Future-Proofing European Pharmaceutical Regulatory and Market Access Practices Based on EU Learnings from the COVID-19 Pandemic: Insights from Multi-Stakeholder Interviews.

Introduction: During the COVID-19 pandemic, regulatory and market access actions were taken to expedite the market entry of COVID-19 medicines. This study aims to (i) capture multi-stakeholder views on these actions, and (ii) provide recommendations for future-proofing routine and health-emergency frameworks.

Methods: Semi-structured interviews were conducted with policy makers/advisors (i.

Barriers to adoption of biosimilar insulins in Europe: A mixed-methods study.

Background: Despite the introduction of biosimilar insulins to improve affordability and enhance patient access, their adoption remains limited in Europe.

Objectives: This study aims to (i) provide an up-to-date overview of EMA-approved insulin products and their reimbursement status across selected European countries, and (ii) explore the k,ey factors influencing physicians' prescribing decisions regarding biosimilar and originator insulin.

Methods: A mixed-method design was applied, comprising (i) a quantitative analysis of EMA-approved insulin products and their reimbursement status in nine European countries, and (ii) semi-structured interviews with physicians (N = 20).

Advanced therapy medicinal products are coming of age: A pipeline analysis of the clinical trial landscape.

Advanced therapy medicinal products (ATMPs) are a unique class of biological medicines that are based on cells, genes, or tissues. Searching four databases, this analysis identified 2008 trials that investigated ATMPs and characterised them according to various characteristics, such as the sponsor type and phase of clinical development. This characterisation revealed that the market for ATMPs is maturing in terms of the number of products in development, the diseases being targeted, and the technologies being pursued.

Patients' Needs and Preferences for Cardiac Pacemaker Implantation: A Qualitative Study on Disease and Medical Device Characteristics to Inform a Quantitative Preference Study.

Background: Cardiac pacemakers are the only long-term treatment for symptomatic bradyarrhythmia. Despite technological advances, conventional pacemakers still face significant device and procedure-related complications. Recently, leadless pacing systems have been developed to address these issues.

Criteria to evaluate unmet health-related needs of persons living with rare diseases and their caregivers: rapid literature review and stakeholder consultations.

Background: Rare diseases affect small populations but present unique challenges in access to healthcare and social support. The needs of patients and their caregivers extend beyond medical treatments, impacting various aspects of their lives. This study provides a narrative overview of these diverse needs experienced by patients and caregivers.

Evolving global regulatory landscape for approval of biosimilars: current challenges and opportunities for convergence.

Introduction: Biosimilars promote price competition, improving affordability and access to biologics without compromising on quality, efficacy, and safety. Biosimilar approvals initially followed a cautious approach, with regulatory requirements developed independently across jurisdictions, complicating global development and increasing costs. Advancements in analytical sciences and two decades of accumulated experience with biosimilar approvals offer an opportunity to reevaluate regulatory requirements.

Synergizing Needs Assessments and Patient Preference Studies for Enhanced Patient-Centered Decision Making in Healthcare.

Objectives: Over the past decades, criticism has grown regarding the supply-driven approach of medicinal product development. In response, patient-centered methods have been developed to inform decision making. This perspective article aims to reflect on two of these methods, which are needs assessments and patient preference studies (PPSs).

Evolution of reported patient and public involvement over time in randomised controlled trials in major medical journals and in their protocols: meta-epidemiological evaluation.

Objective: To investigate the reporting and evolution of patient and public involvement (PPI) in randomised controlled trials published over time in major medical journals and in their trial protocols.

Design: Meta-epidemiological evaluation.

Data Source: PubMed was searched for articles reporting randomised controlled trials published since 2015 in four major medical journals and their corresponding peer reviewed protocols.

Caregivers and multidisciplinary team members' perspectives on shared decision making in Duchenne muscular dystrophy: A qualitative study.

Background: As new therapies for Duchenne muscular dystrophy (DMD) are entering the market, shared decision making (SDM) will become increasingly important. Therefore, this study aimed to understand (1) Belgian stakeholders' knowledge and perceptions of SDM in DMD treatment decision making, (2) the current state of SDM implementation in DMD in Belgium, examining the role of all involved parties, and (3) the barriers and facilitators for SDM in DMD in the Belgian context.

Methods: In this qualitative study, semi-structured interviews with the multidisciplinary team (MDT) of individuals with DMD (n = 18) and caregivers thereof (n = 11) were conducted in Belgium.

Drawing on collective action theory to foster sustainable biosimilar markets - insights from co-creation workshops with UK and Belgian stakeholders.

Background: Research has signaled the need for reformed biosimilar policy frameworks that adopt a behavioral approach, are informed by consensus-generating strategies and thus better align with the requirements of local healthcare communities.

