HTA Processes Research Articles

Time from approval to reimbursement recommendations in healthcare systems with centralized HTA processes. Focus on Polish HTA agency

ABSTRACT Background To analyze the time from drug registration to reimbursement recommendations, we examined medicinal products, including new clinical indications, registered by the EMA between 2014 and 2019 across various therapeutic areas. Materials and Methods The Polish Agency for Health Technology Assessment and Tariffication (AOTMiT) was compared with 11 agencies in England, Wales, Ireland, Scotland, the Netherlands, Norway, France, Germany, New Zealand, Canada, Australia. A total of 1,942 recommendations published by 12 hTA agencies were analyzed. Results The time from registration to recommendation in Poland was statistically significantly longer than for the other countries. The analysis revealed noticeable differences in the time it takes from drug registration to recommendation across the countries included in this analysis. Analyzing trends from 2014 to 2019 in individual countries, it is noteworthy that in most agencies the median time from registration to recommendation is declining. Conclusions This may suggest improvements in the processes of the recommending authorities and the companies responsible for providing data for assessment. Despite Poland having one of the longest times from registration to recommendation among the countries analyzed, there has been a clear year-over-year decrease in the time to publication of reimbursement recommendations.

Key Issues of Economic Evaluations for Health Technology Assessment in China: A Nationwide Expert Survey

ObjectivesHealth technology assessment (HTA) is increasingly crucial in medicine price negotiations in China, yet previous appraisals revealed national discrepancies on key economic evaluation issues: willingness-to-pay threshold, pricing models for multi-indication medicines, and comparator selection principles. This study aimed to collect expert opinions on these issues for future HTA evaluations. MethodsA nationwide anonymous web-based survey encompassing experts across academia, HTA, consultancy/contract research organization/industry, service provider, and payer. In 2023, a generic invitation containing a web link to the questionnaire was disseminated via WeChat using convenience and snowball sampling. Agreement rates for questionnaire views were analyzed using descriptive statistics. The relationship between participants’ responses and demographics was examined using appropriate logistic models. ResultsA total of 303 responses were received from experts in 34 cities. Key expert views include a suggested base willingness-to-pay threshold ranging from 0.5 to 1.5 times gross domestic product (52.1% agreement); elevated thresholds for childhood diseases, rare diseases, end-of-life diseases, and first-in-class medicines (>78.0% agreement); a single pricing model for multi-indication medicines (60.4% agreement); consideration of multiple medicines as comparators (79.9% agreement); and avoiding the use of centrally procured medicines as comparators for medicines with a time to market of less than 3 years (71.0% agreement). Participants who are service provider had lower odds of selecting higher thresholds (odds ratio 0.26; P < .01) than responders from consultancy/contract research organization/industry. ConclusionsExpert views indicate the need for substantial changes in China’s current HTA methods, highlighting the need for increased investment in HTA processes and expertise cultivation.

Expanding access to high-cost medicines under the Universal Health Coverage scheme in Thailand: review of current practices and recommendations

BackgroundThere has been an increasing demand to reimburse high-cost medicines, through public health insurance schemes in Thailand.MethodsA mixed method approach was employed. First, a rapid review of select high-income countries was conducted, followed by expert consultations and an in-depth review of three countries: Australia, England and Republic of Korea to understand reimbursement mechanisms of high-cost medicines. In Thailand, current pathways for reimbursing high-cost medicines reviewed, the potential opportunity cost estimated, and stakeholder consultations were conducted to identify context specific considerations.ResultsHigh-income countries reviewed have implemented a variety of pathways and mechanisms for reimbursing high-cost medicines under specific eligibility criteria, listing processes, varying cost-effectiveness thresholds and special funding arrangements. In Thailand, high-cost medicines that do not offer good value-for-money are excluded from the reimbursement process. A framework for reimbursing high-cost medicines that are not cost-effective at the current willingness-to-pay threshold was proposed for Thailand. Under this framework, specific criteria are proposed to determine their eligibility for reimbursement such life-saving nature, treatment of conditions with no alternative treatment options, and affordability.ConclusionHigh-cost medicines may become eligible for reimbursement through alternative mechanisms based on specific criteria which depend on each context. The application of HTA methods and processes is important in guiding these decisions to support sustainable access to affordable healthcare in pursuit of Universal Health Coverage (UHC).

