The Scottish Health Technologies Group (SHTG) provides evidence support and advice to the National Health Service in Scotland on the use of new and existing health technologies, which, although not medicines, are likely to have significant implications for people's care. The purpose of this paper is to highlight the developments that have taken place in the SHTG's patient involvement processes in the years 2017 to 2019, focusing primarily on specific engagement with patient organizations and considering how the new approaches have been received by stakeholders.

Feedback from patient organizations that participated in the SHTG submission process, alongside SHTG committee members’ views on patient organizations contributions, was gathered primarily via online questionnaires. The number of times that patient organizations were invited and accepted the opportunity to peer-review SHTG advice statements prior to and after the employment of a Public Involvement Advisor (PIA) was analyzed.

Completed questionnaires (n = 4) from three case study examples showed high patient organization satisfaction with their experience of the SHTG process. The feedback from SHTG committee members that was gathered indicated that patient organization participation was generally well received. The number of peer reviews from patient organizations for SHTG advice statements in 2018–2019 doubled to 86 percent of the total advice statements (n = 22), compared with 43 percent (n = 14) in 2016–2017.

Significant progress has been made toward improving the SHTG's patient involvement processes. A dedicated PIA post within the SHTG has allowed for a more tailored support to patient organizations and has encouraged their increased participation in SHTG processes.

The Spanish Network of Agencies for Assessing National Health System Technologies and Performance (RedETS) defined a patient involvement (PI) framework for health technology assessment (HTA) activities in 2016. The aim of this study is to evaluate the process and impact of those PI initiatives that were implemented in the first year following the publication of this new framework.

A survey was sent to those HTA researchers who implemented PI in RedETS projects. Responses were reviewed by two authors. An adapted thematic analysis was performed and the results were later discussed by all authors.

Six responses from six agencies/units were analyzed. The objectives of PI initiatives were the following: inclusion of patient perspectives, preferences and values; elicitation of important health outcomes measures; and barriers, facilitators, or suggestions for implementation. Different methods were used for PI: surveys, focus groups, in depth interviews, and participation in an expert panel. Five main themes emerged: (i) challenges with the recruitment process, (ii) needs identified, (iii) impact of PI, (iv) lessons learned, and (v) suggestions for the future.

PI initiatives within the RedETS framework were tailored to each HTA project, its specific goals and the individual needs and resources of each HTA agency. The results also pointed out how PI has a relevant impact that has enriched RedETS products providing key information on experiences, values, and preferences of patients, contributions that benefit the HTA and the process of drawing up recommendations. The main challenges were related to recruitment processes and capacity building.

The frameworks used by Health Technology Assessment (HTA) agencies for value assessment of medicines aim to optimize healthcare resource allocation. However, they may not be effective at capturing the value of antimicrobial drugs.

To analyze stakeholder perceptions regarding how antimicrobials are assessed for value for reimbursement purposes and how the Australian HTA framework accommodates the unique attributes of antimicrobials in cost-effectiveness evaluation.

Eighteen individuals representing the pharmaceutical industry or policy-makers were interviewed. Interviews were transcribed verbatim, coded, and thematically analyzed.

Key emergent themes were that reimbursement decision-making should consider the antibiotic spectrum when assessing value, risk of shortages, the impact of procurement processes on low-priced comparators, and the need for methodological transparency when antimicrobials are incorporated into the economic evaluation of other treatments.

Participants agreed that the current HTA framework for antimicrobial value assessment is inadequate to properly inform funding decisions, as the contemporary definition of cost-effectiveness fails to explicitly incorporate the risk of future resistance. Policy-makers were uncertain about how to incorporate future resistance into economic evaluations without a systematic method to capture costs avoided due to good stewardship. Lacking financial reward for the benefits of narrower-spectrum antimicrobials, companies will likely focus on developing broad-spectrum agents with wider potential use. The perceived risks of shortages have influenced the funding of generic antimicrobials in Australia, with policy-makers suggesting a willingness to pay more for assured supply. Although antibiotics often underpin the effectiveness of other medicines, it is unclear how this is incorporated into economic models.

