While targeted therapies have substantially improved survival rates for non-small-cell lung cancer (NSCLC), it remains the leading cause of cancer mortality in the US. Companion diagnostics (CDx) measuring programmed death-ligand 1 (PD-L1) expression help inform NSCLC treatment but have limited accuracy. We assessed the potential value of a more accurate PD-L1 CDx for multiple stakeholders.

We developed decision tree models to assess the potential cost-effectiveness of a hypothetical new CDx for atezolizumab, a PD-L1 inhibitor used as a first-line therapy for metastatic NSCLC and as an adjuvant therapy for stage II–IIIa NSCLC patients. The sensitivity and specificity of current PD-L1 assays as well as cost and health payoffs were estimated based on data extracted from the scientific literature. We calculated incremental cost-effectiveness ratios (ICERs) for both indications, conducted headroom and threshold analyses, and used model outputs to estimate the size of the US serviceable addressable market (SAM) for a new PD-L1 CDx in the adjuvant indication.

Approximately five percent of metastatic and seven percent of stage II–IIIa NSCLC patients currently tested for PD-L1 expression receive false negative results. An equivalently priced 100 percent accurate PD-L1 CDx would add an average of 0.04 quality-adjusted life years (QALYs) and cost USD6,069 more per metastatic NSCLC patient (ICER: USD144,512/QALY gained; 95% confidence interval [CI]: USD74,178, USD206,937). It would add 0.08 QALYs and cost USD3,682 more for stage II–IIIa NSCLC patients (ICER: USD49,031/QALY gained; 95% CI: USD47,104, USD50,064). The maximum value-based price in stage II–IIIa NSCLC (assuming a USD100,000/QALY willingness-to-pay threshold) would be ˜USD4,000/patient. At a USD500 price per unit, the SAM would be ˜USD6.5M/year.

Because existing PD-L1 assays for NSCLC are optimized for specificity, the cost-effectiveness of a more accurate CDx reflects that of the immunotherapy drug it is paired with. Stage II–IIIa NSCLC has much greater innovation headroom for a new PD-L1 CDx than metastatic NSCLC. Early cost-effectiveness modeling can identify CDx use cases with higher value headroom and help inform market sizing.

External reference pricing (ERP) serves as a widely adopted method for establishing authorization and reimbursement prices for pharmaceuticals in Europe. However, its application within the medical device (MD) industry remains shrouded in uncertainty. This study seeks to investigate the feasibility of integrating ERP into the realm of MDs, identify pertinent challenges and ambiguities, and propose solutions to bolster affordability and accessibility.

The methodology encompassed a systematic search across diverse databases (Google Scholar, Google, ResearchGate, PubMed), utilizing combinations of relevant keywords, like “medical device,” “external reference pricing,” “price,” “reimbursement,” “affordability,” “challenge,” and “implement.” Articles were thoroughly evaluated by each researcher independently, followed by a consensus-based approach for final assessment. Rigorous scrutiny of relevance, potential insights, study design, and methodology ensured a comprehensive review, maintaining a high standard of research quality. This meticulous process guaranteed the inclusion of pertinent literature on ERP and MD pricing, enhancing the study’s depth and breadth.

Global ERP application in the MD sector exhibits significant variability in country selection, pricing methods, and adaptations to market changes. Pricing and reimbursement policies across international contexts contribute to this diversity, involving authorization procedures, regulatory oversight, technology appraisals, and state financing limitations. Challenges in establishing equitable pricing frameworks underscore the need for clear guidelines, regulatory empowerment, regular audits, and international collaborations. Transparent methodologies for reference price calculation are essential. Addressing observed shortcomings involves negotiating lower prices, implementing subsidies, promoting generic competition and local production, and tailoring reimbursement policies to treatment needs, enhancing affordability and accessibility on a global scale.

Ambiguities in implementing ERP for MDs underscore the necessity of equitable pricing frameworks. The highlighted multidimensional strategies and consideration of market dynamics are deemed essential for the feasibility of ERP in MDs. These insights emphasize the cruciality of a structured and transparent roadmap for optimizing ERP in the realm of MDs, holding significant potential for ensuring affordability and accessibility.

