To assess the health technology assessment (HTA) process in Greece from its establishment in 2018 until 2023 in terms of timeliness and productivity.

Data were collected from the HTA Committee’s database and other publicly available sources. The overall study timeframe was divided into three periods: (i) July 2018–January 2020, (ii) January 2020–July 2021, and (iii) July 2021–February 2023.

During the study period, a total of 1,157 applications for medicinal products (MPs) (including 219 new active substances (NAS) and orphans) were submitted to the HTA Committee. The number of HTA recommendations increased from 60 (first period) to 641 (third period), while the backlog of MPs pending HTA and price negotiations decreased from 89 and 106 (January 2020) to 8 and 44 (February 2023), respectively. The median time intervals for all application types decreased significantly over time. In February 2023, the median time for clinical data assessment of NAS (excluding orphans) almost halved from 207 days in the first period to 114 days; median times for NAS and orphans from regulatory approval to HTA application were 420 and 457 days, and from HTA application to reimbursement 228 and 417 days, respectively.

The performance of the HTA process in Greece improved significantly over time, with increased MP appraisals, backlog reduction, and decreased timelines. Delays in reimbursement of NAS were mainly caused by the long gap between regulatory approval and HTA application. Overall, HTA review times in Greece are now on par with that of well-established European HTA systems.

Discounting the cost and effect for health intervention is a controversial topic over the last two decades. In particular, the cost-effectiveness of gene therapies is especially sensitive to the discount rate because of the substantial delay between the upfront cost incurred and long-lasing clinical benefits received. This study aims to investigate the influence of employing alternative discount rates on the incremental cost-effectiveness ratio (ICER) of gene therapies.

A systematic review was conducted to include health economic evaluations of gene therapies that were published until April 2023.

Sensitivity or scenario analysis indicated that discount rate represented one of the most influential factors for the ICERs of gene therapies. Discount rate for cost and benefit was positively correlated with the cost-effectiveness of gene therapies, that is, a lower discount rate significantly improves the ICERs. The alternative discount rate employed in some cases could be powerful to alter the conclusion on whether gene therapies are cost-effective and acceptable for reimbursement.

Although discount rate will have substantial influence on the ICERs of gene therapies, there lacks solid evidence to justify a different discounting rule for gene therapies. However, it is proposed that the discount rate in the reference case should be updated to reflect the real-time preference, which in turn will affect the ICERs and reimbursement of gene therapies more profoundly than conventional therapies.

Germany’s 2019 Digital Healthcare Act (Digitale-Versorgung-Gesetz, or DVG) created a number of opportunities for the digital transformation of the healthcare delivery system. Key among these was the creation of a reimbursement pathway for patient-centered digital health applications (digitale Gesundheitsanwendungen, or DiGA). Worldwide, this is the first structured pathway for “prescribable” health applications at scale. As of October 10, 2023, 49 DiGA were listed in the official directory maintained by Germany’s Federal Institute for Drugs and Medical Devices (BfArM); these are prescribable by physicians and psychotherapists and reimbursed by the German statutory health insurance system for all its 73 million beneficiaries. Looking ahead, a major challenge facing DiGA manufacturers will be the generation of the evidence required for ongoing price negotiations and reimbursement. Current health technology assessment (HTA) methods will need to be adapted for DiGA.

We describe the core issues that distinguish HTA in this setting: (i) explicit allowance for more flexible research designs, (ii) the nature of initial evidence generation, which can be delivered (in its final form) up to one year after becoming reimbursable, and (iii) the dynamic nature of both product development and product evaluation. We present the digital health applications in the German DiGA scheme as a case study and highlight the role of RWE in the successful evaluation of DiGA on an ongoing basis.

When a DiGA is likely to be updated and assessed regularly, full-scale RCTs are infeasible; we therefore make the case for using real-world data and real-world evidence (RWE) for dynamic HTAs.

Continous evaluation using RWD is a regulatory innovation that can help improve the quality of DiGAs on the market.