11.G. Skills building seminar: Navigating the ocean of data: unpacking ethical values in Health Technology Assessment

Abstract Health technology assessment (HTA) is multidisciplinary process that uses explicit methods to determine the value of a health technology at different points in its lifecycle. The purpose is to implement an informed decision-making process in order to promote an equitable, efficient, and high-quality health system. Ethics has been identified as a key element in HTA because health care is a moral endeavour and potential technology presents complex moral dilemmas, especially with the rise of novel technologies. In evaluating health technologies, ethical aspects must be taken into account such as patients’ privacy and informed consent in terms of (re-)use of their personal sensitive data, alongside with the possibility of unintended consequences (e.g., doing more harm than good: Thalidomide case) and the effects on society in terms of justice and equity, with implications for financial sustainability. Without taking account of the ethical dimension in the process, choices may be made without considering the overall context of implementation, thus, compromising the relevance of decisions and policies, with potentially unintended consequences and/or harmful effects to the health and wellbeing of individuals/patients, to public health, to the environment, and to the society at large. Decision-makers should guarantee that adopted health technologies are meaningful and relevant for the society, with due consideration to their development too, to the extent possible, by also taking ethical aspects into consideration when evaluating health technology. Despite many years of recognising ethics as a foundational part of HTA, studies have shown that the ethical dimension is still not systematically and sufficiently addressed by means of ethical analysis. Furthermore, novel technologies, new types of data, and a paradigm shift in terms of how we generate and synthesize evidence, whilst providing us with unique opportunities in terms of HTA, pose concrete challenges and new (and revisited) dilemmas. EUPHA-HTA, EUPHA-ETH and EUPHAnxt sections and HTAi are committed to build capacity amongst healthcare professionals about evidence-based decision-making with due consideration to ethical aspects. The aim of this workshop is two-fold. First, we will discuss the role of ethics in healthcare decision-making and tools to ensure the integration of ethics in the HTA process. We will then explore case studies and present a simple model with the contribution of EUPHA-HTA, EUPHA-ETH, and HTAi to illustrate ethical dilemmas that can arise during HTA processes. In the second half of workshop, attendees will be divided into groups to discuss ethical challenges in HTA and means to overcome them. We will capture the needs and input of young researchers/audience and move towards developing a EUPHA-HTAi priority map for competency mapping as output. Key messages • Systematic real-world data collection and context-specific analysis of the ethical principles coupled with legal and social analysis contributes to good decision making and policies. • Ethical analysis is an integral part of HTA, ensuring that the value=laden use of a health technology is made transparent, identifying ethical consent and dispute. Speakers/Panelists Elena Petelos EUPHA-HTA, EUPHA-GH Pietro Refolo Università Cattolica del Sacro Cuore, Rome, Italy Els Maeckelberghe University Medical Center Groningen, Groningen, Netherlands Wija Oortwijn HTAi

Shaping a suitable EU HTA dossier template: why the German template is not fit for purpose

From 2025, Health Technology Developers (HTDs) have to submit EU HTA dossiers. The joint clinical assessment (JCA) aims to streamline HTA processes and access to medicinal products across Europe. Currently, German HTA bodies IQWiG and G-BA actively shape the JCA methodology. Here we examine if German HTA dossier requirements are suitable for the JCA. We compare the number of safety endpoint and subgroup analyses in German dossiers with analyses considered in IQWIG’s benefit assessment and evaluate if these analyses were considered by the G-BA. We further investigated how the number of analyses was affected by the latest change in the German dossier template. With the current template, HTDs report in median 2.6 times more analyses on adverse events (AE) and 1.1 times more subgroup categories than in the previous template. IQWiG does not consider 33% of AE analyses and 73% of the subgroup categories presented by the HTD under the current template. G-BA considered the same AE as IQWiG in 76% of cases. Subgroups were uncommented by G-BA in most cases, independent of the template (previous: 93%, current 85%) and unconsidered in the conclusion on additional benefit (previous: 77%, current 69%). Thus, changes in the dossier template drastically increased HTD workload, but additional analyses seem unconsidered by the HTA bodies. With a broader scope in JCA, this effect could be amplified. To mitigate duplicative efforts and ensure prompt availability of medicinal products as envisioned by the HTAR, we suggest well-chosen and precise dossier requirements, early consultations, and early HTD engagement.

