Dear Readers,
The affordability of newly approved innovative health technologies (IHTs) is challenging in many health systems. Health care payers and health care industry across the European Union (EU) have explored different ways of defining payment for new products. ASCERTAIN addresses the need of patients, physicians, payers, regulators, and manufacturers to improve the affordability and accessibility to innovative health technologies (including pharmaceuticals) in Europe.
In this first issue of the ASCERTAIN Newsletter, we are happy to share an interview with Carin Uyl-de Groot, the Project Coordinator of ASCERTAIN. Carin discusses the project’s inception, goals, and its vision to revolutionize patient access to IHTs. She highlights the urgent need to address the delays and limited access to these technologies, which often affect European citizens’ right to health.
In this newsletter, we will also delve into the latest project updates, including the introduction of the ASCERTAIN framework. We will also explore our efforts to develop global models for evaluating clinical and cost-effectiveness and the path we’re paving for dynamic health technology reimbursement. Furthermore, we are excited to share our progress on the ACCESS2MEDS toolbox, a crucial component of our project that will support collaboration, accessibility, and model development within ASCERTAIN.
In the News & Events section, you’ll find information about our upcoming sessions at European School of Haematology Congress and ISPOR EUROPE 2023.
Please enjoy reading our newsletter and learn more about the ASCERTAIN project progress.
Your ASCERTAIN team
DRIVING INNOVATION AND EQUITY IN HEALTHCARE: UNVEILING ASCERTAIN’S VISION TO REVOLUTIONIZE PATIENT ACCESS TO INNOVATIVE HEALTH TECHNOLOGIES ACROSS EUROPE
What inspired the creation of ASCERTAIN, and what are the primary goals you aim to achieve?
Carin Uyl-de Groot: The primary goal is to improve patient access to innovative technologies, including pharmaceuticals, across Europe. I have noticed that there is too much delay from registration to access, and many patients do not have access at all. This conflicts with EU regulations, but so far there is no real solution. Until now, stakeholders do not want to compromise. But if you know that European citizens have a right to health, this right is violated quite often.
How do you envision ASCERTAIN making a positive impact on improving the affordability and accessibility of innovative health technologies in Europe?
Carin Uyl-de Groot: By implementing a combination of strategies to improve entrepreneurship, sustainability, and accessibility to innovative health technologies (IHTs). Governments, (pharmaceutical) companies and other stakeholders can create an ecosystem that promotes and incentivizes innovation in health technologies, ultimately leading to improved healthcare outcomes and patient well-being.
Could you tell us about the key stakeholders involved in ASCERTAIN and how their expertise contributes to the project’s success?
Carin Uyl-de Groot: Collaboration is key in this project. As all stakeholders have their own interest, collaboration and communication are very important. I am glad that we have great partners who are willing to think outside the box and really want to improve the situation. Further, we have an advisory board consisting of all relevant stakeholders coming from Western- and Eastern European member states.
What are the major challenges you anticipate during the project’s implementation, and how do you plan to address them?
Carin Uyl-de Groot: Major challenges are to get everyone on the same page. I will address these challenges by organizing many meetings and listening carefully to the different interests. Achieving progress and results will be an ongoing process that requires patience, empathy, and a willingness to collaborate and find common ground despite differences. One common ground is accelerating patient access to innovative interventions and improving cancer care across EU member states; no stakeholder is against this. How to achieve this will take time, but I am convinced that we will manage these differences and will succeed.
What are the initial steps you’ve taken to launch ASCERTAIN, and how do you plan to engage with the broader healthcare community and policymakers?
Carin Uyl-de Groot: We have an awesome group with all the necessary expertise. The initial steps were to get started by having a Kick-off meeting in January. This meeting was both an internal meeting, to get to know one another better, and an external meeting, with invited stakeholders. In August, we submitted the first draft of the ACCESS2MEDS framework to the EU. This framework is called the ACCESS2MEDS framework as we anticipated that it will be used for a much longer time than the 4-years of the ASCERTAIN project. In this respect, ACCESS2MEDS means access to both medicines and medical devices. Later versions of the framework will be tested by several stakeholders, including policymakers.
