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Objective: Patients have unique insights and are (in-)directly affected by each decision taken throughout the life cycle of medicinal products. Patient preference studies (PPS) assess what matters most to patients, how much, and what trade-offs patients are willing to make. IMI PREFER was a six-year European public-private partnership under the Innovative Medicines Initiative that developed recommendations on how to assess and use PPS in medical product decision-making, including in the regulatory evaluation of medicinal products. This paper aims to summarize findings and recommendations from IMI PREFER regarding i) PPS applications in regulatory evaluation, ii) when and how to consult with regulators on PPS, iii) how to reflect PPS in regulatory communication and iv) barriers and open questions for PPS in regulatory decision-making.Methods: PREFER performed six literature reviews, 143 interviews and eight focus group discussions with regulators, patient representatives, industry representatives, Health Technology Assessment bodies, payers, academics, and clincians between October 2016 and May 2022.Results: i) With respect to PPS applications, prior to the conduct of clinical trials of medicinal products, PPS could inform regulators’ understanding of patients’ unmet needs and relevant endpoints during horizon scanning activities and scientific advice. During the evaluation of a marketing authorization application, PPS could inform: a) the assessment of whether a product meets an unmet need, b) whether patient-relevant clinical trial endpoints and outcomes were studied, c) the understanding of patient-relevant effect sizes and acceptable trade-offs, and d) the identification of key (un-)favorable effects and uncertainties. ii) With respect to consulting with regulators on PPS, PPS researchers should ideally have early discussions with regulators (e.g., during scientific advice) on the PPS design and research questions. iii) Regarding external PPS communication, PPS could be reflected in the assessment report and product information (e.g., the European Public Assessment Report and the Summary of Product Characteristics). iv) Barriers relevant to the use of PPS in regulatory evaluation include a lack of PPS use cases and demonstrated impact on regulatory decision-making, and need for (financial) incentives, guidance and quality criteria for implementing PPS results in regulatory decision-making. Open questions concerning regulatory PPS use include: a) should a product independent broad approach to the design of PPS be taken and/or a product-specific one, b) who should optimally be financing, designing, conducting, and coordinating PPS, c) when (within and/or outside clinical trials) to perform PPS, and d) how can PPS use best be operationalized in regulatory decisions.Conclusion: PPS have high potential to inform regulators on key unmet needs, endpoints, benefits, and risks that matter most to patients and their acceptable trade-offs. Regulatory guidelines, templates and checklists, together with incentives are needed to foster structural and transparent PPS submission and evaluation in regulatory decision-making. More PPS case studies should be conducted and submitted for regulatory assessment to enable regulatory discussion and increase regulators’ experience with PPS implementation and communication in regulatory evaluations.

Current challenges to healthcare, i.e. the emergence of new diseases, lack of therapies for known diseases and life-threatening conditions, identification of patients who do not respond to standard treatment, on the one hand, and the evolution of scientific understanding of disease processes, medicines, therapies, causes of treatment failures, and implementation in clinical practice of innovations related to molecular biology and genetic engineering, on the other hand, create conditions and opportunities for the development of innovative medicinal products. A relatively new class of medicines is based on human cells and tissues (the term used in Russian legislation is biomedical cell products, BCP). However, the inability to accurately predict the efficacy and financial rewards of such medicines for pharmaceutical companies, as well as significant labour and financial costs associated with their development and clinical use, hinder their entry into the market. The aim of the study was to analyse the foreign regulatory setting for the development and launch of human cell- and tissue-based products, as well as approaches of foreign regulatory authorities to scientific advice, which can be drawn upon by the Russian expert authority when providing advice to BCP developers. The paper summarises the results of analysis of regulations establishing the procedure for providing scientific advice by EU, USA, and Russian regulatory authorities, and analyses the advice provided for the human cell- and tissue-based products which are now authorised in the EU and USA. The analysis of advice provided by foreign regulatory authorities shows that the largest number of consultations were given for medicinal products based on genetically modified cells for the treatment of cancer and genetic diseases. The questions were mainly related to the contents of specifications for finished pharmaceutical products, safety evaluation, curtailing of preclinical studies due to the lack of relevant animal/disease models, the number of subjects and efficacy endpoints in clinical studies, assessment of the appearance of replication-competent retroviruses.

