Health Technology Assessment Outcomes Research Articles

Evidence Quality and Health Technology Assessment Outcomes in Reappraisals of Drugs for Rare Diseases in Germany

ObjectivesEvidence on reappraisals of health technologies in Germany is limited, and for rare disease treatments (RDTs), the Federal Joint Committee follows different processes (limited or regular), depending on whether an annual revenue threshold has been exceeded. Our objective is to better understand (re)appraisal processes and their outcomes for RDTs in Germany. MethodsWe analyzed appraisal documents of 55 RDT indications for which an initial appraisal and a reappraisal were conducted between 2011 and 2023. We extracted information for the type of evidence, the risk of bias, the availability of additional evidence, and the change in the maturity of survival data as proxies for evidence quality. Specifically, we reviewed the reasons for conducting reappraisals, examined how evidence quality and the clinical benefit rating (CBR) differed between initial appraisals and reappraisals, and explored the association between evidence quality and (1) the CBR and (2) the change in the CBR after reappraisal. ResultsMost reappraisals were conducted because the annual revenue threshold was exceeded or the initial appraisal resolution was time limited. Almost all initial appraisals used the limited process, whereas the majority of reappraisals used the regular process. The CBR increased in only 9 and decreased in 21 of 55 reappraisals. There was some evidence that reappraisals with an accepted randomized controlled trial were significantly more likely to achieve a higher CBR. ConclusionsFindings confirmed that reasons and processes for conducting reappraisals of RDTs in Germany differ. Further, high CBRs in reappraisals were not common and evidence quality in initial appraisals and reappraisals was limited.

HTA218 Public Engagement and Its Influence on Health Technology Assessment Outcomes in Latin America

HTA218 Public Engagement and Its Influence on Health Technology Assessment Outcomes in Latin America

PP01 Health Technology Assessment Of Cervical Artificial Disc Replacement: Highlighting The Need For A Consistent International Approach

IntroductionCervical artificial disc replacement (C-ADR) is not a new technology but one that has seen many technological advances in the past 10 years. Indeed, a recent review described total disc arthroplasty as the most innovative development in the history of spinal surgery. The primary goals of C-ADR are to reduce or eliminate pain, and restore normal segmental motion. The aim of this analysis was to identify, extract and examine key health outcomes and economic data from published health technology assessment (HTA) reports on C-ADR, with the aim of understanding how the evolution of this technology has influenced assessments internationally.MethodsA comprehensive search of over 90 HTA organization websites and the INAHTA HTA database using key terms for C-ADR surgical procedures was coupled with a literature search of recent systematic reviews. No language restrictions were applied.ResultsTwenty HTA reports of C-ADR surgery published from 2005 to 2022 were included for review. Several HTAs (4/20) were updates or reassessments by the same agency and one was an update across agencies (Italy update of Belgian HTA). While many of the HTAs concluded C-ADR is as effective as standard care and superior in certain outcomes, there was no pattern or consistency in the conclusions or recommendations from these assessments, even as the evidence base expanded over time. Our analysis found this was largely due to variations in HTA approaches among agencies including: differences in research questions asked, PICO (Population, Intervention, Comparator, Outcomes) criteria and methods performed, such as: rapid versus full systematic reviews; inclusion of economic evaluations and/or budget impact analyses.Indeed, one of the only predictive factors for a positive HTA was a favorable cost-effectiveness analysis.ConclusionsC-ADR is an established technology with extensive HTA investigation internationally. The lack of a consistent approach taken by HTA bodies made prediction of successful HTA outcomes difficult. Future alignment of key evaluation processes and methods may help address current international variations and support consistent decision making on patient access.

