Search

Introduction: Meaningful patient involvement in health technology assessment (HTA) is essential in ensuring that the interests of the affected patient population, their families, and the general public are accurately reflected in coverage and reimbursement decisions. Central and Eastern European (CEE) countries are generally at less advanced stages of implementing HTA, which is particularly true for patient involvement activities. As part of the Horizon2020 HTx project, this research aimed to form recommendations for critical barriers to patient involvement in HTA in CEE countries., Methods: Built on previous research findings on potential barriers, a prioritisation survey was conducted online with CEE stakeholders. Recommendations for prioritised barriers were formed through a face-to-face workshop by CEE stakeholders and HTx experts., Results: A total of 105 stakeholders from 13 CEE countries completed the prioritisation survey and identified 12 of the 22 potential barriers as highly important. The workshop had 36 participants representing 9 CEE countries, and 5 Western European countries coming together to discuss solutions in order to form recommendations based on best practices, real-life experience, and transferability aspects. Stakeholder groups involved in both phases included HTA organisation representatives, payers, patients, caregivers, patient organisation representatives, patient experts, health care providers, academic and non-academic researchers, health care consultants and health technology manufacturers/providers. As a result, 12 recommendations were formed specified to the CEE region's context, but potentially useful for a broader geographic audience., Conclusion: In this paper, we present 12 recommendations for meaningful, systematic, and sustainable patient involvement in HTA in CEE countries. Our hope is that engaging more than a hundred CEE stakeholders in the study helped to spread awareness of the importance and potential of patient involvement and that the resulting recommendations provide tangible steps for the way forward. Future studies shall focus on country-specific case studies of the implemented recommendations., Competing Interests: Patient involvement should be open to all and non-discriminative on the grounds of previous experience and presumed support time needed. There are multiple initiatives locally and internationally aiming to ease patient recruitment with education, coordination and/or databases. We recommend organisations leading these initiatives to come together and join forces on the base of commonly agreed principles. We also recommend local HTA/payer organisations to set up their open call for local patients, carers, patient advocates, patient experts and patient organisation representatives to be able to express interest. We suggest to actively promote this opportunity to harder to reach patient communities and a periodical revision of the registry. Regarding ethical and compliance issues, a clear policy on financial and other conflicts of interests (how interests are declared, assessed and addressed) should be in place. Those registered should complete a declaration of interest form both on personal and organisational level conflict of interests and update it periodically. It should be clarified what kind of involvement with industry (e.g., attending a single advisory board meeting with a company versus only in case of direct conflict of interest) would impose restrictions on how a person can be involved in the HTA and decision-making process. We argue that in situations where patient experts and/or patient organisation representatives are difficult to identify, a softer approach should be taken and special measures could be proposed, equivalent to the “expert witness” status at the European Medicines Agency (EMA). Expert witnesses can be heard or participate in the deliberations but are not allowed to take part in the vote. However, the consequences of not being transparent with potential conflict of interests should be serious and communicated clearly from the beginning.KrT, RH, ZP, BN, and ZK were employed by Syreon Research Institute. At the time of the study IJ was the President of the European Patients’ Forum Youth Group, a Board of Trustees member at the EUPATI Foundation and employed by Syreon Research Institute. MN is the founder and co-owner of HealthQuest, a health technology assessment and market access consulting company. DaD is a Trustee of Thrombosis UK. TB was employed by Patvocates GmbH. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2023 Jakab, Dimitrova, Houÿez, Bereczky, Fövényes, Maravic, Belina, Andriciuc, Tóth, Piniazhko, Hren, Gutierrez-Ibarluzea, Czech, Tesar, Niewada, Lorenzovici, Kamusheva, Manova, Savova, Mitkova, Tachkov, Németh, Petykó, Dawoud, Delnoij, Knies, Goettsch and Kaló.)