IntroductionEarly advice in the process of developing health technologies allows manufacturers to plan their production and transfer to health care systems more accurately. This review aims to describe frameworks used within HTA and their current use by HTA Agencies.Material and methodsWe carried out a systematic literature review in Pubmed, Embase, Scopus, and WoS, including all references published in Spanish and English. This was last updated in March 2022. We extracted all available information regarding the organizations involved, services offered, types of technology, collaborators involved, fees, output and impact. Websites of several HTA organizations and Google were also searched in order to update and complete the information obtained from this generic search.ResultsFive-hundred and forty one articles were identified and screened, of which 26 met the eligibility criteria and were selected. Seven of them were non-systematic reviews that described two or more HTA organizations. Ten studies were focused on the advice offered by individual organizations, and eight described the EMA and EUnetHTA parallel or joint advice. We found variations in the technology assessed, services offered, stage of development and costs for advisory services.ConclusionsEarly and scientific advice would help manufacturers focus their product development on what is needed for the management of specific diseases. Most of the examples or services found refer to drugs as well as to some medical devices and diagnostics. A common definition of the type of advice that could be offered for different health technologies by HTA bodies to ascertain health care systems and manufacturers' needs, in addition to the timeline in which that advice needs to be given, would help HTA bodies provide the right support at the right time.Systematic review registrationhttps://www.crd.york.ac.uk/prospero/display_record.php?ID=CRD42020219401, PROSPERO CRD42020219401.

Science is increasingly expected to help in solving complex societal problems in collaboration with societal stakeholders. However, it is often unclear under what conditions this can happen, i.e., what kind of challenges occur when science interacts with society and what kind of quality expectations prevail. This is particularly pertinent for Social Sciences and Humanities (SSH), which are part of the object they study and whose knowledge is always subject to provisionality. Here we discuss how SSH researchers can contribute to societal problems, what challenges might occur when they interact with societal stakeholders, and what quality expectations arise in these arrangements. We base our argumentation on the results of an online consultation among 125 experts in Germany (representatives from SSH, learned societies, stakeholders from different societal groups, and relevant intermediaries).

International environmental advisory bodies are tasked with the daunting challenge of collecting and synthesizing knowledge about a specific issue-area by speaking in one ‘global scientific voice’. However, the ‘type of knowledge’ issuing from these bodies can hardly meet the expectations of ‘getting the science right’, as scientific issues inevitably end up being framed in different ways. Although accounts of contestation between various knowledge claims are abundant in the literature focusing on international advisory bodies, the implications of these tensions for science–policy interplay remain poorly understood. In particular, analyses of and reflections on the relationship between knowledge outputs and particular institutional arrangements are underdeveloped. This paper attempts to address this gap, postulating a link between knowledge outcomes and institutional design. By introducing the concept of ‘epistemic framings’, it explores the different ways in which soil and land degradation issues are framed by scientific advisory bodies at the global level. The analysis, conducted through the Qualitative Content Analysis (QCA) method, suggests that international scientific advisory bodies may frame epistemic issues in ways that are influenced by the policy setting to which they are institutionally bound. Further research on the dynamics of science–policy interaction at the global level could test the assumptions made in this paper and shed light on the structural (including institutional design) and agential factors influencing advisory bodies’ epistemic framings.

