable healthcare solutions.
We are also pleased to present our animated video! Discover ASCERTAIN innovative approach to tackling inequalities in access to health technologies. Learn how ASCERTAIN seeks to improve affordability and sustainability while fostering innovation and addressing environmental impacts.
Meet our Advisory Board shines a spotlight on the diverse group of experts leading the ASCERTAIN project. Their collective expertise and insights are invaluable as we work to develop innovative models for accessibility and equity in healthcare.
In our Updates on our work section, we provide a comprehensive overview of the latest developments and milestones of the ASCERTAIN project. From setting the agenda at our consortium meeting to advancing precision medicine and addressing inequalities in access to next-generation sequencing tests, we are making great progress towards our goals.
Learn more about our latest webinar on the fundamentals of drug pricing in Europe. Find out about the processes and policies that influence the pricing of medicines and their impact on patients and healthcare systems.
In the News & Events section you will find a review of ISPOR 2023 and the 6th European CAR T-CELL Meeting. You can also find out which events we will be attending next.
Enjoy reading this newsletter and happy Easter!
Your ASCERTAIN team
ADVOCATING FOR ACCESS: MARIA PIGGIN DISCUSSES THE MISSION OF THE PNH GLOBAL ALLIANCE
What does the PNH Global Alliance do?
Maria Piggin: It is a non-profit organisation that brings together 9 PNH patient groups from 8 different countries across the globe with a shared vision for PNH patients globally to have access to optimal care and treatment, achieved through collectively advocating on behalf of all PNH patients with various stakeholders. We also lobby relevant stakeholders to assist individual patients where we can with access to treatment or trials and provide guidance and support to patients in countries where no patient organisations exist.
Can you share your personal experiences or the experiences of those you represent about the impact of high drug prices in the treatment of PNH?
Maria Piggin: My homeland of New Zealand is one of approximately 80% of countries which does not currently have access to available effective treatments for PNH due to their prohibitive cost. I was lucky that my dual citizenship in the UK meant I could access PNH treatment once this became necessary however this is a privilege most are not afforded. Untreated PNH has a risk of death or serious life-changing complications. Very soon, some PNH patients will be taking a tablet twice a day whilst others continue to be at risk of death without access to any effective treatments.
To what extent do you believe that affordable access to innovative therapies can significantly improve the lives of PNH patients and their families?
Maria Piggin: It is more than a belief, it is a fact. Untreated PNH patients are at high risk of blood clots and other serious life-changing complications. The improved quality of life of patients treated with innovative PNH therapies (of which there are 3 licensed in Europe and 4 licensed in the US not including biosimilars) also expands to the quality of life of their carers and families. It enables both patients and carers to start working (or work more), start studying or take on caring responsibilities for dependants and therefore reduce the burden on the healthcare system and the State to support them.
Why do you participate in ASCERTAIN as an advisory board member?
Maria Piggin: Representation by the PNH Global Alliance on ASCERTAIN was important to bring the perspectives of those with our ultra-rare disease which is in a privileged situation i.e. with a number of licensed innovative treatments (as well as biosimilars) and more in development but which treatments are only available to few patients globally due to the prohibitive cost and no clear solution for this situation to change anytime soon. Therefore, the need for new models of pricing is imperative for the PNH global patient community.
What actions or changes do you think could help improve access to life-saving treatments for rare diseases like PNH?
Maria Piggin: New pricing models are welcome as well as systemic revision of the medicines licensing and health technology assessment (HTA) processes to ensure there is affordable and sustainable access to innovative medicines for patients. I look forward to the outcomes of the ASCERTAIN project.
For more information on PNH Global Alliance please visit www.pnhglobalalliance.org
MEET OUR ADVISORY BOARD
The ASCERTAIN Advisory Board comprises a diverse group of experts from various sectors of the healthcare industry, including patient advocacy, academia, pharmaceutical industry, and policy-making. Their collective expertise and insights contribute to guiding the ASCERTAIN project towards achieving its objectives of developing innovative models for healthcare accessibility and equity. With backgrounds spanning patient advocacy, medical research, healthcare policy, and investment, the Advisory Board members bring a wealth of experience and perspectives to the table. Through collaboration and strategic guidance, they play a crucial role in shaping the direction of the project, ensuring that it remains relevant, impactful, and aligned with the needs of stakeholders across the healthcare ecosystem.
Antonella Cardone emphasizes, “Accessibility and time to access innovative treatments for cancer patients means more than quality of life. It means surviving or not a cancer diagnosis.” Yannis Natsis highlights the urgency, stating, “ASCERTAIN is about one of the elephants in the room: the high prices of some new treatments. It is encouraging that ASCERTAIN will test ways and foster some creative thinking to help better prepare us for what is coming our way.”
Get to know all advisory board members here.
WATCH OUR ANIMATED VIDEO ABOUT THE PROJECT
Discover how ASCERTAIN is tackling inequalities in access to healthcare technologies with our latest animated video and listen to Carin Uyl-de Groot! Learn about the project’s mission to improve affordability and sustainability while promoting innovation and considering environmental impacts.