The first phase of a health technology assessment (HTA) is to define the scope of the assessment and choose the appropriate type of studies. The HTA in Regulation has been produced in cooperation with the Oswaldo Cruz Foundation (Brasilia) and the Brazilian Health Regulatory Agency (Anvisa). This work reports the Brazilian experience with HTA priority-setting within the time period 2022 to 2023.

Focusing on Anvisa’s 2022 to 2030 research themes, a workgroup developed a priority-setting cycle addressing the following four steps: (i) survey application to define problems and topics (August to October 2022); (ii) data compilation and exploratory searches by topic; (iii) workshops with technical areas (March to June 2023), aiming to refine themes, define research questions, and determine what types of studies will be conducted); and (iv) presentation of prioritized themes.

Forty-six topics at the intersection of HTA and health regulation were prioritized. Macro-agendas for medical devices and medicines were the most frequent. The studies (39 scoping reviews, nine rapid HTAs, and four technical reports) aimed at post-market surveillance, definition of regulatory problems, selection of the best regulatory options, and identification of the legal basis for the selected problem. These studies will be used to assess the impact of the proposed solutions on stakeholders and identify any potential risks associated with them. Lastly, the studies will provide recommendations for decision-making regarding health regulatory measures.

By establishing technical cooperation between stakeholders to gather the best available evidence for regulatory decision-making processes through HTA studies, scoping definition based on the real needs of decision-makers has taken place. In this collaboration, experts were mobilized to expand response capacity and technical-scientific knowledge to support decision-making in health regulation.

Starting from 2030, vaccines in the European Union (EU) require joint clinical assessments (JCAs), and joint scientific consultations (JSCs) can be requested from 2025. Involvement of national immunization technical advisory groups (NITAGs) is crucial to address vaccine specificities. However, NITAGs are currently not considered in the EU health technology assessment (HTA) framework. This study highlights potential risks for non-applicability of vaccine JCA reports nationally.

This study examined where in the JCA and JSC implementation process NITAGs could play an important role and highlighted the practical challenges of incorporating vaccine JCA reports into already very heterogeneous and complex vaccine access pathways across EU Member States. The EU HTA Regulation process was tested for three countries with different vaccine market access characteristics. This study was conducted using JCA guidance documents, NITAG vaccine assessment guidelines, and communications with the respective decision-making bodies.

The EU HTA Regulation framework requires adjustments for vaccine-specific considerations. NITAGs across the EU vary in experience, resources, expertise, and influence on decision-making regarding vaccine assessment and inclusion in the national immunization program. In addition to JCA and JSC, the EU HTA framework also covers horizon scanning, which several NITAGs are currently involved with at national level.. However, the EU HTA framework currently lacks explicit requirements for NITAG input in horizon scanning, JSC, and JCA processes. This could lead to unnecessary duplication of work, further complexity of the processes, and lengthening of population time to access to new vaccines.

The EU HTA framework of vaccines aims to avoid duplicate efforts and enhance patient access, but current processes that will be introduced may not achieve this optimally. Early and systematic inclusion of NITAGs in the horizon scanning, JSC, and JCA processes is pivotal to mitigate the risk of non-applicability and to successfully realize the objectives of the EU HTA framework.

Infertility affects one-sixth of women worldwide, with over seven million assisted reproductive cycles performed annually. Oral dydrogesterone is recommended alternatively for luteal phase support in in vitro fertilization (IVF), preventing miscarriages and improving live birth rates. This study aims to develop an outcomes model comparing oral dydrogesterone treatment with the standard of care in the IVF cycle over a 10-year period.

A two-level Markov cohort model in Microsoft Excel includes six health states: IVF, pregnancy, miscarriage, live birth, perinatal death, and maternal death. Miscarriage, live birth, and perinatal death are sub-states of pregnancy. Transition probabilities are based on published rates with medical intervention limited to the first 12 weeks of gestation. A sensitivity analysis of treatment was performed. Data from a published meta-analysis of nine dydrogesterone studies for IVF luteal phase support were used. The baseline cohort is 10,000 Australian females undergoing IVF annually over a 10-year period.