Algorithms and heuristics of health technology assessments: A retrospective analysis of factors associated with HTA outcomes for new drugs across seven OECD countries

ContextPositive health technology assessment (HTA) outcomes can have important implications for equity, efficiency and timely patient access to novel therapies. Several outcomes and dimensions of benefit beyond utility feed into HTA processes. ObjectiveWe analyse a proprietary dataset of HTA outcomes in 7 countries, to (a) test whether HTA decision-making is grounded in welfarist or extra-welfarist approaches; and (b) empirically determine the factors associated with positive HTA outcomes, the time to achieve these and establish the magnitude of inter-country differences in assessment processes. MethodsData were extracted from publicly available HTA reports on drugs that received marketing authorisation between 2009 and 2018 (N = 1415). The outcomes of interest were the probability of positive HTA outcomes and the time-to-HTA outcome; these were examined with respect to clinical, regulatory, product- and disease-related, evidence uncertainty and contextual variables. Econometric models utilising survival analysis and multinomial logistic regression were specified. FindingsPositive HTA outcomes accounted for 87.3% of the sample (n = 1235), of which 71% (n = 1004) were restricted. Drugs with positive HTA outcomes were subject to clinical restrictions (n = 652, 46%), financial risk-sharing (n = 439, 31%) or had been rejected at least once (n = 282, 20%). Significant predictors of positive HTA outcomes were orphan drugs with cancer indications, high quality of evidence linked to clinical and economic evidence uncertainties which had been overcome, and contextual considerations, particularly innovativeness and unmet need. Comparative analyses revealed systematic differences between countries in their propensity to accept the same drugs, particularly oncology and orphan drugs. ConclusionsOur results are contextual and reinforce arguments in favour of explicitly accounting for social value judgements, establishing separate assessment frameworks for highly uncertain products, adopting risk mitigation strategies for novel therapies with early phase evidence, and sharing of HTA practices across settings. Lastly, HTA agencies have adopted an extra-welfarist approach to value assessment and resource allocation.

HPR161 International Comparisons of Health Technology Assessment and Reimbursement Outcomes for Oncology Drugs with Regulatory Review through Project Orbis

Project Orbis is an international regulatory collaboration led by the US Food and Drug Administration (FDA) Oncology Center of Excellence. It represents a framework for concurrent submission and review of oncology products, with the aim to give patients faster access to promising cancer treatments. However, products with limited clinical evidence can pose challenges for health technology assessment (HTA) and public-payer decisions. We examined HTA and reimbursement outcomes for Project Orbis drugs reviewed in Canada and other jurisdictions globally from program inception in 2019 to end of 2022.

Health Technology Assessment–Informed Decision Making by the Federal Joint Committee/Institute for Quality and Efficiency in Health Care in Germany and the National Institute for Health and Care Excellence in England: The Role of Budget Impact

ObjectivesThis study aimed to test (official) evaluation criteria including the potential role of budget impact (BI) on health technology assessment (HTA) outcomes published by the Federal Joint Committee (Gemeinsamer Bundesausschuss [GBA]) and the Institute for Quality and Efficiency in Health Care (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen [IQWiG]) in Germany as well as the National Institute for Health and Care Excellence (NICE) in England. MethodsData were extracted from all publicly available GBA decisions and IQWiG assessments as well as NICE single technology appraisals between January 2011 and June 2018, and information with regard to evaluation criteria used by these agencies was collected. Data were analyzed using logistic regression to estimate the effect of the BI on the HTA outcomes while controlling for criteria used by GBA/IQWiG and NICE. ResultsNICE recommendations are largely driven by the incremental cost-effectiveness ratio and, if applicable, by end-of-life criteria (P < .01). While IQWiG assessments are significantly affected by the availability of randomized controlled trials and patient-relevant endpoints (P < .01), GBA appraisals primarily focus on endpoints (P < .01). The BI correlated with NICE single technology appraisals (inverted-U relationship, P < .1) and IQWiG recommendations (increasing linear relationship, P < .05), but not with GBA decisions (P > .1). Nevertheless, given that IQWiG assessments seem to be more rigorous than GBA appraisals regarding the consideration of evidence-based evaluation criteria, decisions by GBA might be negatively associated with the BI. ConclusionsResults reveal that GBA/IQWiG and NICE follow their official evaluation criteria consistently. After controlling for all significant variables, the BI seems to have an (independent) effect on HTA outcomes as well.

HTA Barriers for Conditional Approval Drugs.