Click here to watch the video on YouTube.
SETTING THE AGENDA: CONSORTIUM MEETING IN JANUARY 2024
The ASCERTAIN Consortium met online on January 16-17 to advance project work and discuss key findings. Participants included ASCERTAIN’s Advisory Board, European Commission representatives, and various stakeholders. Highlights of the meeting included a keynote by Yannis Natsis on the EU pharmaceutical revision, insights from a fair pricing review, updates on the ACCESS2MEDS Framework, and findings from scoping reviews on pricing methods.
During discussions on pricing methods for new health technologies, participants emphasized the need for fairness and effectiveness. They highlighted the oversight of not considering the cost of capital in pricing equations and suggested integrating it into research and development (R&D) costs. Additionally, there were discussions on capturing the long-term value of curative treatments and the importance of adopting a lifetime approach to pricing models.
The conversation then shifted to cost-effectiveness and budget impact models, with a focus on mandatory trial updates and incorporating additional data under new EU Health Technology Assessment (HTA) regulations. Participants also discussed optimizing pricing models to reduce complexity and increase transparency, emphasizing collaboration and transparency to achieve equitable access and fair pricing worldwide.
Concerns about medical device availability and pricing were addressed, with calls for safety and transparency in product evaluation. Participants agreed on the importance of viewing medicines as global public goods, stressing equitable access and fair pricing. In conclusion, the discussions underscored the multifaceted nature of pricing methods and the importance of ongoing dialogue and collaboration to develop fair, transparent, and effective pricing models for innovative health technologies.
The meeting highlighted the critical need for ongoing dialogue, collaboration, and transparency to ensure fair and effective pricing models for innovative health technologies, ultimately striving for equitable access and improved healthcare outcomes.
We are currently reviewing and analysing the literature on these topics, as well as conducting interviews regarding fair pricing. The initial results will be shared with our Advisory Board in June at the EHA2024 Hybrid Congress!
EXPLORING FAIR PRICING IN HEALTHCARE TECHNOLOGIES
As part of a multi-step approach to developing new pricing models in this Work Package, we reviewed how others have previously defined “fair pricing” for health technologies. We found four core themes that were commonly included in existing definitions of fair pricing for new pharmaceuticals: prices should be affordable to health systems and patients, linked to the costs for developing and producing them, reflect value, and provide companies with appropriate profits. While these core themes were common across existing definitions, we noticed that stakeholder groups, such as health care payers, patient organizations, and industry, among others, emphasized different aspects. Notably, factoring the environmental impacts of health technologies into pricing was not mentioned in any of the definitions.
Another important finding was that none of the definitions focused on fair pricing for medical devices. This lack of definitions suggests that more work is needed to understand how prices for specific devices are derived and perceived, and how they impact on health systems and patient access to these technologies. In ASCERTAIN, we will focus on pricing for a specific type of in-vitro diagnostic devices, next-generation sequencing.
The review findings were presented as a poster at ISPOR Europe in Copenhagen on 14 November 2023 and the abstract was published in the conference supplement in Value in Health.
BASICS OF MEDICINES PRICING IN EUROPE
On 7th of March 2024 Myeloma Patients Europe (MPE) held an introductory webinar on the topic of medicine pricing in Europe. The main speaker was Sabine Vogler, Director of the WHO Collaborating Centre for Pharmaceutical Pricing and Reimbursement Policies and Head of Pharmacoeconomics Department, Austrian National Public Health Institute. The webinar provided foundational knowledge on the basics of drug pricing (with a focus on high-cost branded medicines) and the processes and policies that influence it. Aimed at patients, patient advocates and the general public, the presentation was given in accessible language with clear real-world examples.
The webinar gathered high interest, with 152 registered participants and 82 unique viewers. Questions from the audience focused on pricing models used by European countries and the implications for rare diseases, cooperation among EU Member States and the role of the EU in pricing and reimbursement practices. The feedback received after the webinar was very positive with participants appreciating the clarity of the explanations. The event was live for registered participants but was also recorded and disseminated by MPE, ASCERTAIN and other partners.
The webinar forms part of a wider engagement strategy for ASCERTAIN, designed to inform and involve patients, patient advocates and other relevant stakeholders in the project. The next topic of focus will be a webinar on the EU HTA regulation and the implications for patients across Europe. Please message gro.eporuepm@ativocar if you have any questions!
Click here to watch the webinar on YouTube.
ADVANCING PRECISION MEDICINE: FRAMEWORK DEVELOPMENT AND EXPERT ELICITATION
We are currently working on developing a general framework for WP5. The framework includes building blocks, pilot model analyses, and a quality assurance framework for the three Use Cases: precision cancer medicine, gene/cell therapy, and whole genome sequencing. These will ultimately inform and guide the development of the three global models.
The building blocks and quality assurance framework that underpin the model development processes consist of several components relevant for all three Use Cases, such as data collection and management, standardization of coding practice, verification, and validation.