Over the 10-year time horizon, compared to standard care, the group treated with dydrogesterone was estimated to increase the number of live births by 3.5 percent (range: 3.4 to 3.7%), reduce the number of miscarriages by 69.4 percent (range: 66.2 to 72.7%), reduce the perinatal death by 10.9 percent (range: 10.4 to 11.4%), reduce the IVF cycles by 11.56 percent (range: 11.0 to 12.11%), and reduce the death of the mothers by 10.9 percent (range: 10.4 to 11.4%).

The outcomes model projected that treatment with oral dydrogesterone significantly reduced the number of miscarriages and improved the number of live births compared to the standard of care used for IVF patients in Australia.

As health technology assessment (HTA) becomes vital in healthcare decision-making, the demand for specialized education grows. A new HTA course, tailored to the specific needs of the Republic of Moldova’s health system , was launched under a joint project—a collaboration between the World Bank, the Swiss Cooperation Office, Radboud University Medical Center, and the School of Public Health Management. The course targets first- and second-year master’s students, and health professionals enrolled in continuing education.

The course aimed to introduce the fundamentals of HTA. Participants included 49 master’s students and 26 health professionals. A pre- and post-test model was employed, with participants completing a 10-(multiple) question HTA knowledge assessment at both the start and end of the course.

Initial pre-test results showed an average score of 30 percent, reflecting limited prior HTA knowledge. Following course completion, the post-test average escalated to 80 percent. This 50 percent increase in knowledge was consistent across student and professional groups. While a significant 79 percent of participants accurately answered questions about the use of HTA reports from other jurisdictions, identification of stakeholders, and the elements of establishing PICO (population, intervention, comparator, outcome), they found it more challenging to understand the deeper aspects of HTA. Notably, about 72 percent had difficulties with questions related to the main goals of HTA, evaluating the broader impacts of health technologies, and starting the evidence-based deliberative decision-making process.

The HTA course successfully covered basic concepts, yet it also highlighted the need for more comprehensive teaching of complex topics. The participants showed varying levels of understanding. This underscores the necessity for an HTA curriculum that equally emphasizes fundamental knowledge and in-depth analysis, preparing future healthcare professionals for complex decision-making in their roles.

Patient organizations play a crucial role in health technology assessment (HTA), especially for rare diseases. Despite their important role, the contributions and impact of patient organizations have been overlooked in the literature. This study aims to address this gap by examining the contributions of patient organizations and their nominated experts in National Institute for Health Care and Excellence (NICE) highly specialised technology (HST) appraisals.

We thematically analyzed the scope and frequency of contributions from patient organizations and experts associated with 10 NICE HST appraisals completed between January 2022 and September 2023. First, to allow for a representation of what patient contributions focus on, their written submissions were categorized according to themes following a deductive/inductive approach, employing a tiered system for disease-, technology-, or submission-specific themes. Second, we compared the themes identified in written submissions with those found in the final NICE recommendations, included in the final evaluation determination (FED), to understand whether and to which extent patients’ contributions were considered.

From 2013 to 2023, 22 drugs underwent HST assessment, with nearly half appraised during 2022 and 2023. All technologies received positive recommendations. A total of 475 unique patient contributions—from both patient organizations and their nominated experts—were identified in their written submission in support of the 10 HST appraisal assessed, predominantly emphasizing disease-specific themes (53%), such as quality of life. While 42 percent of raised themes aligned with FED content, 52 percent did not. When looking at individual appraisals, the share of themes mentioned in patients’ written submission explicitly considered in the FEDs ranged from nine percent to 73 percent, with a median of 50 percent.

Despite progress in integrating patient inputs into HTA, this study reveals a discrepancy between patient priorities and explicit consideration in NICE’s final recommendations. While NICE consistently adheres to its methodology, certain patient-raised aspects are overlooked. Further research is important to discern the optimal areas and timing for patient contribution, refining NICE’s involvement strategies in their decision-making processes.

The new Regulation (EU) 2021/2282 on health technology assessment (HTAR) highlights the increasing importance of patient perspectives, emphasizing their crucial role in HTA. The European Capacity Building for Patients (EUCAPA) project aims to empower patients for active involvement in HTA. EUCAPA focuses on equipping patients with capacities through the development of HTA training, including introductory, fast-track, and extended training programs.

