The management of the COVID-19 pandemic is a challenge for Health Technology Assessment (HTA) methodology due to the need to formulate evidence-based recommendations in times of uncertainty in minimal time - for a large number of publications and with changing or even contradictory information. Living systematic reviews (LSRs) are systematic reviews that are continually updated, incorporating relevant new evidence as it becomes available. Since the COVID-19 pandemic fits all criteria to perform LSRs: (i) the Review question is a particular priority for decision-making, (ii) there is an high level of uncertainty about the existing evidence, and (iii) there is likely to be emerging evidence that will impact on the conclusions of the LSR, the aim of which is to analyze the role of LSRs as an innovative approach to HTA in recent years, and its impact on the management of the pandemic.

A systematic search of LSRs (published or protocols) was run on the main biomedical databases (Medline, Embase and Cochrane Library) in November 2020 and it was rerun in June 2021 without time limit. The results will be analyzed and classified by year and category (epidemiology, treatment, prognosis, symptoms, diagnosis and vaccines).

The literature research has returned a total of 187 publications. The LSR concept emerged in 2014, from which some LSRs began to be published, but an exponential increase has been observed in 2020 with 76 references of which 66 percent were focused on the SARS-CoV-2. By category, 81.8 percent were focused on treatment, 41.8 percent on epidemiology, 20.9 percent on rehabilitation, 15.1 percent on diagnosis, 10.2 percent on prognosis and 2.2 percent on symptoms until June 2021. There wasn't any LSR for vaccines and 28 percent was focused on other fields.

LSRs are particularly important during the COVID-19 pandemic, with research evidence emerging rapidly, current evidence being uncertain, and new research changing policy or decisions on health. The majority of LSRs published up to June 2021 were focused on the treatment of COVID-19.

In 2020, the Swedish regions cemented a national managed-introduction project for medical devices, to ensure equal, cost-effective, and appropriate use. Health technology assessment (HTA) is an important component of the project. Swedish national health registers have world-class coverage and completeness but may lack information enabling adequate evaluation of medical devices. This study reviews the current situation and ongoing initiatives. Additionally, the potential for medical device health economics and outcomes research (HEOR) using Swedish registers is assessed.

A review of Swedish national health registers was undertaken, focusing on available data, and contextualized for the purpose of HEOR for medical devices. Additionally, the review included an evaluation of the Swedish reimbursement authority's (The Dental and Pharmaceutical Benefits Agency, TLV) ongoing initiatives to improve the potential to follow-up the impact of the technologies they assess and develop value-based pricing schemes.

Five registers were deemed the most relevant national health registers for device research. They include high-quality longitudinal data and are linkable on a per-patient basis. For devices, main limitations include limited data on specialized outpatient care, lags in updating certain registers, lack of laboratory data, and challenges in identifying the specific device used. Reports indicate that certain limitations are being addressed, including pilot-studies investigating the opportunity for automated reporting of data from regional systems, and app-collected patient-reported health data.

Swedish registers provide comprehensive sources for HEOR studies, but limitations related to the assessment of medical device impact remain. As is common with register data reporting grouped diagnoses and interventions, specific devices are not directly identifiable in the national health registers. For some devices, this might be addressable through linkage with other data-sources. Swedish authorities are undertaking several initiatives that will likely improve the potential for HTA and follow-up of medical devices using national health register data.

The Evidence Directorate produced eighteen rapid responses during the early stages of the COVID-19 pandemic. To address this need while retaining methodological integrity a three-tiered system for rapid responses was developed.

All rapid responses answer specific research questions rather than broad health system issues. The appropriate level varies depending on the time and resource available, and the requester's need:

1. • Level 1 – Reference List (turnaround 4–8hrs, delivered by an information scientist): a quick search for best available evidence, and results presented as a reference list.

2. • Level 2 – Summary of evidence (turnaround 1–2 days, delivered by an information scientist): a quick search and brief summary of the best available evidence.

3. • Level 3 – Synthesis of evidence (turnaround 3–7 days, delivered by a Health Services Researcher or Health Economist): a quick search and then a narrative summary and synthesis of the best available evidence, with a brief appraisal of validity, reliability and generalizability.

Since the launch of the three-tiered model in September 2020 there have been five rapid responses. Two were Level 2 products and three were Level 3 products.

The Evidence Directorate of Healthcare Improvement Scotland now has an agile rapid response product which can be applied to a variety of settings and needs. This was borne out of a need for a rapid turnaround and evidence synthesis during the COVID-19 pandemic.

Sudden onset severe headache is usually caused by a primary headache disorder but may be secondary to a more serious problem, such as subarachnoid hemorrhage (SAH). Very few patients who present to hospital with headache have suffered a SAH, but early identification is important to improve patient outcomes. A systematic review was undertaken to assess the clinical effectiveness of different care pathways for the management of headache, suspicious for SAH, in the Emergency Department. Capturing the perspective of patients was an important part of the research.

The project team included a patient collaborator with experience of presenting to the Emergency Department with sudden onset severe headache. Three additional patients were recruited to our advisory group. The patient's perspective was collected at various points through the project including at team meetings, during protocol development and when interpreting the results of the systematic review and drawing conclusions.