Conditional approval pathways facilitate accelerated marketing authorisation based on immature clinical evidence for drugs that address an unmet medical need in a life-threatening or chronically debilitating condition. Lowering evidence requirements for marketing authorisation results in higher clinical uncertainty, which may present challenges for the health technology assessment (HTA) of these products. The objective of this study is to assess whether conditionally approved drugs face higher probabilities of HTA rejection or delays in HTA approval relative to drugs with standard marketing authorisation. This paper adopts a mixed-methods approach to provide a meta-analysis of HTA outcomes across 80 drug-indication pairs in France, England, Scotland and Canada. Differences in the characteristics (i.e. disease rarity and clinical trial design) of conditionally approved drugs and drugs with standard marketing authorisation and drivers of HTA outcomes are assessed through logistics regressions. Delays in HTA approval are assessed through a survival analysis. Relative to standard approval drugs, conditionally approved drugs are less likely to include phase III trial designs, less likely to include clinical endpoints and less likely to include an active comparator. Uncertainties in clinical and economic evidence are raised more frequently by HTA agencies for conditionally approved drugs, which have a marginally lower probability of receiving HTA approval relative to drugs with standard approval. Conditionally approved drugs face moderate delays (an average of 6 months) in receiving HTA approval relative to standard approval drugs. Overall, conditionally approved drugs likely face increased barriers at the HTA level.

PP115 Analysis Of Previous Joint Clinical Assessment And Potential Transferability To Four European Countries: Case Study And Conceptual Approach

IntroductionThe European Network for Health Technology Assessment (EUnetHTA) has led together, with member states, several joint actions, including Joint Clinical Assessments (JCA), under the form of project-based voluntary cooperation, which outputs and transferability of those projects in other European countries remains somehow limited. In June 2021, the European Council has reached an agreement on the European Health Technology Assessment (HTA) regulation, which is entering into force gradually. Initially limited to oncology products, then extended to orphan/advanced therapy, and after a five to eight-year additional period, it will apply to all centrally approved products. The JCA will consist of a focused scientific analysis on relative effectiveness assessment, including the health condition, technology description, clinical effectiveness, and safety. These analyses will also include information relating to the degree of certainty. We consider that the evidence appraisal might have limitations, and transferability would not be generalizable. We aim to determine the potential drivers and barriers for HTA transferability in EU4, employing the analysis of a case example where JCA was conducted.MethodsEmploying an oncology JCA, we will compare an HTA analysis conducted in EU4 countries (Germany, France, Italy, and Spain). Overview and background information on countries involved in the JCA, and EU4 HTA system will be provided, followed by HTA outcome and main evidence requirements, reimbursement outcome, and pricing agreements.ResultsStudy results supporting HTA outcomes may focus on the population assessed, the comparator considered, and uncertainty management. A conceptual adaptation about the scope of the EU JCA regulation will be discussed, to understand its potential advantages to individual HTAs in Europe and remaining gaps to effectively inform HTA or decision-making process.ConclusionsThe analysis of pricing and reimbursement outcomes can further help understand potential barriers and drivers for JCA transferability and potential areas of evidence generation requirements.

HPR37 Comparing Reimbursement of New Drugs During the COVID-19 Pandemic in Ireland and Scotland

Following the onset of the COVID-19 pandemic, the Scottish Medicines Consortium (SMC) suspended monthly meetings from April to August 2020, to support the National Health Service (NHS) Scotland in maintaining essential work. Following the return of meetings, a fast-track process was introduced to expedite reimbursement for certain medicines. Conversely, the Irish health technology assessment (HTA) agency, the National Centre for Pharmacoeconomics (NCPE), continued to assess new reimbursement applications throughout this period, using the two-stage process of a rapid review (RR), followed by full HTA for some medicines. This research explores how these different approaches impacted reimbursement submissions and recommendations during this time. A database was developed using published data (from 01/03/2020 to 01/03/2022) on the NCPE, SMC and Irish Health Service Executive (HSE) websites, including; • Breakdown of submissions received • Assessment outcomes • Breakdown of reimbursement recommendations Data was extracted into Excel® and analysed using summary and descriptive statistics. SMC advice for 169 medicines were identified (n=89 full submissions; n=36 abbreviated submissions; n=28 non-submissions; n=13 resubmissions; n=3 ultra-orphan assessments). Positive reimbursement recommendations were made for 136 of these medicines (n=68 accepted for use, n=58 accepted for restricted use, n=7 accepted on interim basis, n=3 available under ultra-orphan assessment), with 48 recommendations made using the fast-track process. Contrastingly, the NCPE published 148 RR outcomes and 61 HTA outcomes, with 70 reimbursement recommendations published by the HSE (n=38 positive recommendations, n=11 restricted reimbursement, n=21 reimbursement not recommended). While a similar number of submissions were assessed by the Irish and Scottish HTA agencies during the COViD-19 period, there was a significantly higher number of positive reimbursement recommendations from the SMC, which may be explained by the SMC fast-track process. This research further explores the reimbursement recommendations for each category of submission and compares the reimbursement timelines for each jurisdiction.