Within the EU-funded EU4Health program, the EUCAPA consortium was tasked with capacity building for patients and patient representatives. Training programs were developed using a coproduction approach, integrating patient representatives’ perspectives into both curriculum development and the design of training sessions. This innovative approach involved close collaboration between patient representatives and HTA scientists, jointly shaping the program content. The curriculum was designed to incorporate the heterogeneous medical and cultural backgrounds and varying levels of prior knowledge among participants, recognizing diverse experiences that individuals bring to the training. To enhance accessibility, a blended learning approach with offline, virtual, and in-person parts was adopted.

The training comprised introductory (two hours), fast-track (eight hours), and extended (four days) training sessions. Training modules include competencies such as a profound understanding of HTA processes and key principles, domains, impact of HTA, outcome measurements, and study designs to raise awareness, promote, and enhance patient engagement as foreseen in the HTAR. The training modules focus on empowering patients to advocate for their rights and communities and to contribute meaningfully to comprehensive treatment evaluation. Specifically, modules on communication and personal skills, patient involvement, and engagement at both national and European levels underscore promotion of patient empowerment. Training sessions can be accessed online (EUCAPA.eu).

The training development within the EUCAPA project addresses the evolving landscape of joint HTA by emphasizing the significance of patient perspectives, fostering core competencies, and responding to the increasing demand for active patient involvement and advocacy at both national and supranational levels.

Over the past 10 years, the Scottish Medicines Consortium (SMC) has strived to strengthen the involvement of patients and citizens in its work and improve public understanding of health technology appraisal (HTA). A series of innovations have been brought in since 2014 to increase levels of engagement and satisfaction of participants. This work describes these and their impact.

SMC has introduced numerous transformational changes since 2014. Innovations include a new Patient Group Partner (PGP) registration system, strengthened patient group submission process, participation of patient representatives at Patient and Clinician Engagement (PACE) and SMC Committee meetings, embargoed early release of SMC decisions to PGPs, provision of Summary Information for PGPs (from submitting company), and revised role of public representatives. The creation of the SMC Public Involvement Network Advisory Group has underpinned these achievements, as has the provision of comprehensive one-to-one support, information and education to PGPs. SMC has continually evaluated the satisfaction of participating PGPs using an online questionnaire.

There has been a sustained increase over the past 10 years in both the number of patient groups engaging with SMC and the number of PGP submissions that SMC receives. Adopting a continuous improvement approach, working in partnership with PGPs and public representatives, has helped SMC to ensure that stakeholders in the HTA process are effectively engaged and informed about the HTA of new medicines in Scotland. Surveys of public involvement in SMC consistently show an extremely high level of satisfaction from PGPs who work with SMC, with most PGPs consistently rating their experience of working with SMC as excellent.

Over the past 10 years, SMC has strived to strengthen how it involves citizens and patient representatives in HTA. Various innovations and a continuous improvement approach have helped to ensure that there are high levels of satisfaction and understanding of the HTA process from patient groups who engage with SMC. This is underpinned by a partnership approach to working.

In 2020, health technology assessment (HTA) became mandatory for any new medicine covered by public funds. The aim of the review is to present the impact of HTA implementation despite Russia’s full-scale war against Ukraine. Thanks to the introduced reforms, including HTA introduction, the coverage from the public payer in providing patients with medicines increased from 11 percent in 2013 to 34 percent in 2023.

This review summarizes all Department of HTA conclusions with recommendations made during 2023, decisions made by the Ministry of Health (MOH) based on these recommendations and their impact on patient access, and the steps made to further institutionalize HTA in Ukraine. In 2023, the HTA guidelines for medicines were revised based on the lessons learned and global experience along with knowledge exchange with the United Kingdom HTA agency, NICE International.

In 2023, the HTA Department prepared 19 HTA conclusions. The majority concerned treatment of oncologic diseases (32%), orphan diseases (21%), and multiple sclerosis (16%). HTA assessments resulted in recommendations for listing (84% cases). Fifty-three percent of medicines were recommended for managed entry agreement, and the MOH decided to forward 10 medicines for negotiations in 2023. Thirty-two percent of recommendations considered inclusion into the National Essential Medicines List (NEML); 16% with further access through the reimbursement program. Almost 70 percent of total need in NEML is currently covered by public funds, compared to 40 percent in 2020 (according to the Support in Market Development (SMD) analytical database).