Research Design And Methods: Through a series of co-creation workshops, the current study explores the feasibility of applying learnings from Collective Action Theory to formulate evidence-based multistakeholder-supported policy recommendations.

Results: Insights from the conducted workshops indicate that future policy frameworks would benefit from: 1) a working system of incentives and rewards aligned with stakeholder needs; 2) evaluating the cost-benefit balance for stakeholders prior to policy implementation; 3) involving multistakeholder panels in policy co-design; 4) adopting a long-term vision; 5) fostering coordination at the interface between levels of governance; 6) defining shared goals and efficient systems to monitor policy compliance; and 7) using policy outcome data to adapt current policy frameworks based on evolving needs.

Towards implementing new payment models for the reimbursement of high-cost, curative therapies in Europe: insights from semi-structured interviews.

Background: New ways of reimbursement for high-cost, one-shot curative therapies such as advanced therapy medicinal products (ATMPs) are a growing area of interest to stakeholders in market access such as industry representatives, legislative and accounting experts, physicians, hospital managers, hospital pharmacists, patient representatives, policymakers, and sickness funds. Due to the complex nature of ATMPs, new payment models and reimbursement modalities are proposed yet not widely applied across Europe.

Objectives: This study aimed to elicit opinions on and insights into the governance aspect of implementing outcome-based spread payments (OBSP) in Belgium for the reimbursement of innovative therapies.

Unmet medical needs definition and incentives: stakeholders perspectives on the reform of the EU pharmaceutical legislation.

Introduction: The 2020 pharmaceutical strategy for Europe stressed that rethinking regulatory policies to foster innovation in disease areas with unmet medical needs (UMN) is one of the European Commission's (EC) priority areas. To understand stakeholders' views regarding appropriate UMN criteria and incentives, the EC developed a survey and launched it for public consultation between September and December 2021. This study aims to assess stakeholders' views on the policy revisions proposed by the EC, particularly those regarding the definition of UMN, its criteria and incentives and evaluate how stakeholders' views are reflected in the proposed reform of the EU pharmaceutical legislation of 2023.

Information Service "My Cancer Navigator" to Support Shared Decision-Making: An Online Survey Among Patients with Cancer and Their Caregivers.

The personalized information service My Cancer Navigator (MCN) answers therapy-related questions of patients with cancer and their caregivers, to address information needs and contribute to shared decision-making (SDM). An explorative and descriptive cross-sectional study using online surveys was conducted to assess whether users perceived a change in factors contributing to SDM after using the service. Of 253 invited MCN users, 109 (43.

Do European regulatory measures accelerate national market access in Belgium? A retrospective analysis of medicines centrally authorised between 2015 and 2020.

Background: At the European level, several regulatory measures (ie, priority medicines (PRIME) scheme, accelerated assessment, conditional marketing authorisation and authorisation under exceptional circumstances) are in place with the aim to expedite the marketing authorisation process for medicines targeting unmet medical needs (UMNs). However, the potential impact of these measures on subsequent decisions regarding market access at the national level, and ultimately if medicines making use of these supporting measures reach the patient earlier, remains unclear.

Objectives: This study seeks to (1) assess the impact of such European regulatory measures on the number of successful applications and time to reimbursement of this group of medicines in the national context of Belgium and (2) evaluate the association between the application of European regulatory measures and Belgian measures (ie, early access pathways and managed entry agreements).

Evolution of Eligibility Criteria in Inflammatory Bowel Disease Clinical Trials: A Clinical Trial Databank Analysis.

Background: Eligibility criteria in clinical trials have been criticised for being overly restrictive without clinical justification.

Objective: We aimed to investigate the types, evolution, and current status of eligibility criteria in clinical trials for inflammatory bowel diseases (IBD).

Methods: We performed a clinical trial databank search on clinicaltrials.

Mitigating the Nocebo Effect in Biosimilar Use and Switching: A Systematic Review.

Background: In the context of biosimilar use and switching, a patient's negative perception of a biosimilar might trigger a nocebo effect, where negative expectations unrelated to its pharmacologic action may lead to worsened symptoms, resulting in less perceived therapeutic benefit or worsened adverse events. Considering the crucial role biosimilars have in reducing health care costs and expanding access to safe biologics globally, it is essential to develop and implement effective strategies to mitigate the possible occurrence of such nocebo effects.

Objective: The aim of this systematic literature review was two-fold: (i) to review strategies that have been applied and/or tested for minimising the nocebo effect in clinical practice, within and outside the context of biosimilar switching, and (ii) to propose recommendations on useful mitigation strategies to minimise the occurrence of the nocebo effect in the context of biosimilar switching.