Early access schemes (EASs) are approaches used by payers to balance and facilitate earlier patient access to innovative health technologies while evidence generation is ongoing. Schemes require investment from payers and are associated with significant risk since not all technologies will be routinely reimbursed. The purpose of this study was to gain the perspectives of policy experts about the key challenges for EASs and potential solutions for their optimal design and implementation.

Two virtual workshops were convened including (i) UK-based policy experts (England, Wales, and Scotland) and (ii) representatives from multiple healthcare systems (England, France, Sweden, Canada, Poland, and Norway). Participants were encouraged to share their experiences with EASs in their healthcare system and highlight key challenges for policy makers. Discussions were transcribed and analyzed using framework analysis.

Participants agreed that EASs have value when targeted toward innovative technologies with the potential for significant clinical benefit in an area of high unmet need. Participants discussed potential solutions to the challenges faced by payers implementing EASs, including defining eligibility criteria, supporting evidence generation, and approaches to reimbursement.

Participants agreed that EASs are one possible solution for their healthcare systems and have the potential to deliver significant clinical value to patients. However, widespread adoption of EASs is limited due to concerns about the risks for patients and healthcare budgets, further solutions are needed to deliver EASs for targeted therapies.

This study aimed to assess whether there have been changes in the quality of clinical evidence submitted for government subsidy decisions on cancer medicines over the past 15 years.

We reviewed public summary documents (PSDs) reporting on subsidy decisions made by the Pharmaceutical Benefits Advisory Committee (PBAC) from July 2005 to July 2020. Information was extracted on the study design, directness of comparison, sample size, and risk of bias (RoB). Changes in the quality of evidence were assessed using regression analysis.

Overall, 214 PSDs were included in the analysis. Thirty-seven percent lacked direct comparative evidence. Thirteen percent presented observational or single-arm studies as the basis for decisions. Among PSDs presenting indirect comparisons, 78 percent reported transitivity issues. Nearly half (41 percent) of PSDs reporting on medicines supported by head-to-head studies noted there was a moderate/high/unclear RoB. PSDs reporting concerns with RoB increased by a third over the past 7 years, even after adjusting for disease rarity and trial data maturity (OR 1.30, 95% CI: 0.99, 1.70). No time trends were observed regarding the directness of clinical evidence, study design, transitivity issues, or sample size during any of the analyzed periods.

Our findings indicate that the clinical evidence supplied to inform funding decisions for cancer medicines is often of poor quality and has been deteriorating over time. This is concerning as it introduces greater uncertainty in decision making. This is particularly important as the evidence supplied to the PBAC is often the same as that supplied to other global decision-making bodies.

Out of 185 orphan medicinal products (OMPs) registered in 2015–2021, a mere 110 (59 percent) were available to Czech patients, and only 54 (29 percent) were officially reimbursed. Moreover, this proportion has steadily decreased over time. After years of public debate induced by this unsatisfactory OMP patient access, the national viewpoint shifted toward creating a special pathway for the reimbursement of OMP. Thus, a rigorous pricing and reimbursement procedure with strict timelines and elaborated methodology has been recently adopted in Czechia.

The innovative legislation follows the recommendations for value assessment and funding processes for rare diseases and incorporates additional elements of value, such as the societal perspective. First, the application with clinical evidence, cost-effectiveness, and budget impact analyses is submitted to the governmental health technology assessment (HTA) agency by the Marketing Authorization Holder or a Health Insurance Fund. Moreover, professional associations and patients’ organizations are rightful participants in the proceeding, providing evidence and comments. Then, the HTA agency performs the assessment/appraisal of the evidence. It subsequently publishes the assessment report summarizing available information. The report is then forwarded to the Ministry of Health and its advisory body consisting of patients, clinical experts, health insurance funds, and the State. They critically evaluate the documents and issue a binding opinion following prespecified decision-making criteria. Based on this binding opinion, the decision is issued by the HTA agency. Thus, the role of the advisory body in this process is crucial.

We believe that this novel approach may offer satisfactory patient access to orphan drugs. Moreover, it serves as a real-world example of “value-based” decision making.