HTA38 When and How Does RWE Impact HTA Outcomes in Oncology? A Qualitative Evaluation of Case Studies to Identify Drivers of RWE Acceptance Today and in the Future

Understand how different health technology assessment (HTA) bodies perceive and evaluate real world evidence (RWE) in oncology HTA submissions, and identify drivers of RWE acceptance today and in the future.

Value and Price of Multi-indication Cancer Drugs in the USA, Germany, France, England, Canada, Australia, and Scotland.

PurposeOncology drugs are often approved for multiple indications, for which their clinical benefit varies. Aligning a single price to this differing value remains a challenge. This study examines the clinical and economic value, price, and reimbursement of multi-indication cancer drugs across seven countries, representing different approaches to value assessment, pricing, and coverage decisions: the USA, Germany, France, England, Canada, Australia, and Scotland.MethodsTwenty-five multi-indication cancer drugs across 100 indications were identified with US Food and Drug Administration (FDA) approval between 2009 and 2019. For each indication data on Health Technology Assessment (HTA) recommendations, disease prevalence, and drug prices were obtained. Quality-adjusted life years (QALYs) gained, disease prevalence, list prices, and HTA outcomes were then compared across indications and regions.ResultsFirst approved indications provide a higher clinical benefit whilst targeting a smaller patient group than indication extensions. Quality-adjusted life year gains were higher for first (0.99, 95% CI 0.05–3.25) compared to second (0.51, 95% CI 0.02–1.63, p < 0.001) and third (0.58, 95% CI 0.05–2.07, p < 0.01) approved indications. Disease prevalence per 100,000 inhabitants was 20.7 (95% CI 0.2–63.3) for first compared to 27.1 (95% CI 1.5–109.6, p = 0.907) for second and 128.3 (95% CI 3.1–720.1, p < 0.001) for third approved indications. With each approved indication drug prices declined in Germany and France, remained constant in the UK, Canada, and Australia, whilst they increased in the USA. Negative HTA outcomes, clinical restrictions, and managed entry agreements (MEAs) were more frequently observed for indication extensions.ConclusionsResults suggest that indication development is prioritised according to clinical value and disease prevalence. Countries employ different mechanisms to account for each indication’s differential benefit, e.g., weighted-average prices (Germany, France, Australia), differential discounts (England, Scotland), clinical restrictions, and MEAs (England, Scotland, Australia, Canada). Value-based indication-specific pricing can help to align the benefit and price for multi-indication cancer drugs.Supplementary InformationThe online version contains supplementary material available at 10.1007/s40258-022-00737-w.

POSA280 Using Artificial Intelligence and Machine Learning to Inform Evidence Development for Optimising HTA Outcomes in Europe

Biopharmaceutical companies prepare for European Health Technology Assessment (HTA) submission of their products by understanding HTA guidelines and looking at past analogue examples, supplemented with primary market research. This work explores the application of advanced analytics techniques including AIML (artificial intelligence and machine learning) and their potential to improve understanding of submission factors leading to specific HTA outcomes.

POSC274 Does Conditional Marketing Authorisation Speed Up Patient Access?