Development and Characterization of a Human Mammary Epithelial Cell Culture Model for the Blood-Milk Barrier-A Contribution from the ConcePTION Project.

It is currently impossible to perform an evidence-based risk assessment for medication use during breastfeeding. The ConcePTION project aims to provide information about the use of medicines during lactation. The study aimed to develop and characterize an in vitro model of the blood-milk barrier to determine the extent of the milk transfer of xenobiotics, relying on either on human mammary epithelial cells (hMECs) or immortalized cell lines derived from breast tissue.

Patient preferences for inflammatory bowel disease treatments: protocol development of a global preference survey using a discrete choice experiment.

Background: As the therapeutic landscape for inflammatory bowel disease (IBD) continues to expand, a need exists to understand how patients perceive and value different attributes associated with their disease as well as with current and emerging treatments. These insights can inform the development and regulation of effective interventions for IBD, benefiting various stakeholders including healthcare professionals, drug developers, regulators, Health Technology Assessment bodies, payers, and ultimately patients suffering from IBD. In response to this, the present patient preference study was developed with the aim to (1) determine the relative preference weights for IBD treatment and disease related attributes, and (2) explain how preferences may differ across patients with different characteristics (preference heterogeneity).

EU-US data transfers: an enduring challenge for health research collaborations.

EU-US data transfers for health research remain a particularly thorny issue in view of the stringent rules of the EU General Data Protection Regulation (GDPR) and the challenges related to US mass surveillance programs, particularly the manner in which US law enforcement and national security agencies can access personal data originating from the EU. Since the entry into force of the GDPR, evidence of impeded collaborations is increasing, particularly in the case of sharing data with US public institutions. The adoption of a new EU-US adequacy decision in July 2023 does not hold the promise for a long-lasting solution due to the risks of being challenged and invalidated - yet again - at the Court of Justice of the EU.

Budget Impact of Disease-Modifying Treatments and a CRISPR Gene-Edited Therapy for Sickle Cell Disease.

Background And Objective: Treatment of sickle cell disease (SCD) has traditionally focused on symptomatic and preventative care. Recent advances in novel therapeutic developments, likely to be orphan-designated, are anticipated to carry a substantial price tag. This study assesses the potential budget impact of adopting disease-modifying treatments, crizanlizumab and voxelotor, and pioneering CRISPR gene-edited therapy, CTX001, in the Belgian healthcare system.

Opportunities and Challenges in Cross-Country Collaboration: Insights from the Beneluxa Initiative.

National pricing and reimbursement agencies face growing challenges with complex health technologies, prompting European policy advancements. Beneluxa is a cross-country collaboration involving Belgium, the Netherlands, Luxemburg, Austria, and Ireland that aims to address sustainable access to medicines. In view of the soon-to-be-implemented EU HTA Regulation, insights and experiences from stakeholders with Beneluxa cross-country collaboration could provide possible transferable learnings.

Heart failure patients' perspectives on treatment outcomes and unmet medical needs: A qualitative preference study.

Aims: Decision-makers still predominantly focus on the perspective of non-patient stakeholders, which may deviate from the unique perspective of heart failure (HF) patients. To enhance patient-centred decision-making, there is a need for more patient-based evidence derived directly from the patients themselves. Hence, this study aimed to understand (i) HF patients' unmet medical needs and preferred treatment outcomes; (ii) patients' risk tolerance; and (iii) their information needs, uncertainties and satisfaction towards HF treatment.

Implementing the EU HTA regulation: Insights from semi-structured interviews on patient expectations, Belgian and European institutional perspectives, and industry outlooks.

The goal of the Health Technology Assessment (HTA) Regulation 2021/2282 is to establish a more harmonized HTA framework, fostering member states cooperation and enabling equal patient access to innovative health technologies in Europe. This research aimed to assess the impact of the regulation on national HTAs, the strategic implications for health technology developers, and its influence on price and reimbursement negotiations. A scoping literature review encompassing peer-reviewed literature as well as grey literature was conducted.

An Inclusive Civil Society Dialogue for Successful Implementation of the EU HTA Regulation: Call to Action to Ensure Appropriate Involvement of Stakeholders and Collaborators.

Objectives: Stakeholder involvement has long been considered a success factor for a joint European health technology assessment (HTA) process, and its relevance is now anchored in the EU HTA Regulation's (EU HTAR) legislative wording. Therefore, we aimed to explore the roles, challenges, and most important activities to increase the level of involvement per stakeholder group.

Methods: At the 2022 Fall Convention of the European Access Academy (EAA), working groups addressed the involvement of patients, clinicians, regulators, health technology developers (HTD), and national HTA bodies and payers within the EU HTA process.