The HTA function in Ukraine has evolved since 2020 with a visible impact on patients’ access. Introduction of new procedures, such as HTA for medical devices, may improve patient access to health technologies and provide evidence for rational and transparent decision-making processes, with gradual transformation of the Department of HTA at the State Expert Centre into an independent HTA agency by 2026.

Effective participation of individuals with disabilities in health technology assessment (HTA) processes is paramount. Aware of the reality of people with physical and organic disabilities, COGAMI (a not-for-profit umbrella organization of disability associations) conducted an internal study to gather perspectives on the participation of people with disabilities in HTA processes.

An ad hoc questionnaire of four open-ended questions was designed and distributed via email to COGAMI’s socio-health commission, representing 23 entities and 4,000 people in Galicia. A thematic analysis of the responses obtained was carried out.

Consensus underscores the fundamental role of individuals with disabilities and their representative organizations in HTA processes, though currently, only those with greater resources actively participate. The participants found that insufficient information reaching patient organizations hinders participation (e.g., lack of awareness in proposal submission), complicating their involvement. Additional challenges include accessibility and the digital divide. Proposed solutions involve enhancing communication channels and information accessibility, establishing collaborative frameworks nationally, and actively considering the disability condition to ensure a fair and equitable implementation.

This study suggests the need for concrete actions to enhance the participation of individuals with disabilities in HTA processes. Recommendations include improving communication channels, capacity building, and recognizing disability as a key element in HTA.

In 2022, the Brazilian public health system has adopted an explicit cost-effectiveness threshold of USD24,232.91 per quality-adjusted life years (QALY) for evaluating technologies intended for rare diseases treatment. Although regarded as a strategy for increasing efficiency, the National Committee for Health Technology Incorporation (Conitec) has recommended that the threshold should not be used as a knockout parameter.

A retrospective analysis of Conitec’s recommendations regarding technologies for rare diseases issued between January and October 2023 was conducted. The following data were extracted from Conitec’s reports: (i) disease (rare or ultra-rare); (ii) health technology evaluated; (iii) scientific evidence regarding efficacy, effectiveness, and safety of the technology; (iv) incremental cost-effectiveness ratio (ICER); (v) recommendation issued; and (vi) rationale for recommendation. Identified technologies will be divided into two groups according to the recommendation issued (positive or negative). The rationale for each recommendation, along with other information, will be reviewed to look for explicit threshold utilization and its impact on decision-making.

Twelve technologies for nine rare diseases evaluated in 2023 were retrieved and eleven were included in the analysis. Six drugs have received a positive recommendation for funding. The ICERs estimated varied between ‒USD5,278,362.06 for emicizumab for hemophilia A and USD116,407.47 for elexacaftor/tezacaftor/ivacaftor for cystic fibrosis. Two of them have received a positive recommendation despite their associated ICER exceeding the explicit threshold. All negative recommendations were associated with ICERs higher than the threshold, which varied between USD52,456.32 and USD847,942.97. For two, the threshold was specifically mentioned as the rationale for the negative.

After the adoption of an explicit cost-effectiveness threshold in 2023, Conitec has issued two positive funding recommendations for rare diseases technologies despite their associated ICER exceeding the threshold: agalsidase alpha for Fabry disease and elexacaftor/tezacaftor/ivacaftor for cystic fibrosis. For both recommendations, the drug’s favorable impact on the natural history of the disease was considered decisive.

The value assessment of robotic surgical platforms poses several challenges on existing health technology assessment (HTA) frameworks of robotic-assisted surgery (RAS). Following widely RAS adoption, this work aimed to assess current RAS evaluation from the Spanish HTA network perspective taking into consideration, in the absence of local guidance, the principles published by Erskine J et al. in 2023.