The development of HTA collaboration at European level and the challenge of the EU regulation

Abstract Issue The establishment of HTA has been an important topic in Europe for many years. The engagement of HTA agencies, scientists, politicians and many other stakeholders throughout more than 20 years lead to the EU-HTA regulation, which came into force beginning of 2022. Now it is time to prepare for a sustainable European Network for HTA. Description The implementation of HTA in Europe is of high importance of the EU-commission for more than 20 years. Started with the first projects in 1994 (EUR-ASSESS) it needed quite a bunch of different projects and, in the end, three joint actions for finally providing legal ground for HTA with the EU-HTA regulation. This journey seemed a long one. However, referring to the need of addressing the requirements of Health Care of 27 Member States combined with the demand of reliable high methodological quality and applicability, investing all the time and effort for a sustainable European Network of HTA was definitely worthwhile. Joint Scientific Consultation (JSC) and Joint Clinical Assessments (JCA) will play a central role for national HTA and decision making in health care. But for understanding role, content, national uptake and further development of means, products and results of a European Network for HTA it is quite important to get a perception of the development of HTA collaboration in Europe. Based on this the challenge of the EU-HTA regulation and its implementation will be tackled easier. Effects Through provision of the main pillars of the previous EU-HTA collaboration the appreciation and understanding of the differences and complexities behind the HTA processes in the EU-healthcare systems will made aware. Based on this understanding the challenges of the implementation of the EU-HTA regulation will be discussed.

A Conceptual Framework for Life-Cycle Health Technology Assessment

ObjectivesHealth technology assessment (HTA) uses evidence appraisal and synthesis with economic evaluation to inform adoption decisions. Standard HTA processes sometimes struggle to (1) support decisions that involve significant uncertainty and (2) encourage continued generation of and adaptation to new evidence. We propose the life-cycle (LC)-HTA framework, addressing these challenges by providing additional tools to decision makers and improving outcomes for all stakeholders. MethodsUnder the LC-HTA framework, HTA processes align to LC management. LC-HTA introduces changes in HTA methods to minimize analytic time while optimizing decision certainty. Where decision uncertainty exists, we recommend risk-based pricing and research-oriented managed access (ROMA). Contractual procurement agreements define the terms of reassessment and provide additional decision options to HTA agencies. LC-HTA extends value-of-information methods to inform ROMA agreements, leveraging routine, administrative data, and registries to reduce uncertainty. ResultsLC-HTA enables the adoption of high-value high-risk innovations while improving health system sustainability through risk-sharing and reducing uncertainty. Responsiveness to evolving evidence is improved through contractually embedded decision rules to simplify reassessment. ROMA allows conditional adoption to obtain additional information, with confidence that the net value of that adoption decision is positive. ConclusionsThe LC-HTA framework improves outcomes for patients, sponsors, and payers. Patients benefit through earlier access to new technologies. Payers increase the value of the technologies they invest in and gain mechanisms to review investments. Sponsors benefit through greater certainty in outcomes related to their investment, swifter access to markets, and greater opportunities to demonstrate value.

Innovative Patient Involvement During Covid-19: Keeping Patients at the Heart of HTA.

The COVID-19 pandemic and lockdown measures in the United Kingdom resulted in significant challenges and created opportunities for innovation to keep patients at the heart of HTA. The introduction of the Coronavirus Act 2020 and the associated public health guidance meant that NICE's conventional HTA methods were no longer feasible. NICE introduced rapid, innovative updates to patient and public involvement (PPI), decision-making meetings, and consultations to harness the expertise of patients and the public to ensure guidance addressed the expected concerns and identified barriers which could impact access. This article describes the PPI support for NICE's rapid shift to virtual meetings and virtual engagement. We utilize the authors' experience and patient and public contributor feedback to understand the experience of participating in a virtual setting and identify four themes: accessibility; inclusivity; transparency; and intrapersonal relationships and committee dynamics. The article also considers how patient representatives participated in, and facilitated, the development of guidance for a hypothetical technology to keep patients and the public at the heart of expedited and novel HTA processes to identify and understand the expected patient concerns and potential barriers for when a technology would be introduced.