Further, the Advisory Board has been installed, and the board will be asked regularly to reflect and give their input on our work, including the framework. Also, I have contacted members of the European Network for Health Technology Assessment (EUnetHTA) to explore how we can work together. In January 2025, the first joint clinical assessment of oncology drugs and Advanced Therapy Medicinal Products (ATMPs) will be performed. It would be very worthwhile to examine how ASCERTAIN can help and bring joint assessments a step further by offering pricing, HTA, and reimbursement models to countries as well.
We are a consortium of ten partners from seven countries (Austria, Belgium, The Netherlands, Slovakia, Norway, Germany, and Italy) representing academia, industry, and patient organisations collaborating together on this project. In January, we had our first in-person meeting of the ASCERTAIN project in Rotterdam! All consortium members gathered to kick off this HORIZON-funded initiative with the goal of enhancing access to innovative health technologies in Europe.
INTRODUCING THE ASCERTAIN (ACCESS2MEDS) FRAMEWORK
The ASCERTAIN (ACCESS2MEDS) framework is a set of principles and definitions, including new methods to develop models for value-based pricing, cost-effectiveness analyses, budget impact calculations, and reimbursement and payment schemes. These are underpinned by a set of core values and objectives, such as access to good healthcare, equity, and (financial and environmental) sustainability.
The framework consists of an iterative process that takes into account vertical stakeholder consultation, changes to the pharmaceutical and medical devices sectors, variations in EU market conditions, and the state of the art of pricing policies, health technology assessment (HTA) frameworks, and reimbursement models. Moreover, it aims to reconcile and balance the underlying core values and objectives with the European regulatory framework policies, namely the New European Innovation Agenda, the Pharmaceutical Strategy for Europe, the EU Pharmaceutical Legislation reform, and, in particular, the EU HTA Regulation.
The first version of the ACCESS2MEDS framework was concluded in August, after which stakeholder consultations will follow to ensure relevance and adequacy and to collect requirements for subsequent versions. Additionally, and as part of the iterative process, the ongoing state-of-the-art mapping efforts include country surveys, literature reviews, as well as a health technology selection process for a case study on medical devices and in vitro diagnostics, respectively.
REDEFINING HEALTHCARE AFFORDABILITY
In recent years, we have seen new health technologies (particularly medicines) entering the market with unprecedented price tags. This has created challenges for health systems aiming to provide fair access to healthcare for their patients. In ASCERTAIN, we focus on developing new approaches for pricing innovative technologies. We are trying to understand what factors are required to come up with a price that balances the cost of developing these technologies with sufficient reward for needed innovation.
Learning from the experience of our partner AIM and their European Fair Pricing Calculator, we are aiming to come up with new approaches to determine an appropriate price. As initial steps, we are currently reviewing the literature and preparing interviews with stakeholders across Europe. We have also been collecting definitions for fair prices of new health technologies, as published by researchers and various stakeholder organizations (e.g., this definition from the WHO Fair Pricing Forum) and are currently reviewing these to inform our next steps.
GLOBAL MODELS TO EVALUATE CLINICAL AND COST-EFFECTIVENESS, AND BUDGET IMPACT
In ASCERTAIN, we will develop open-access models for three use cases (precision cancer medicine, gene and cell therapy, and diagnostics), each representing unique aspects of innovative health technologies. Two primary challenges must be addressed:
- estimating clinical effectiveness when data are derived from non-randomized trials and
- managing uncertainty linked to documentation and small study samples.
The ACCESS2MEDS framework, described above, will underpin all aspects of the work involving global models and data collection.
To craft global models aimed at improving access to innovative health technologies in Europe, we must explore the connection between these models and local conditions. Consequently, we are conducting a literature review to describe and compare national guidelines for health technology assessments (HTA). This helps identify factors to consider in global models, such as perspective, discount rate, and comparator. Additionally, we are conducting a literature review to identify instances where cost-effectiveness models utilised input from non-randomized trials to determine clinical effectiveness.