Recent advances in biomedicine are opening the door to new approaches, and treatment and prevention are being transformed by novel medicines based on genetic engineering, innovative cell-based therapies and tissue-engineered products, and combinations of a medical device with embedded cell or tissue components. These advanced therapy medicinal products (ATMPs) hold one of the keys to making a reality of genuinely personalised medicine. There are an estimated 450 companies across the globe working on the development of gene therapies and more than 1,000 clinical trials underway worldwide, and some 20-30 new ATMPs filings are expected in Europe annually over the next 5 years. But challenges confront the sector, complicating the translation from research into patient access. Scientific, clinical development and regulatory issues are compounded by limited experience with clinical and commercial use, limited manufacturing know-how, high costs, and difficulties in accessing development funding and investment. Pricing and reimbursement and market access issues are an additional challenge, particularly in Europe, where unfamiliarity with the technology and uncertainty over the use of real-world evidence induce caution among clinicians, health technology assessment bodies and payers. There is a need for a review of the suitability of the regulatory and market access framework for these products, focused development of data, public/private partnerships, and fuller collaboration governments, doctors, insurers, patients, and pharmaceutical companies. This paper makes specific recommendations for all stakeholders, ranging from early dialogue on potential products, linking of clinical data and patient registries or standardisation of control frameworks, to a comprehensive approach to evidence generation, assessment, pricing, and payment for ATMPs., Competing Interests: The authors declare that they have no competing interests., (Copyright © 2020 by S. Karger AG, Basel.)

Rapid and continuing advances in biomarker testing are not being matched by take-up in health systems, and this is hampering both patient care and innovation. It also risks costing health systems the opportunity to make their services more efficient and, over time, more economical. This paper sets out the potential of biomarker testing, the unfolding precision and range of possible diagnosis and prediction, and the many obstacles to adoption. It offers case studies of biomarker testing in breast, ovarian, prostate, lung, thyroid and colon cancers, and derives specific lessons as to the potential and actual use of each of them. It also draws lessons about how to improve access and alignment, and to remedy the data deficiencies that impede development. And it suggests solutions to outstanding issues - notably including funding and the tangled web of obtaining reimbursement or equivalent coverage that Europe's fragmented health system implies. It urges a European evolution towards an initial minimum testing scenario, which would guarantee universal access to a suite of biomarker tests for the currently most common conditions, and, further into the future, to an optimum testing scenario in which a much wider range of biomarker tests would be introduced and become part of a more sophisticated health system articulated around personalised medicine. For exploiting genomics to the full, it argues the need for a new policy framework for Europe. Biomarker testing is not an issue that can be treated in isolation, since the purpose of testing is to improve health. Its use is therefore always closely linked to specific health challenges and needs to be viewed in the broader policy context in the EU and more widely. The paper is the result of extensive engagement with experts and decision makers to develop the framework, and consequently represents a wide consensus of views on how healthcare systems should respond from push and pull factors at local, national and cross-border and EU level. It contains strong views and clear recommendations springing from the convictions of patients, clinicians, academics, medicines authorities, HTA bodies, payers, the diagnostic, pharmaceutical and ICT industries, and national policy makers., Competing Interests: Benjamin Horbach is a Roche employee and has stock options in Roche. Stephen Hall is a Novartis employee and has stock options in Novartis. Benjamin Gannon is a Myriad Genetics employee and has stock options in Myriad. Rocio Varea Menendezis Eli Lilly employee and has stock options in Eli Lilly. Sarah Mee is an AstraZeneca employee and has stock options in AstraZeneca. Susana Banerjee receives grants from Astrazeneca, GSK & Honoraria − Astrazeneca/MSD, Amgen, Clovis, GSK, Mersana, Seattle Genetics, Merck Sereno, Genmab, Roche, Immunogen., (Copyright © 2020 by S. Karger AG, Basel.)

Since developments are global in the healthcare arena, more should be done to align EU and other big markets' regulatory practices for rare disease patients. Notwithstanding efforts and cooperation between the US and EU aimed to harmonize their strategic plans in the field of orphan drugs, regulatory criteria and procedures to gain the designation, terms and classifications should be still harmonised. Aligning the criteria of prevalence and support to orphan medicines in the various jurisdictions internationally, would facilitate patient recruitment eventually at global level, so as to gain the data and the biological insights required to identify biomarkers and appropriate endpoints needed for progressing clinical development. A conducive regulatory environment can further support the development of medicines to treat rare diseases. Overall there is a need for joined-up regulatory process coordination. Better integration of regulatory pathways and better integration of regulatory systems, such as scientific tools and methods to generate evidence, would be helpful. There is a need to revise and agree the current frameworks to be improved which will take into account the considerations and challenges to diagnose and treat different rare diseases and improve quality of life. Deliberative processes with multi-stakeholders' involvement for reimbursement should be considered. This paper explores the successes and limitation of both the regulation and its implementation mechanisms in the current regulatory context, and suggests some improvements that could maximise its benefits and boost rare disease research even further., Competing Interests: All authors declare that they have no competing interests. Ivana Cattaneo is a Novartis employee and has stock options in Novartis., (Copyright © 2020 by S. Karger AG, Basel.)