PCN145 Clinical Pathways As Input to HTA Processes: Lessons from the Experience of a Referral Cancer Centre

Over 95,000 patients annually receive medical attention at Arturo López Pérez Foundation Oncology Institute (FALP). In 2019, to address the challenges faced by decision makers regarding oncological interventions, the FALP Health Technology Assessment unit (FALP-HTA) developed a process to design and implement clinical pathways (CPs).

PNS72 Charting the Progress of HTA in Asia: Are We Ready for Evidence Based Decision-Making?

This paper provides an update on the growing use of HTA in Asia and lessons for other countries seeking to advance HTA. This paper builds upon the research by Chootipongchaivat et al and the World Health Organization (WHO) identifying 18 “factors conducive to the development of HTA in Asia”. These factors were used to create a balanced scorecard to assess the progress of HTA. The authors scored the progress against each of the factors made in China, India, Indonesia, Malaysia, Philippines, South Korea, Taiwan, Thailand, and Vietnam. A scoring system on a scale of 1 to 5 was used wherein: 1=No progress on milestone; 2=milestone at early stages, ad hoc HTA use; 3=progress on milestone, routine HTA but limited impact; 4 = significant progress on milestone, routine HTA use but limited remit; and 5 = significant progress on milestone, routine HTA use that informs several decisions. Total scores indicated progress of HTA while milestone scores provided contextual insights within countries. A literature review and expert interviews were used to complete scorecards. South Korea and Thailand had highest scores of 73 and 71 points respectively, while Vietnam scored lowest at 28.5. Advanced HTA programs, such as Thailand and South Korea, have independent HTA agencies with a broad remit, explicit process and methods, network of researchers, and routine use of HTA. Countries like Taiwan and Malaysia fall in a middle tier, with established HTA programs with limited remit. The final tier consists of countries like China, India, Indonesia, Philippines, and Vietnam, emerging HTA processes. Universal Health Coverage (UHC) goals have catalyzed expansion of HTA. However, political will, technical expertise, and sustained financing remain challenges for sustainable HTA programs. While legislation supporting HTA is helpful, political will is key. Authors provide recommendations on areas for additional investments and opportunities for regional collaboration.

PDG45 “the Domino Effect”: An International Comparison of Economic Evaluation Submission Sequences in Advanced Melanoma

Pharmaceutical companies apply for reimbursement at different times in every country. The rationale for such practices resides in opaque profit-maximising launch strategies, influenced by factors like reference pricing. The objective was to identify sequences in the timing of drug economic evaluation submissions across the world. First, relevant economic evaluation bodies for medicines’ reimbursement, or lack thereof, were identified. Advanced melanoma was chosen as a recent therapy area with few drugs and homogeneous indications to validate our hypothesis before widening focus. Relevant databases were found and searched for the main treatments: nivolumab, pembrolizumab, ipilimumab, dabrafenib, trametinib, cobimetinib, vemurafenib, and their combinations. Submission dates were collected and ranked. Each country has its specific reimbursement process, with few countries leveraging economic evaluation. Those who do seldom share their conclusions. Even fewer publish details about the timelines of assessments, especially for older products. When they are available, methodological difficulties arise from interpreting the different types of dates published. Based on the data available, we estimated the months of submission for Canada, Brazil, England, France, Ireland, New Zealand, Norway and Romania. Submissions for treatments were made in a different sequence for all products and no trend can be identified, even for the same company, and some countries are not applied to. The main commonality is that England is generally one of the first submission countries. Pharmaceutical companies’ launch strategies seem to go beyond a set sequence. Sequences cannot be identified through economic submissions for advanced melanoma products. This reflects varied reimbursement practices but also a lack of transparency conceivably politics or resource-driven. The disparities between countries highlight the need for harmonisation that is an incentive for agencies to collaborate through organisations like EUnetHTA. Disclosing information used for decision making is key to involving more stakeholders like patients in HTA processes and improving accountability.