Abstract Envisioning the expertise of the future in the field of food safety is challenging, as society, science and the way we work and live are changing and advancing faster than ever before. Future challenges call for multiple and multidimensional responses, some of which were addressed at EFSA's Third Scientific Conference. The participants indicated that risk assessment bodies involved in food safety such as EFSA must recognise that data, methods and expertise (i.e. people) are the three basic elements underlying risk assessments. These elements need constant consideration and adaptation to ensure preparedness for the future. Moreover, it should be recognised that knowledge and expertise are distributed throughout society and are thus not limited to scientists. Aspects considered during the breakout session included: (1) increased complexity, (2) the crowd workforce, (3) citizen science, (4) stakeholder engagement, (5) talent pools and (7) entrepreneurship. To account for future challenges, behavioural, attitudinal and cultural changes must be implemented successfully. At a societal level, people are increasingly going hand in hand with robotics and artificial intelligence in sharing expertise and producing outcome. This needs consideration on ethics and values, both for organisations and individuals. At an organisational level, risk assessment bodies will have to tap into new talent pools and new solutions for a more fluid and ad hoc‐based workforce. Future risk assessment bodies will have to actively engage with stakeholders when performing their assessments. It is expected that the impacts of citizen science and involvement of the crowd will become part of risk assessment practices. Consequently, EFSA will have to continue to invest in massive, ongoing skills development programmes. At an individual level, potential recruits will need to be assessed against a whole new set of competencies and capabilities: technical competencies in data science, computational science and artificial intelligence, alongside a large set of soft skills.

Abstract Governments and international organizations frequently convene scientific advisory committees (SACs) to support decision‐making with scientific advice. In this study, thematic analysis of interviews with 35 senior WHO staff identified five main themes characterizing WHO's experience with designing SACs to ensure quality, relevance, and legitimacy of scientific advice. First, in addition to technical matters, SACs are established to serve broader strategic objectives, including consensus building to promote high‐level political messages. Second, for SACs to be fully independent, they must have autonomy from the institutions convening or funding them, from the institutions from where SAC members are recruited, and from the institutions to whom the advice is directed. Third, since choices affecting quality, relevance, and legitimacy are closely linked, designing SACs often require trade‐offs among these three attributes. Fourth, staff supporting SACs need to balance between safeguarding SACs from external influence and being receptive to the external political environment. Fifth, the design of SACs need to balance the involvement of stakeholders with the power to act on recommendations against the need to protect the independence and integrity of the scientific process. Overall, this study highlights key choices conveners of SACs must make when seeking to promote quality, relevance, and legitimacy of scientific advice.

Advanced therapy medicinal products (ATMPs), i.e., cell and gene therapy products, is a rapidly evolving field of therapeutic development. A significant proportion of the products are being developed by academia or small/medium-sized enterprises (SMEs). The many challenges in translation posed by this class of products include aspects covering: manufacturing, non-clinical development plan as relevant to clinical trial, marketing authorization, and reimbursement. In this context, the term translation refers to the relevance of non-clinical data in relation to how it impacts on appropriate and efficient clinical development. In order to successfully overcome these challenges, a clear understanding of the requirements and expectations of all the stakeholders is critical. This article aims to cover the potential challenges related to such translation and suggested approaches to find solutions based on experience and learnings from the perspective of European Union. While commercial challenges have a significant impact on the ATMPs in general, it is considered outside the scope of this article. However, by adopting a strong scientific basis for translation as suggested in this article, it is likely such an approach would help rather than harm successful real world clinical use of ATMPs.

Over its first three mandates the EFSA Panel on Dietetic Products, Nutrition and Allergies (NDA Panel) has provided scientific advice on a range of issues related to human nutrition. The scientific outputs adopted over this time are summarised within the six main areas within the remit of the Panel: (i) tolerable upper intake levels for nutrients; (ii) safety of novel foods; (iii) food allergens; (iv) dietetic products; (v) population reference intakes for nutrients; and (vi) nutrition and health claims on foods. The scientific advice of the Panel was provided mainly in response to requests from the European Commission and applications for authorisation of novel foods, health claims and for exemption from allergen labelling. The outputs of the Panel have been extensively used by the European Commission and Member States in the implementation of European Union (EU) policy and legislation. In addition, some of the scientific opinions assess key elements of the evidence on nutrition in obesity and other diet related diseases and are an important source of scientific advice for policy makers who wish to address these important issues in public health. Throughout the different areas of its work, the Panel has placed considerable focus on consultation with stakeholders. In the area of health claims in particular, EFSA has had continuing consultation with stakeholders in developing extensive guidance for applicants. This article summarises the nature of the advice provided by the Panel, its regulatory context and how it is applied in the implementation of EU policy and legislation.