Depending on the health system context and the demands of relevant stakeholders in countries, the need, organizational structure, and prerequisites for enabling capacity building and development in health technology assessment (HTA) will vary. Core competencies are instrumental in this and include essential knowledge, skills, and attitudes (KSAs). They provide building blocks for delivering high-quality and effective practices of HTA. We aimed to systematically explore and develop an overview of the core competencies necessary for HTA.

This study was conducted during 2016–19 using different methods in a structured manner. We drew concepts of KSAs from various literature sources, surveyed universities and HTA professionals, and conducted expert workshops to arrive at a common understanding of the required competencies.

The terminology for KSAs defining competencies in HTA programs has been clarified. In addition, a list of competencies offered through different educational and training programs has been created. The surveys provided clarity on a common understanding of KSAs among HTA stakeholders. Thereafter, a set of competencies was described and classified according to the HTA domains.

Our study shows that there is diversity in HTA programs offered by educational institutions. The content of the programs varies due to differences between countries regarding the level of HTA development and the need for HTA, including the understanding of what HTA is. The preparation of a competency checklist or a “menu” of options mirroring the diversity of HTA will ensure that the specific needs of the HTA community will be covered.

The aim of this study was to determine if magnetic resonance-guided focused ultrasound (MRgFUS) is cost-effective compared with medication, for refractory pain from bone metastases in the United States.

We constructed a Markov state transition model using TreeAge Pro software (TreeAge Software, Inc., Williamstown, MA, USA) to model costs, outcomes, and the cost-effectiveness of a treatment strategy using MRgFUS for palliative treatment of painful bone metastases compared with a Medication Only strategy (Figure 1). Model transition state probabilities, costs (in 2018 US$), and effectiveness data (quality-adjusted life-years [QALYs]) were derived from available literature, local expert opinion, and reimbursement patterns at two U.S. tertiary academic medical centers actively performing MRgFUS. Costs and QALYs, discounted at three percent per year, were accumulated each month over a 24-month time horizon. One-way and probabilistic sensitivity analyses were performed.

In the base-case analysis, the MRgFUS treatment strategy costs an additional $11,863 over the 2-year time horizon to accumulate additional 0.22 QALYs, equal to a $54,160/QALY ICER, thus making MRgFUS the preferred strategy. One-way sensitivity analyses demonstrate that for the base-case analysis, the crossover point at which Medication Only would instead become the preferred strategy is $23,341 per treatment. Probabilistic sensitivity analyses demonstrate that 67 percent of model iterations supported the conclusion of the base case.

Our model demonstrates that MRgFUS is cost-effective compared with Medication Only for palliation of painful bone metastases for patients with medically refractory metastatic bone pain across a range of sensitivity analyses.

Managed Entry Agreements (MEAs) are increasingly used to address uncertainties arising in the Health Technology Assessment (HTA) process due to immature evidence of new, high-cost medicines on their real-world performance and cost-effectiveness. The literature remains inconclusive on the HTA decision-making factors that influence the utilization of MEAs. We aimed to assess if the uptake of MEAs differs between countries and if so, to understand which HTA decision-making criteria play a role in determining such differences.

All oncology medicines approved since 2009 in Australia, England, Scotland, and Sweden were studied. Four categories of variables were collected from publicly available HTA reports of the above drugs: (i) Social Value Judgments (SVJs), (ii) Clinical/Economic evidence submitted, (iii) Interpretation of this evidence, and (iv) Funding decision. Conditional/restricted decisions were coded as Listed With Conditions (LWC) other than an MEA or LWC including an MEA (LWCMEA). Cohen's κ-scores measured the inter-rater agreement of countries on their LWCMEA outcomes and Pearson's chi-squared tests explored the association between HTA variables and LWCMEA outcomes.

A total of 74 drug-indication pairs were found resulting in n = 296 observations; 8 percent (n = 23) were LWC and 55 percent (n = 163) were LWCMEA. A poor-to-moderate agreement existed between countries (−.29 < κ < .33) on LWCMEA decisions. Cross-country differences within the LWCMEA sample were partly driven by economic uncertainties and largely driven by SVJs considered across agencies.