PMU64 HTA Best Practice for Inpatient Therapies: Comparing Sweden and Netherlands

Several markets have very distinct HTA processes for therapies used in the outpatient versus inpatient setting. In Sweden, the HTA body (the TLV) issued recommendations for outpatient medicines but inpatient drugs were reimbursed without any pharmacoeconomic assessment by each county council. Similarly, in the Netherlands the HTA body (CVZ/Zorginstituut) appraised outpatient therapies, with inpatient medicines reimbursed automatically upon marketing authorisation. However, both Sweden and Netherlands introduced new processes for inpatient medicines. In Sweden, since 2011, a national coordinating group of Swedish county councils (NT) can request selected in-patient therapies undergo a health economic assessment by the TLV, based on which the NT conduct price negotiations and issue a national recommendation. In Netherlands, a ‘lock’/sluice was introduced in July 2015 for high cost inpatient medicines for financial negotiations prior to reimbursement. This research compares inpatient drugs evaluated by Sweden’s NT and Netherland’s ‘lock’ system. All publicly-available NT recommendation and reimbursement ‘lock’ documents were screened and key information extracted (21/01/2015-29/06/2020). In Sweden, 113 drug:indication pairings were evaluated by the NT council. 52% (59/113) were in oncology and 20% (23/113) were for rare diseases. 43% (49/113) NT decisions were recommendations, 17% (19/113) restricted recommendations (special price discount agreements [5/19], restrictions in reimbursed population [4/19]), 23% (26/113) rejected, and 17% (19/113) other (deferred/unclear). In Netherlands, 36 therapies were identified that were subject to the ‘lock’ system. 78% (28/36) were for oncology and 50% (18/36) were for orphan indications. 58% (21/36) had been removed from the lock after an average of 11.1 months (range: 1.2-21.7). Many more therapies are being appraised under the NLT system in Sweden, which applies to all new inpatient treatments than the reimbursement lock system in the Netherlands, which only applies to high budget impact inpatient therapies.

Patient participation and representativeness in HTA processes

Abstract The final speaker will discuss the participation of patients in deliberative processes and HTA, along with what patients, their carers, but also survivors, envision in terms what constitutes a fair process, equitable access and fair pricing, at a time when out-of-pocket catastrophic payments are on the rise across developed nations, whereas access to innovative treatments is seriously hindered by the forbidding price they carry.

PCN263 ONCOLOGY DRUGS - INNOVATION ON HIGH SPEED

The number of new pharmaceuticals with a marketing authorization is on rise and moreover they increasingly target small populations. This trend is sharply outlined in the field of oncology. The aim of this study is to provide a better understanding of the timeliness of HTA processes in Bulgaria and to illustrate the implications with regard to delays in market and patient access of innovative oncology drugs.

Совершенстование системы оценки технологий здравоохранения в Казахстане с целью принятия решений в области здравоохранения

health field in many developed countries. Modern tendencies give plenty of evidence of wide development of HTA processes and their implementation on a global scale. Along with these tendencies, there are number of issues that hinder active implementation and use of HTA worldwide including Kazakhstan. Major issues that limit further development and implementation of HTA processes in our country are lack of human resources, evidence-based medicine data, and tight deadlines for HTA reporting. Furthermore, they affect the quality of HTA reports followed by inadequate decision-making by the members of the Joint Commission for the Quality of Health Services and overall decreased collaboration with the international HTA agencies. To resolve these issues in the HTA system development, we propose policies that involve training and re-training specialists for the HTA field starting from undergraduate and postgraduate education. We also suggest optimization of HTA structures funding and enhancement of regulatory framework, which, in turn, control not only HTA processes, but also decision-making based on HTA results.