A review of the HTA reports comparing RAS versus open surgery (OS) and laparoscopic surgery (LPS) conducted by the Spanish HTA network and published between 2017 and 2023 was performed and compared against the consensus statement on RAS evaluation set out around seven key themes (i.e., evidence inclusion and exclusion, patient- and clinician-reported outcomes [PRO, CRO, respectively]), the learning curve, allocation of costs, appropriate time horizons, economic analysis methods, and robotic ecosystem/wider benefits].

Four HTA reports, developed by AQuAS (Agència de Qualitat i Avaluació Sanitàries de Catalunya), assessing RAS versus OS and LPS were identified and reviewed in depth. Seven groups of indications and 57 outcomes were retrieved and classified according to the principles set out in the paper. Our findings show there is a strong lack of compliance with the consensus statement: real-world evidence studies are deprioritized or considered insufficient, and none of the reports provides either an economic evaluation or an assessment on the robotics ecosystem. Only PROs comply fully or partially, except for one report.

Spanish HTA evaluation of robotic surgical platforms diverges significantly from the consensus. Discrepancies were observed across crucial domains, notably in evidence-selection criteria, economic evaluations, and holistic robotic ecosystem assessment, despite occasional compliance with PROs. Capturing specific, relevant outcomes to enable a comprehensive HTA beyond the context of therapy is key to informing decision-making and enhancing reliability of future evaluations.

Ireland is a member of the International Horizon Scanning Initiative (IHSI) and has access to the full horizon for medicines across multiple disease areas and specialized reports on high-impact technologies within disease areas (IHSI High Impact Reports [HIRs]). Health technology developers (HTDs) provide a notification to HTA agencies and payers on intent to seek reimbursement for a technology in the preceding two years.

Information on technologies submitted to the national HTA agency in 2023 was selected for comparison to information in the IHSI database. The IHSI system detects products prior to regulatory approval. The national notification system (NNS) can receive information post-licensing. We examined how many products were received in the NNS that were either in the regulatory process or licensed in the EU. We sought to compare key information in the NNS and the IHSI database to identify areas of duplication. Areas where information from NNS could complement the IHSI database were highlighted. HIRs were not included in this assessment.

In 2023, 20 percent of products submitted by HTDs via the NNS were already licensed. To compare the information provided in IHSI and the NNS, a sample of 35 technologies common to both systems was selected. The IHSI and the NNS contain multiple data fields (154 in IHSI and 72 in NNS) across domains including information on product characteristics, regulatory information, clinical trial data, and cost. The exceptional fields specific to the Irish context were compassionate access scheme availability in Ireland, Ireland-specific patient population estimates, and local pricing and reimbursement proposals.

Receipt of information from HTDs remains important, particularly country-specific information. There are efficiencies in identifying technologies via an independent horizon scanning system, not reliant on submissions by HTDs. Such a system could facilitate joint assessment, joint early advice, and joint problem-solving between countries. Joint efforts can also facilitate broader scope on how horizon scanning can be used to increase health system preparedness.

The Agency for Care Effectiveness (ACE) in Singapore has established a horizon scanning (HS) system to provide advance notice of new and emerging medical technologies (MedTechs) to inform service planning since 2020. To remain fit for purpose, HS needs to evolve over time to better serve its stakeholders. This abstract aims to share ACE’s early HS experience and future opportunities.

To gather insights into ACE’s HS successes and potential gaps, a mixed-methods approach was used. A review of key HS activities was conducted, including different HS outputs produced, stakeholder engagements conducted, and dissemination strategies employed. Feedback from key stakeholders was gathered to understand the potential utility of our outputs. Lastly, potential gaps and opportunities were identified to inform future initiatives.

As of July 2023, 20 HS reports were developed, comprising three high-level overviews and 17 in-depth briefs. Output of briefs increased five-fold from 2021 (n=2) to 2023 (n=10). Efforts to drive impact included industry submission of pipeline MedTechs to supplement identification sources; report dissemination to over 100 policymakers and healthcare professionals for early service planning; and participation in network activities and conferences to share knowledge. Initial stakeholder feedback was positive, citing the reports’ relevance for clinical practice. The review recommended diversifying HS outputs to include environmental scan and trends reports, strengthening patient involvement and formal impact assessment through stakeholder surveys.