A set of HTA-related variables driving the uptake of MEAs across countries was identified. These findings can be useful in future research aimed at informing country-specific, “best-practice” guidelines for successful MEA implementation.

Accounts of patient experiences are increasingly used in health technology assessment (HTA) processes. However, we know little about their impact on the decision-making process. This study aims to assess the level and the type of impact of patient input to highly specialised technologies (HSTs) and interventional procedures (IPs) guidance at the National Institute for Health and Care Excellence (NICE).

A questionnaire was developed to capture quantitative and qualitative data on the amount and type of impact of patient input into NICE HTAs. It was completed by committee members of the guidance-producing programs after a discussion of the considered topics. The data were analyzed by topic and overall, for each program, and compared across programs.

Patient input was assessed on ten pieces of HST guidance published between January 2015 and November 2019, and on twenty-six pieces of IP guidance scoped between February 2016 and October 2018. A total of 96 responses were collected for HST and 440 for IP. The level of impact of patient input was higher for HST than for IP. For HST, no respondents stated that it had no impact, whereas in IP, 35 percent of respondents did. The most common types of impact found for HST and IP were that it helped interpret the other evidence and that it provided new evidence.

The impact of patient input is not necessarily explicit in changing recommendations, but it provides context, reassurance, and new information to the committee for the decision-making process in HTAs.

Patient Support Programs (PSPs) have become a trend among pharmaceutical companies and a standard service offering to patients. The objective of the present study is to describe the status of PSPs in Lebanon and to assess the extent of knowledge and awareness among Lebanese patients about the PSPs.

A cross-sectional study was conducted between April and July 2017. A convenient sample of patients was randomly selected from outpatient clinics at four hospitals within the Greater Beirut Area. A questionnaire was used to address the study objective. Bivariate analysis was performed using the Chi-square test. Data were analyzed by using SPSS version 23.

Out of 385 patients who participated in the study, 45.45 percent were aged between 46 and 66 years. None of them indicated that they were enrolled in a PSP, and only 13 percent of the respondents were aware of the existence of such a program. In terms of adherence habits, 55.6 percent of the patients self-reported that they do not skip any dose of their medication and consume their medication as prescribed by their healthcare providers. The main reason for nonadherence reported by the majority of nonadherent participants 144 (84.2%) was simple forgetfulness.

There is a severe lack of awareness of PSPs in Lebanon. Given the important role that PSPs play in creating value for patients—in terms of healthcare follow-up practices, improved adherence habits, and cost savings—there should be a more substantial effort by pharmaceutical companies to expand and promote their PSPs in the Lebanese market.

To summarize current evidence on patient and public involvement (PPI) in health technology assessment (HTA) in order to synthesize the barriers and facilitators, and to propose a framework to assess its impact.

We conducted an update of a systematic review published in 2011 considering the recent scientific literature (qualitative, quantitative, and mixed-methods studies). We searched papers published between March 2009 (end of the initial search) and December 2019 in five databases using specific search strategies. We identified other publications through citation tracking and contacting authors of previous related studies. Reviewers independently selected relevant studies based on prespecified inclusion and exclusion criteria. We extracted information using a pre-established grid.

We identified a total of 7872 publications from the main search strategy. Ultimately, thirty-one distinct new studies met the inclusion criteria, whereas seventeen studies were included in the previous systematic review. PPI is realized through two main strategies: (i) patients and public members participate directly in decision-making processes (participation) and (ii) patients or public perspectives are solicited to inform decisions (consultation or indirect participation). This review synthesizes the barriers and facilitators to PPI in HTA, and a framework to assess its impact is proposed.

The number of studies on patients or public involvement in HTA has dramatically increased in recent years. Findings from this updated systematic review show that PPI is done mostly through consultation and that direct involvement is less frequent. Several barriers to PPI in HTA exist, notably the lack of information to patients and public about HTA and the lack of guidance and policies to support PPI in HTA.