PNS247 THE IMPLICATIONS OF EUROPEAN JOINT CLINICAL ASSESSMENT FOR MEMBER STATE PRICING & MARKET ACCESS

In January 2018, the European Commission proposed a new mandatory clinical efficacy assessment to be facilitated at the EU level for use by Member State HTA authorities as part of national or regional HTA processes. Following amendments to the proposal in the European parliament, implementation is now being discussed in the European Council. The objective of this research is to determine requirements for the practical implementation of the Joint Clinical Assessment into national level pricing and market access processes and implications for patient access and uptake for new medicines. An in-depth online consultation was undertaken with a representative sample of former HTA and budget holding stakeholders from across Member States, to determine the implications of the mandatory European Joint Clinical Assessment for national HTA processes, timelines, evidence requirements and pricing agreements. Whilst the potential for a central EU assessment of clinical benefit to improve efficiency, reduce duplication and accelerate patient access is recognised, considerable uncertainties remain regarding the value and implementation of the assessment reports at the national level. Priority issues for national HTA stakeholders are ensuring accurate representation of the burden of disease, the assessment of incremental benefit vs. a nationally relevant comparator, and ensuring definitions of ‘patient relevant endpoints’ and acceptable clinical study designs align with currently accepted standards. Under the framework for the Joint Clinical Assessment, voluntary collaboration between member states to undertake pricing negotiations is also predicted to increase. Mandatory European Joint Clinical Assessment represents a significant opportunity to improve the efficiency of European HTA processes and accelerate patient access to new medicines. In order for key benefits to be realised, the value of the methodologies and standards for evidence assessment must be recognised and agreed at Member State level to directly support the efficiency, scope and quality of national level decision making.

PCN365 TITLE: REVIEW OF PUBLISHED EFFICIENCY OPINIONS OF THE FRENCH ECONOMIC AND PUBLIC HEALTH ASSESSMENT COMMITTEE (CEESP) IN ONCOLOGY

Since 2013, innovative products claiming an added clinical benefit (ASMR) level ≤III with a substantial impact on healthcare expenditures (≥€20 million the second year) are evaluated by the French economic and public health assessment committee (CEESP). The study objective was to review and analyse the CEESP opinions in oncology indications and to summarize the major concerns. All available CEESP opinions for oncology indications published until May 2019 were reviewed. Specific indications, nature of the request, model type used for cost-effectiveness analysis, and methodological concerns were extracted. Overall, 38 CEESP opinions out of 88 opinions published on the HAS website were based on cancer treatment assessment (43%), including 14 in hematology (37%), with 71% of them being intended for first inscription (10/14). One third (8/24) of the reports evaluated in solid tumors (12 for extension and 9 for first inscription) were for lung cancers. The majority of the manufacturers used a partitioned survival model to assess the cost-effectiveness of their product (27/38). 64% (9/14) of the reports in hematology received ≥1 major concern, vs. 21% (5/24) of the opinions in solid tumors. Half of a total of 22 major concerns related to lack of robustness and uncertainty of clinical effectiveness. Lack or inconsistency in relevant strategies lead to a major concern in 3 reports. Other major concerns were associated to non applicability of health outcomes (3/22), non relevant simulated population (2/22), lack of sufficient sensitivity analyses (1/22), poor quality of utility data (1/22) or anonymity of the independent clinical experts solicited (1/22). While cancer remains the main area for innovative products, oncology therapies face challenges around uncertainty in the clinical effectiveness evidence. The specificities of the evidence that is available for innovative cancer drugs, in particular immunotherapies, may require special consideration within HTA processes.

PCN287 THE ROLE OF EFFICACY AND EFFECTIVENESS ANALYSES IN HEALTH TECHNOLOGY ASSESSMENTS IN DETERMINING TENDERING RANKS FOR ONCOLOGY DRUGS IN DENMARK & NORWAY

The Danish Medicines Council and Norwegian Medicines Agency appraise pharmaceuticals on clinical and economic grounds. Following recommendations from these institutions, manufacturers enter tendering processes which result in rankings of drugs within an indication. The influence of clinical outcomes on these ranks in oncology in practice has not been assessed. This review aims to determine whether efficacy/effectiveness assessments from HTAs are a key driver of the rankings in Denmark and Norway. Tendering ranks for oncology indications in both countries as of May 2019 were extracted. Indications where at least two different drugs/regimens were ranked were included. HTA reports for the included regimens were identified from agency websites, and preference orders presented by the appraisers based on clinical outcomes and other reported decision criteria (e.g. cost-effectiveness) were extracted, if reported. Were these not available, overall survival and progression-free survival outcomes were extracted and rank orders generated using surface under the cumulative ranking (SUCRA) method in network meta-analyses. Rank orders based on clinical data and HTA reports were compared to tendering rank orders and reasons for differences explored. Across both countries, 26 multiple-regimen ranks were being used in oncology where at least one corresponding HTA had been conducted. In most cases, the most efficacious medicines were included in tendering ranks, but in 38% of indications, efficacy ranks were not aligned with tendering ranks. Possible justification for differences relates to added uncertainty in indirect comparisons and interpretation of clinical evidence. The HTA processes in Denmark and Norway allow market access for the most effective drugs, however it appears to have less of a role in treatment prioritisation in oncology. Tendering presents one method for cost containment for pharmaceuticals, but the impact this has on patient access to efficacious treatments in oncology and other indications needs to be evaluated further.