Conditional marketing authorisation (CMA) facilitates early approval for medicines intended for seriously debilitating or life-threatening diseases whose benefit of immediate availability outweighs the risk of less mature clinical data. While CMA has the potential to accelerate patient access, the perceived value at the regulatory level may not translate to value at the health technology assessment (HTA) level. This research explores whether CMA can accelerate time to patient access while maintaining optimal reimbursement outcomes. HTA outcomes of medicines with CMA from 2018-2020 were analysed, followed by 30-minute, qualitative, in-depth interviews with payer advisors from France and Germany to predict the evolution of HTA frameworks to accommodate future CMA candidates. 22 medicines received CMA from the European Medicines Agency (EMA) from 2018-2020. Despite substantial unmet medical need across indications, CMA translated to poor HTA outcomes. Of 18 available HTAs in France, 8 medicines received a negative reimbursement recommendation of SMR ‘Insufficient’ (in primary indication or subgroup), while the remaining outcomes were split between ‘Important’, ‘Moderate’ and ‘Low’. Most of the reimbursed medicines demonstrated poor quality of evidence, and received ASMR V. Of 13 available HTAs in Germany, only one medicine achieved an added benefit rating, while most demonstrated ‘no’ or ‘non-quantifiable’ added benefit. Payers expect future CMA candidates will face similar reimbursement hurdles if market-specific HTA requirements are not met. While CMA accelerates regulatory approval, limited data packages create huge uncertainty in the appraisal of medicines. This leads to significant risk of unfavourable reimbursement outcomes and delays in patient access. While HTA frameworks continue to evolve over time, they are unlikely to reward immature data submissions particularly as payer bodies face ever-increasing funding constraints likely exacerbated by the COVID-19 pandemic. Manufacturers should ensure their drug development programs meet both regulatory and payer requirements to support accelerated patient access.

Similarities and Differences in Health Technology Assessment Systems and Implications for Coverage Decisions: Evidence from 32 Countries.

Health technology assessment (HTA) systems across countries vary in the way they are set up, according to their role and based on how funding decisions are reached. Our objective was to study the characteristics of these systems and their likely impact on the funding of technologies undergoing HTA. Based on a literature review, we created a conceptual framework that captures key operating features of HTA systems. We used this framework to map current HTA activities across 32 countries in the European Union, the UK, Canada and Australia. Evidence was collected through a systematic search of competent authority websites and grey literature sources. Primary data collection through expert consultation validated our findings and further complemented the analysis. Sixty-three HTA bodies were identified. Most have a national scope (76%), are independent (73%), have an advisory role (52%), evaluate pharmaceuticals predominantly or exclusively (76%), assess health technologies based on their clinical and cost-effectiveness (73%) and involve various stakeholders as members of the HTA committee (94%) and/or through external consultation (76%). The majority of HTA outcomes are not legally binding (81%). Although all study countries implement HTA, the way it fits into decision-making, negotiation processes, and coverage and funding decisions differs significantly across countries. HTA is a dynamic and transformative process and there is a need for transparency to investigate whether evidence-based information influences coverage decisions.Supplementary InformationThe online version contains supplementary material available at 10.1007/s41669-021-00311-5.

PCN169 A Retrospective Review of HTA Outcomes for Oncology Treatments Assessed in France and Germany from 2018 to 2020

Payers in France and Germany utilize an added benefit system to determine recommendations of new medicines. This study aims to analyze the latest trends in health technology assessment (HTA) outcomes published by the Federal Joint Committee (G-BA; Germany) and the National Authority for Health (HAS; France) for oncology treatments. An in-house Oncology HTA database containing over 1,200 HTA decisions for oncology treatments approved by the European Medicines Agency was used. A retrospective analysis of assessments from the G-BA and HAS in 2020 was conducted and compared to HTA outcomes in 2018 and 2019. The G-BA published guidance on 43 oncology treatments in 2020, where 25.6% were considered to provide no added benefit; a marked decrease from 50.0% in 2019. In the remaining submissions, 18.6% had non-quantifiable added benefit, 37.2% minor added benefit (a notable increase from previous years), and 18.6% considerable added benefit. No oncology treatments demonstrated major added benefit. HAS published guidance on 32 oncology treatments in 2020, where 18.8% of treatments were determined to have insufficient medical benefit (SMR). No clinical improvement (ASMR V) was reported in 46.9% of HTAs, an overall increasing trend since 2018. Minor improvement (ASMR IV) was reported in 18.8%, showing a gradual decrease from 32.3% in 2018 and 26.3% in 2019. Moderate improvement (ASMR III) was reported for 12.5% of treatments, lower than previous years. No treatments received an ASMR score of II or I (important or major). In 2020, 24 oncology treatments were assessed by both HAS and G-BA. There was general agreement in the HTA decisions from both agencies. There appears to be a trend of fewer treatments receiving the lowest benefit ratings from the G-BA, but not from HAS. Among the oncology treatments assessed by both agencies in 2020, general agreement on added value was observed.