The practice of public and patient engagement (PPE) in health technology assessment (HTA) has spread worldwide, yet gaps in knowledge remain. We carried out an institutional ethnography of the Canadian Agency for Drugs and Technologies in Health (CADTH) public and patient involvement in HTA.

The research took place over 15 months and included observational work in the institutional settings, text review, and interviews with individuals working for or involved with the agency.

We found that despite demonstrated commitment to PPE, organizational history, governance structure, and practices were impediments to a unified approach to PPE. Unclear role descriptions for committee members and differences in philosophy and priority given to PPE across the organization presented challenges to effective participation. The high degree of value given to evidence-based principles at times conflicted with meaningful integration of patient input. A lack of clear goals and processes, roles, and differential treatment of evidence in PPE served to minimize the importance of patient experiences and to displace their validity. An acknowledgment of conflicts between multiple epistemic traditions at work within HTA activities may strengthen organizational approaches to PPE.

HTA organizations can learn from this study by reflecting on the challenges described and the recommendations offered to address them. We suggest solidifying CADTH's commitment to PPE with clear agency-wide roles and direction, values, and outcomes, a comprehensive framework, and policy and procedures. An acknowledgment of diverse epistemic traditions, as well as leadership and expertise in PPE, will strengthen CADTH's PPE activities and sustain its leadership position in the HTA field.

The American College of Surgeons National Surgical Quality Improvement Program (ACS NSQIP®) is a validated, risk-adjusted database for improving the quality and security of surgical care. ACS NSQIP can help participating hospitals target areas that need improvement. The aim of this study was to systematically review the literature analyzing the economic impact of using NSQIP. This paper also provides an estimation of annual cost savings following the implementation of NSQIP and quality improvement (QI) activities in two hospitals in Quebec.

In June 2018, we searched in seven databases, including PubMed, Embase, and NHSEED for economic evaluations based on NSQIP data. Contextual NSQIP databases from two hospitals were collected and analyzed. A cost analysis was conducted from the hospital care perspective, comparing complication costs before and after 1 year of the implementation of NSQIP and QI activities. The number and the cost of complications are measured. Costs are presented in 2018 Canadian dollars.

Out of 1,612 studies, 11 were selected. The level of overall evidence was judged to be of moderate to high quality. In general, data showed that, following the implementation of NSQIP and QI activities, a significant decrease in complications and associated costs was observed, which improved with time. In the cost analysis of contextual data, the reduction in complication costs outweighed the cost of implementing NSQIP. However, this cost analysis did not take into account the costs of QI activities.

NSQIP improves complication rates and associated costs when QI activities are implemented.

This study aims to assess stakeholder perceptions on the challenges and value of real-world evidence (RWE) post approval, the differences in regulatory and health technology assessment (HTA) real-world data (RWD) collection requirements under the German regulation for more safety in drug supply (GSAV), and future alignment opportunities to create a complementary framework for postapproval RWE requirements.

Eleven semistructured interviews were conducted purposively with pharmaceutical industry experts, regulatory authorities, health technology assessment bodies (HTAbs), and academia. The interview questions focused on the role of RWE post approval, the added value and challenges of RWE, the most important requirements for RWD collection, experience with registries as a source of RWD, perceptions on the GSAV law, RWE requirements in other countries, and the differences between regulatory and HTA requirements and alignment opportunities. The interviews were recorded, transcribed, and translated for coding in Nvivo to summarize the findings.

All experts agree that RWE could close evidence gaps by showing the actual value of medicines in patients under real-world conditions. However, experts acknowledged certain challenges such as: (i) heterogeneous perspectives and differences in outcome measures for RWE generation and (ii) missing practical experience with RWD collected through mandatory registries within the German benefit assessment due to an unclear implementation of the GSAV.

This study revealed that all stakeholder groups recognize the added value of RWE but experience conflicting demands for RWD collection. Harmonizing requirements can be achieved through common postlicensing evidence generation (PLEG) plans and joint scientific advice to address uncertainties regarding evidence needs and to optimize drug development.