ACE’s HS has made significant progress in achieving its intended objectives to enable service planning for new and emerging MedTechs. However, this remains an iterative process with new opportunities for growth. Future initiatives were identified, such as impact assessment, enhancing stakeholder engagement to include patients, and broadening outputs to meet stakeholder demands.

The Scottish Medicines Consortium (SMC) assesses new medicines for NHSScotland, based on company submissions. The abbreviated process previously applied to new formulations of medicines with anticipated low net budget impact. During the COVID-19 pandemic, SMC extended the abbreviated process to new medicines with alternatives within the same therapeutic class already approved in Scotland. This evaluation explored stakeholder perspectives on this process

A mixed-method approach, with focus groups and online surveys, was utilized to collect data from stakeholders (n=56) including Patient Group Partners (n=14), Public Partners (n=2), pharmacists employed in local health boards and in national procurement (n=17), pharmaceutical industry representatives (n=10), SMC senior clinical assessors (n=6), and the SMC Executive Team (n=7). All participants were asked a series of open and semi-open questions about specific aspects of the process, its advantages, disadvantages, and potential areas for improvement. A thematic analysis, based on initial color coding and grouping of responses into themes, was employed to analyze the qualitative data

Overall, the abbreviated process was commended for its time and resource efficiency, particularly benefiting the SMC assessment teams, decision-making committees, and the pharmaceutical industry. The decision-making committees experienced a decrease in workload and capacity gains, which allowed them to focus on more innovative new medicines. Overall, stakeholders welcomed rapid access to new medicines, and resource savings for pharmaceutical companies, patient groups, and the SMC assessment teams. Suggestions for improvement included strengthening patient group involvement in the process, improving transparency of the decision-making process, in particular the budget impact assessment, and providing further guidance on the definitions of “same therapeutic class.”

Regulatory agencies are licensing more new medicines, in terms of volume and complexity. HTA bodies must consider innovative ways to undertake proportionate assessments. Extension of SMC’s abbreviated process was well commended by stakeholders, primarily due to resource-savings benefits and expedited access to new medicines. Evaluation of the change identified further areas for improvement.

Early access to innovative medications is a key factor for the quality of medical care. We investigated differences in approval dates of new active substances between the U.S. and Europe and a potential connection with company headquarter location.

Data of new active substances (no generics, biosimilars, and hybrids) approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) from 1 January 2018 to 29 November 2023 were retrieved from FDA and EMA websites. We calculated the time difference between the drug approval dates between the agencies. We also retrieved the location of the headquarters of pharmaceutical companies.

Since 2018, 390 medicines were approved by FDA or EMA: 113 (29.0%) only by FDA versus 40 (10.3%) only by EMA, and 237 (60.8%) by both. Approval of 32.1 percent of medicines occurred within six months. Fifty-seven percent were approved more than six months earlier by FDA and 11.0 percent more than six months earlier by EMA. Overall, 45.6 percent of the headquarters are in Europe and 44.1 percent are in North America. For medicines approved by FDA only, 63.7 percent of headquarters are in North America and 26.5 percent are in Europe. This is reversed for medicines approved by EMA only. For substances approved by both, 50.6 percent of headquarters are in Europe and 39.2 percent are in North America.

Almost 30 percent of new medicines approved by FDA within the last five years are not yet approved in Europe whereas only 10 percent are approved by EMA only. There is a tendency for companies with headquarters in North America to seek approval from FDA first. For medicines with approval from both authorities, most companies are primarily located in Europe.

The process of health technology assessment (HTA) is a valuable tool for the pursuit of equitable and sustainable healthcare systems. Various countries have established organizations dedicated to conducting HTAs, adapting such institutions to local healthcare ecosystems. The aim of this study was to evaluate the structure, methods, and processes of organizations responsible for national-level HTAs globally.

A scoping review was conducted assessing organizations responsible for conducting HTAs for national-level decision-making in any country. Identification of eligible organizations was performed through a review of member organizations of INAHTA, EUnetHTA, RedETSA, and HTAsiaLink networks, as well as organizations evaluated in reviews with a similar scope. For each organization, the following data were searched: country, year of foundation, organizational nature, role in decision-making, funding, technologies assessed, criteria considered for decision-making (such as efficacy and safety, costs, impact on equity, among others), type of economic evaluation, and patient involvement.