Companies' Health Technology Assessment Strategies and Practices in Australia, Canada, England, France, Germany, Italy and Spain: An Industry Metrics Study.

Background: Health technology assessment (HTA) has increased in importance in supporting payer decision making by assessing the relative effectiveness and cost effectiveness of new medicines. Thus, pharmaceutical companies need to address the HTA requirements early during development to improve reimbursement outcomes. Currently, there is a lack of research to assess the impact of HTA on development and jurisdictional outcome from companies’ perspectives. This study aimed to assess companies’ HTA strategy and characterise HTA practice in seven jurisdictions. Methods: A multi-year, annual study collected information for individual products, focusing on development activities regarding inclusion of HTA requirements and selection of global comparators. The generation of local contextual information, submission strategies and predictability of HTA outcomes was examined jurisdictionally in Australia, Canada, England, France, Germany, Italy and Spain. The study questionnaire was built into a secure online data collection platform and data were provided annually by participating companies. Results: Data for 169 compounds were provided by nine international companies between 2014 and 2018. HTA requirements were implemented in evidence generation plan for 63% of products during development. Global comparators were accepted by HTA bodies for more than half of studied products; Spain showed the highest acceptance rate (85%). Companies took advantages of parallel process in Australia and Canada to shorten product rollout time. Australia demonstrated general consistency in HTA review time, and England had the longest variation (interquartile range, 216 days). Requirements for additional information after submission occurred at all HTA bodies. Germany and Italy showed the highest percentage of products being reimbursed as per regulatory label (80 and 68%, respectively). Canada was the most predictable jurisdiction, with the highest proportion of review outcome (90%) that met companies’ expectations. Conclusion: Companies are addressing HTA requirements during development for many products; however, they are challenged by varying requirements and practices and product success ultimately depends on how HTA organisations and payers assess added value in the context of the national healthcare systems. This ongoing study created a baseline to help capture fact-based changes for company HTA strategies and HTA body practices.

PPM7 Unlocking EARLY Access for One-Time Treatments

The rise of one-time therapies introduces new challenges for regulatory and payer bodies, including the often-overlooked considerations for early access (defined as access prior to Health Technology Assessment (HTA) or national coverage/reimbursement). This study aims to understand the current early access landscape in Europe and assess the adequateness/relevance of existing frameworks for innovative one-time therapies. A systematic review of local/regional early access frameworks across Europe was conducted by reviewing laws, guidelines and publications relating to early access at both the centralized and regional levels. Recent case studies of one-time therapies were then used to evaluate key challenges and the relative adequateness of existing frameworks for innovative one-time therapies. While a number of early access mechanisms exist in Europe to enable early access, the vast majority are designed to provide treatment free-of-charge: except for France and Italy, where the ATU and ‘326/’648 programs enable financial remuneration for early access, countries in Europe are broadly unaccustomed to paid early access. In fact, local regulations frameworks often mandate a free-of-charge period or make it challenging for financial remuneration prior to HTA outcomes as is exemplified by the EAMS compassionate use program in England or the 210-day mandatory free of charge period for PAP in Portugal. For one-time therapies, providing such products free-of-charge directly removes patients from the future patient pool and thus potentially deters cell and gene therapy (C&G) manufacturers from pursuing such programs (unlike the case for chronic therapies, where early access patients continue treatment post-early access). There is a clear need for payer/HTA bodies to evolve in order to accommodate new early access mechanisms for curative, one-time therapies. As demonstrated through recent successes with early access for C&G therapies in Europe, payers have demonstrated openness to adapting the system to accommodate the new treatment paradigm.