The aim of this study was to find ways of bridging the gap in opinions concerning health technology assessment (HTA) in reimbursement submission between manufacturers and payers to avoid access delays for patients of vital medicines such as oncology drugs. This was done by investigating differences and similarities of opinion among key stakeholders in Australia.

The survey comprised of nine sections: background demographics, general statements on HTA, clinical claim, extrapolations, quality of life, costs and health resource utilization, agreements, decision making, and capability/capacity. Responses to each question were summarized using descriptive statistics and comparisons were made using chi-square statistics.

There were ninety-seven respondents in total, thirty-seven from the public sector (academia/government) and sixty from the private sector (industry/consultancies). Private and public sector respondents had similar views on clinical claims. They were divided when it came to extrapolation of survival data and costs and health resource utilization. However, they generally agreed that rebates are useful, outcomes-based agreements are difficult to implement, managed entry schemes are required when data are limited, and willingness to pay is higher in cancer compared to other therapeutic areas. They also agreed that training mostly takes place through on the job training and that guideline updates were a least favored opportunity for continued training.

Private sector respondents favor methods that reduce the incremental cost-effectiveness ratio when compared to the public sector respondents. There still exist a number of challenges for HTA in oncology and many research opportunities as a result of this study.

Although health technology assessment (HTA) and healthcare quality improvement are distinct processes, a greater level of alignment in outcome measures used may increase the quality and efficiency of data collection. This study evaluates the agreement in outcome measures used in oncology for healthcare quality improvement and HTAs, and how these align to the International Consortium for Health Outcomes Measurement (ICHOM) standard sets.

We conducted a cross-sectional comparative analysis of ICHOM sets focusing on oncological indications and publicly available measures for healthcare quality and HTA reports published by the National Health Care Institute from the Netherlands and the National Institute for Health and Care Excellence from the United Kingdom.

All ICHOM sets and HTAs used overall survival, whereas quality improvement used different survival estimates. Different progression estimates for cancer were used in HTAs, ICHOM sets, and quality improvement. Data on health-related quality of life (HRQoL) was recommended in all ICHOM sets and all HTAs, but selectively for quality improvement. In HTAs, generic HRQoL questionnaires were preferred, whereas, in quality improvement and ICHOM sets, disease-specific questionnaires were recommended. Unfavorable outcomes were included in all HTAs and all ICHOM sets, but not always for quality improvement.

Although HTA and quality improvement use outcome measures from the same domains, a greater level of alignment seems possible. ICHOM may provide input on standardized outcome measures to support this alignment. However, residual discrepancies will remain due to the different objectives of HTA and quality improvement.

Precision medicines rely on companion diagnostics to identify patient subgroups eligible for receiving the pharmaceutical product. Until recently, the Belgian public health payer, RIZIV-INAMI, assessed precision medicines and companion diagnostics separately for reimbursement decisions. As both components are considered co-dependent technologies, their assessment should be conducted jointly from a health technology assessment (HTA) perspective. As of July 2019, a novel procedure was implemented accommodating for this joint assessment practice. The aim of this research was to formulate recommendations to improve the assessment in the novel procedure.

This study evaluated the precision medicine assessment reports of RIZIV-INAMI of the last 5 years under the former assessment procedure. The HTA framework for co-dependent technologies developed by Merlin et al. for the Australian healthcare system was used as a reference standard in this evaluation. Criteria were scored as either present or not present.

Thirteen assessment reports were evaluated. Varying scores between reports were obtained for the domain establishing the co-dependent relationship between diagnostic and pharmaceutical. Domains evaluating the clinical utility of the biomarker and the cost-effectiveness performed poorly, whereas the budget impact and the transfer of trial data to the local setting performed well.

Based on these results we recommend three amendments for the novel procedure. (i) The implementation of the linked evidence approach when direct evidence of clinical utility is not present, (ii) incorporation of a bias assessment tool, and (iii) further specify guidelines for submission and assessment to decrease the variability of reported evidence between assessment reports.