PAVING THE WAY FOR DYNAMIC HEALTH TECHNOLOGY REIMBURSEMENT
The development of dynamic and cyclic reimbursement procedures is an integral part of the ACCESS2MEDS framework. The current reimbursement systems have several pitfalls, including affordability and sustainability issues, inequitable access, and delays in access and time-to-market. Further, the evidence of a drug’s effectiveness and safety is sometimes uncertain, leading to uncertainties in the reimbursement process. These procedures will introduce fresh reimbursement models and payment schemes for costly health technologies, along with innovative threshold definitions. An evaluation will assess their effects on equitable access, payer risk, and the values and preferences of EU patients and citizens. This evaluation will take a broad perspective, involving academia, patients, professional organizations, and industry.
To set new thresholds, we began with a literature search. We review published articles about cost-effectiveness threshold values, multiple thresholds, and alternatives to QALY (Quality-Adjusted Life Years). Another search is ongoing to design preference elicitation studies in multiple countries. These studies will focus on the independent impact of disease severity, age, and rarity on treatment preferences.
ACCESS2MEDS TOOLBOX: ADVANCING COLLABORATION AND ACCESSIBILITY IN ASCERTAIN
The ACCESS2MEDS toolbox will be a web-based platform designed to host, manage, and securely provide access to ASCERTAIN models for all project stakeholders and end-users. Our initial focus on understanding end-user needs and defining the ACCESS2MEDS policy support tool has paved the way for developing the comprehensive ACCESS2MEDS toolbox. We are developing this toolbox with all project partners, incorporating their stakeholders’ and end-users’ perspectives, with the aim to complete it by the end of 2023.
From our preliminary end-user needs analysis, we’ve identified the initial requirements for the ACCESS2MEDS Toolbox, including:
- A secure web-based system for managing ASCERTAIN models.
- Interfaces for online collaboration.
- Improved model accessibility through user-friendly web interfaces.
- The ability to integrate with other tools and applications.
- User-friendly visualisation interfaces for ASCERTAIN models, eliminating the need for advanced IT skills.
DATA MANAGEMENT AND ETHICAL EVALUATION IN ASCERTAIN
During these initial months, we have overseen the collection of data from individual partners and the development of the first version of the data management plan. In the upcoming months, we will engage in ongoing monitoring of the collected data, commence the evaluation of ethical and legal aspects in collaboration with our partners, and generate various documents, including the data security architecture, the Risk Assessment, and a compliance interim report. Additionally, we will produce Data Processing Agreements, Joint Controllership agreements, and other documents related to personal data in alignment with the agreements and requirements of the Consortium’s partners.
ASCERTAIN PROJECT AT EHA2023: IMPROVING ACCESS TO INNOVATIVE HEALTH TECHNOLOGIES IN EUROPE
Prof. Carin Uyl-de Groot presents ASCERTAIN, Focusing on Affordable CAR-T Cell Therapies and Cancer Care in Eastern Europe.
EUROPEAN SCHOOL OF HAEMATOLOGY CONGRESS
- 2nd How I Manage: CAR-T Therapies and Bispecific Antibodies for my patients
- Roundtable: Affordability and access to CAR-T cell and bispecific antibody treatment
- On Friday, 29 September 2023
- With Frederick Thielen
ISPOR EUROPE 2023
- Our session: Horizon Europe Project Ascertain: Supporting a Sustainable and Transparent Legal EU HTA Framework, Accessibility of Innovative Technologies and Health Equity
- On Monday, 13 November 2023 | 17:00 – 18:00
- With Carin Uyl-de Groot, Mirjana Huić, Isabelle Durand-Zaleski and Nicolas Xander
- Our session: Unveiling the EU-HTA Regulation: A Deep Dive Into the Joint Clinical Assessment (JCA)’
- On Sunday, 12 November 2023 | 13:00- 17:00
- With Maureen Rutten-van Mölken and Frederick Thielen
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