1584O Health technology assessment (HTA) in England, France and Germany: What do we know about variations in cancer-related HTA outcomes?

Variations in HTA outcomes can primarily be explained with reference to institutional context and administrative rules. Here we focus on the German Federal Joint Committee (Gemeinsamer Bundesausschuss, GBA), the French Haute Autorité de Santé (HAS) and the British National Institute for Health and Care Excellence (NICE) to identify matched drug pairs as a basis to compare HTA outcomes and to analyze cancer-related HTA results in detail.

PND89 NEGATIVE HTA RECOMMENDATIONS FOR MEDICINES IN NEUROLOGY THERAPEUTIC AREAS – FOURFOLD HIGHER ODDS IN POLAND COMPARED WITH THE UNITED KINGDOM

Technology appraisals (TA) by health technology assessment (HTA) bodies continue to pose a potential hurdle to broad access of innovative medicines in Europe, including Poland. Risk-sharing agreement (RSA) are a potential means of increasing chances of acceptance. The aim of this study was to compare TA outcomes of the Polish HTA body (Agencja Oceny Technologii Medycznych i Taryfikacji; AOTMiT) and UK reimbursement bodies (National Institute for Health and Care Excellence; NICE, Scottish Medicines Consortium; SMC) for medicines in neurology area (F01-G99 of the ICD-10-CM Codes) and analyze RSA trends in Poland. Published outcomes of TAs carried out by AOTMiT between 2015 and 2018 were reviewed and matched to the TAs carried out by NICE/SMC. Fisher's exact test for a 2x2 contingency table was carried out to obtain odds ratio (OR) for a negative HTA outcome. Recommendations on including RSA in positive HTA decisions by AOTMiT were analyzed in two periods (2018 vs. 2015-2017). A total of 76 HTA submissions were identified (47 from AOTMiT and 29 from NICE/SMC). The rejection rate was 40.4 % by AOTMiT and 13.8% by NICE/SMC. The OR for a negative HTA recommendation was 4.24 (95% CI 1.27 to 14.20, p=0.018) between AOTMiT and NICE/SMC. RSA were recommended in 90% of positive TAs by AOTMiT in 2018 compared with 33% in positive TAs in 2015-2017 (OR=2.4; 95%CI 0.47 to 12.61, not significant). Poland remains a challenging market for innovative neurology medicines with fourfold higher odds of HTA rejection compared to the UK. Increasing trend of positive HTA recommendations with suggestions on RSA may positively influence future acceptance rate by AOTMiT for innovative medicines in neurology area.

Impact of health technology assessment and managed entry schemes on reimbursement decisions of centrally authorised medicinal products in Belgium.

Centrally authorised medicinal products (CAMPs) in the European Union may offer added therapeutic value (ATV) but may be linked to high prices and limited efficiency. Health technology assessment (HTA) and managed entry schemes (MES) may facilitate the reimbursement decision by providing reliable estimates of the medicinal product's value and costs and by controlling the remaining uncertainty, respectively. We investigated the impact of HTA criteria and the initiation of a MES on the reimbursement decision of CAMPs in Belgium. We selected all reimbursement submissions for new centrally authorised medicinal products in the 2010-2015 period. We retrieved data relating to the reimbursement decision, the HTA outcome and the use of a managed entry scheme. The decision of the Minister was available for 115 dossiers, covering 36 (31.3%) orphan medicinal products (OMPs) and 79 ATV products. A MES was used in 41 submissions. A positive reimbursement decision was obtained in 65% of cases. The significant factors affecting the reimbursement decision were the approval of ATV, the medical need if it was considered 'important or major' and the use of a managed entry scheme. Price, budget impact and efficiency had no significant impact. Added therapeutic value and high medical need increase the odds for a positive reimbursement decision. No impact could be demonstrated of the cost-related HTA criteria. Cost elements may be biased by the use of a confidential MES. Without a MES, only 53% of the centrally authorised medicinal products, including OMPs, are reimbursed in Belgium.