PMU95 IS THERE REGIONAL VARIATION IN WORSE AND SLOWER COST/QALY HTA OUTCOMES FOR COMBINATION PRODUCTS INVOLVING TWO VS. ONE MANUFACTURER?

Combinations of existing therapies are increasingly being developed and marketed across many indications. Whilst they promise substantial clinical benefits, dual-branded therapies may have substantial financial impacts. Obtaining positive HTA approvals and public reimbursement may be a major challenge, which may be amplified if each monotherapy is marketed by a different company. We evaluated whether combination therapies developed by two manufacturers had slower and/or worse outcomes than those where each constituent was developed by a single manufacturer in HTA bodies that use cost-effectiveness as a key decision-making criterion and whether this varied by based on region. EC-approved combination products, comprising at least one on-patent therapy marketed under a separate brand were identified (01/01/2015–5/6/2019) and the number of manufacturers identified. Corresponding HTA reports were identified from CADTH, NCPE, NICE, PBAC, and SMC websites and relevant data extracted. 169 HTA assessments across 35 combination products:indications were identified (same manufacturer: 91 assessments, two manufacturers: 78 assessments). For both EU and ex-EU HTA bodies, the rate of positive recommendations was higher for combination therapies developed by one vs. two manufacturers (EU: 82% vs. 57%; ex-EU: 83% vs. 63%), with corresponding shorter median time from regulatory approval to positive recommendation for one vs. two manufacturers (EU: 5.0 vs. 6.5 months; ex-EU: 3.0 vs. 5.0 months). HTA bodies assessing cost-effectiveness were more likely to issue positive recommendations, with shorter time to positive recommendations, for combination therapies if each constituent monotherapy was marketed by one company vs. two companies, which was consistent between EU and ex-EU. However, the time to positive recommendation was numerically faster across the board ex-EU vs. EU which may reflect the parallel regulatory and HTA processes in Australia and Canada.

Striking a balance between accuracy and timeliness in HTA: an elusive task

Abstract Issue HTA is a decision-making tool. Users need high-quality and timely evidence to ensure sound resource allocation and timely access to innovation. High-quality HTA recommendations are time-consuming to generate, requiring high-quality evidence and timely evidence synthesis, as well as mechanisms to ensure timely recommendation release and feasible implementation of guidance. Description of the problem HTA submissions, issuing recommendations and implementation guidance oftentimes have no transparent timelines, with high accuracy posing unique challenges for timeliness. We can broadly examine such challenges in the context of methodologies and processes. Accuracy ensures longer-term validity of decision-making, however, HTA should not be used only for new technologies but also revision of previous decisions through timely re-assessment, ensuring obsolete technology disinvestment. Effects/changes Through the exploration of models and tools previously implemented across jurisdictions and settings, such as early-warning systems, conditional approvals and horizon scanning, it will be demonstrated that well-defined prioritisation criteria accepted by both doers and users, adequate capacity and sound preparation of stakeholders are all key elements to ensure the highest possible degree of timeliness and accuracy, and to strike a balance between the two. Particular attention will be paid to critical challenges for public health access and delivery, i.e., emerging technologies and complex interventions, highlighting how horizon scanning ought to start at the discovery, rather than development phase and transparency challenges. Technology tracking and mechanisms for standardizing methodologies and processes will also be discussed. Lessons Monitoring and evaluating the impact of HTA methodologies and processes in a transparent manner needs to become an inherent part of HTA to ensure existing and future challenges of accuracy and timeliness can be adequately addressed.