Pilot case selection and model development are currently underway. For example, we have selected the pilot case for the precision cancer medicine Use Case and are currently building a pilot model for precision cancer medicine with a single-arm trial design.
More specifically, we are evaluating the use of expert elicitation, following the framework suggested by Bojke et al (2022) to inform the relative treatment effect. The analysis is based on a phase 1/2 study LIBRETTO-001, including patients with RET-fusion positive non-small-cell lung cancer (NSCLC) treated with selperatinib (we are also doing a second analysis on RET-mutant thyroid cancer).
With co-funding and collaboration with the public-private network CONNECT and collaboration with Oslo Economics on conducting the interviews with clinicians, we are evaluating expert elicitation as a source of documentation in cost-effectiveness analysis (CEA).
The CEA results from the phase 1/2 study with expert elicitation will be compared and validated with the CEA results based on documentation from the phase 3 studies (LIBRETTO-431 and LIBRETTO-531). The phase 3 study was a randomized controlled trial enabling validation of the expert elicitation. We are currently working on the analyses.
ADDRESSING DISPARITIES IN ACCESS TO NEXT-GENERATION SEQUENCING (NGS)-BASED TESTS
Developing Flexible Models for Pricing, Cost-Effectiveness, and Reimbursement
Considering the relevant differences in pricing, health technology assessment (HTA), and reimbursement of pharmaceuticals and medical devices, including in vitro diagnostic (IVD) medical devices, the ASCERTAIN project aims to develop new flexible models for pricing, cost-effectiveness, and reimbursement that can contribute to the accessibility of different innovative technologies, as well as to health equity.
Identification and Selection Process
In the first year of the ASCERTAIN project, the identification and selection of medical devices applied in oncology (for use in WP5) were provided by targeted searches on relevant organisational websites (e.g., regulatory bodies like the FDA, HTA bodies, notified bodies), scientific literature (systematic reviews and clinical guidelines), and grey literature. This process included the involvement of different stakeholder organisations at the EU level (patient organisations, HTA bodies, healthcare providers, payers, and manufacturers), using online questionnaires or email correspondence, and discussion with clinical experts. The NGS-based tests, specifically the whole group of Targeted panel sequencing tests, were selected as IVD medical devices, including companion diagnostics, in indications related to solid tumors (e.g., colorectal cancer) and hematologic malignancies (e.g., acute leukemia/myelodysplasia).
Assessment and Recognition of Benefits
Currently, an appropriate, fit-for-purpose assessment framework for such tests is lacking. However, different potential benefits of the NGS-based tests are recognized, such as delivering the right treatment for the right patients, delivering benefits to healthcare systems and payers, as well as broader societal impact (e.g., added value of knowing for patients, relatives, and the community; more efficient use of healthcare system resources; helping direct future areas of research).
Challenges in Availability and Reimbursement
In many European countries, there are restrictions on the availability and reimbursement of such tests. In 2021, the uptake of NGS-based tests is highly varied, ranging from 0% in Slovakia to more than 50% in Denmark and the Netherlands. Additionally, in several countries, there is no link between precision medicine and matched biomarker test approval.
Future Directions
With this in mind, ASCERTAIN partners will develop appropriate flexible HTA frameworks and generalizable pricing and reimbursement models that enable country-specific adaptations. These models will reflect the various stakeholder perspectives involved in the adoption of NGS-based tests in the healthcare system.
ENHANCING DATA SECURITY MEASURES
During recent months we have dedicated efforts to collecting data across various tasks and ensuring the security of the infrastructure responsible for hosting and processing this data for the project’s objectives. Numerous calls were conducted with partners to develop distinct data flow diagrams and assist them in correctly completing them.
In addition to these efforts, several meetings were held to initiate the definition of the architecture and technical security measures for the infrastructure crucial for upcoming project developments.
Subsequently, following discussions with the EUR Data Steward and upon request from the EUR ethical committee, an updated (intermediate) version of the Data Management Plan was released in January 2024. This version contains the latest updates relevant to the progress of the project.
ASCERTAIN at the 6th European CAR T-Cell Meeting in Valencia
A catalyst for collaborative advances in cancer immunotherapy.
ASCERTAIN at ISPOR 2023 in Copenhagen
Navigating Challenges in Health Economics and Outcomes Research
EAA SPRING COVENTION
Join partners from ASCERTAIN project the EAA SPRING CONVENTION 2024 in Rotterdam.
18 – 19 April 2024
5TH PPRI CONFERENCE
Ensuring access to affordable medicines through innovative policies
Organisers: WHO Collaborating Centre for Pharmaceutical Pricing and Reimbursement Policies, Pharmacoeconomics Department at the Austrian National Public Health Institute (Gesundheit Österreich GmbH)
25 – 26 April 2024 in Vienna
Don’t miss the latest updates and exciting developments in the world of innovative health technologies. Follow us on LinkedIn and YouTube to stay informed about our mission to enhance affordability and accessibility in healthcare. Join the ASCERTAIN community today and be part of the conversation for a healthier and more equitable future!