We identified 69 organizations, from 56 countries, mainly European (n=39; 56%). Fifty-three (77%) are government-affiliated; most (n=51; 74%) have a consultative role. Public funding is the main funding, and 12 (17%) organizations charge fees for conducting HTA. Technologies assessed include drugs (n=61; 88%), devices (n=47; 68%), and procedures (n=33; 48%). HTA is usually initiated upon request from the manufacturer (n=45; 65%). Patient involvement is not clearly described in 32 organizations (46%); in 29 organizations (42%), the role of patients is to provide information that is considered during decision-making.

Among the evaluated organizations, it is observed that the majority are government-affiliated, have public funding, and play a consultative role. The results of this study serve as an important reference for the development and improvement of organizations responsible for conducting HTAs.

The development of clinical practical guidelines (CPGs) has been evolving towards a standard methodology based on GRADE. While reviewing CPGs, we found valid recommendations and no evidence to update. Including these initial recommendations in addition to new recommendations as part of the scope can improve understanding of the issue.

To develop a standardized approach to the writing of recommendations when updating a CPG, we consulted several methodological handbooks: Scottish Intercollegiate Guidelines Network (SIGN) (2011 and 2019 version), National Institute for Health and Care Excellence (NICE) last update (2024), New Zealand Guidelines Group (NZGG) grading system, and Oxford Centre for Evidence-Based Medicine (OCEBM) Levels of Evidence 2. We compared the wording and specific symbols used to express strength of recommendations and quality of evidence with those used by GRADE, as described in the methodology handbook for developing clinical practice guidelines in the Spanish National Health System (SNS).

A table was developed to express the equivalence of wording and symbols of quality of evidence and grading of recommendations to align the expression of SIGN, NICE, OCEBM, and NZGG methods with GRADE terminology. SIGN and NICE methodologies did not utilize specific symbols to express strength of recommendations; SIGN did not utilize the GRADE approach to assess the quality of evidence. The NZGG system applied its own classification for quality assessment and grading of recommendations. Oxford OCEBM keeps the terminology of level of evidence and grading of recommendations.

Using wording and symbols equivalence to standardize the recommendations of different methods to GRADE could assist the process of updating CPGs with the GRADE-Adolopment approach. Recommendations that are still valid and have been developed rigorously could be integrated in an updated CPG avoiding the duplication of efforts, but this generates uncertainty, especially when the quality assessment is focused on the studies designs.

In health technology assessment (HTA), “value for money” is frequently conceptualized as incremental cost-effectiveness (CE), with effectiveness measured as (quality-adjusted) life years gained (LYG). Commonly used CE thresholds have been subject to controversial debate. We reviewed and analyzed the worldwide economic literature on the value of a statistical life (VSL), which reported empirical estimates during the last 25 years.

We conducted an extended systematic literature search in the EconBiz and EconLit databases, spanning the period from January 1995 to December 2020. We transformed VSL data into the implied values of a statistical life year (VSLY) and analyzed the results by regional origin, elicitation method, and annual gross domestic product (GDP) per capita. Then, we performed a regression analysis using the ordinary least squares (OLS) model after log-transforming the VSLY estimates.

We identified 156 empirical economic studies with 169 unique VSL estimates. Overall, the median VSLY was EUR168,367 (mean, EUR256,701) or 6.3 times annual GDP/capita. The median VSLY (expressed as multiples of GDP/capita) differed by regional origin (North America: EUR288,994; 7.2; Europe: EUR168,367, 5.2; Asia: EUR45,260, 4.5) and by elicitation method (revealed preference/wage risk studies: EUR274,625, 9.1; stated preference/contingent valuation studies: EUR113,246, 4.4; stated preference/discrete choice experiments: EUR178,130, 5.2). Regression analyses confirmed that studies with data originating from North America resulted in significantly higher VSLY estimates. The difference remained statistically significant after adjusting for GDP/capita.

The empirical economic literature, while showing heterogeneity by elicitation method, suggests that the adoption of a preference-based or “demand-side” perspective leads to willingness-to-pay estimates for a LYG that exceed commonly used CE thresholds in the context of HTA.