Patients were reassured by the very high diagnostic accuracy of computed tomography (CT) for detecting SAH. Patients and clinicians emphasized the importance of shared decision making about whether to undergo additional tests to rule out SAH, after a negative CT result. When lumbar puncture was necessary, patients expressed a preference to have it on an ambulatory basis; further research on the safety and acceptability of ambulatory lumbar puncture was recommended.

Patient input at the protocol development stage helped researchers understand the patient experience and highlighted important outcomes for assessment. Patient involvement added context to the review findings and highlighted the preferences of patients regarding the management of headache.

Sudden onset severe headache is usually caused by a primary headache disorder but occasionally is secondary to a more serious problem, such as subarachnoid hemorrhage (SAH). Guidelines recommend non-contrast brain computed tomography (CT) followed by lumbar puncture (LP) to exclude SAH. However, guidelines pre-date the introduction of more sensitive modern CT scanners. A systematic review was undertaken to assess the clinical effectiveness of different care pathways for the management of headache in the Emergency Department.

Eighteen databases (including MEDLINE and Embase) were searched to February 2020. Studies were quality assessed using criteria relevant to the study design; most studies were assessed using the QUADAS-2 tool for diagnostic accuracy studies. Where sufficient information was reported, diagnostic accuracy data were extracted into 2 × 2 tables to calculate sensitivity, specificity, false-positive and false-negative rates. Where possible, hierarchical bivariate meta-analysis was used to synthesize results, otherwise studies were synthesized narratively.

Fifty-one studies were included in the review. Eight studies assessing the accuracy of the Ottawa SAH clinical decision rule were pooled; sensitivity was 99.5 percent, specificity was 23.7 percent. The high false positive rate suggests that 76.3 percent SAH-negative patients would undergo further investigation unnecessarily. Four studies assessing the accuracy of CT within six hours of headache onset were pooled; sensitivity was 98.7 percent, specificity was 100 percent. CT sensitivity beyond six hours was considerably lower (≤90%; 2 studies). Three studies assessing LP following negative CT were pooled; sensitivity was 100 percent, specificity was 95.2 percent. LP-related adverse events were reported in 5.3–9.5 percent of patients.

The evidence suggests that the Ottawa SAH Rule is not sufficiently accurate for ruling out SAH and does little to aid clinical decision making. Modern CT within six hours of headache onset (with images assessed by a neuroradiologist) is highly accurate, but sensitivity reduces considerably over time. The CT-LP pathway is highly sensitive for detecting SAH, although LP resulted in some false-positives and adverse events.

Health Technology Assessment (HTA) is an important but time-consuming process to inform decision-making. Following requests from stakeholders in Scotland to provide advice on technologies that had recently undergone HTA in other jurisdictions, SHTG recognized a gap in their ‘product menu’. Colleagues within the SHTG team devised a mechanism through which an original HTA could be adapted for Scotland, taking into account local contextual factors.

SHTG Adaptations comprise the following: i) assessment of the original HTA using the EUnetHTA HTA Adaptation Toolkit and checklist; ii) draft Adaptation using the outcome of the assessment and contextual information for Scotland; iii) consultation group of relevant Scottish clinicians is provided with the original HTA and draft SHTG Adaptation; iv) modified Delphi approach (max. three rounds of questioning) is used to ascertain the relevance of the original HTA to Scotland; v) the Adaptation is submitted to SHTG Council for endorsement.

SHTG Adaptations have a timeline of 2–3 months, three have been published since this product was launched. The process has run smoothly with excellent clinical engagement from across NHS Scotland. Key learning focusses on the role of the SHTG Council (i.e. appraisal committee) in this process and in handling of expert opinion of evidence which has already been appraised by another agency.

The SHTG Adaptation is a new product which offers a timely assessment and utilization of an HTA from another agency.

The importance of patient-centered outcome (PCO) evidence is increasingly recognized, but its inclusion in Health Technology Assessment (HTA) submissions remains inconsistent. We explored the impact of PCO evidence on HTA decision-making.

A framework was developed to assess the impact of PCO evidence (excluding EQ-5D) on HTA appraisals. An impact rating was determined by reviewing company, committee and Evidence Review Group (ERG) opinion. This was applied to publicly available appraisal documents (National Institute for Health and Care Excellence [NICE]: 8; Scottish Medicines Consortium [SMC]: 2) in a pilot study. The framework was then refined and applied to a larger dataset.

PCO evidence had ‘substantial impact’ in 3/8 NICE and 1/2 SMC appraisals, and ‘some impact’ in those remaining. PCO evidence informed the cost-effectiveness model in 2/8 NICE and 1/2 SMC submissions, and was considered superior to EQ-5D evidence in one NICE and one SMC submission. The ERG considered PCO evidence relevant to decision-making in 5/8 NICE appraisals. PCO evidence was mentioned in guidance for 7/10 appraisals (deemed relevant in 5/10). In one assessment, committee comments were notably more favorable than ERG comments. Larger dataset analysis results provided further insights to the pilot study.

The framework allows a systematic approach to evaluating the impact of PCO evidence on HTA appraisals.

BL, AP, DGB and NY are employees of Symmetron Ltd, which received funding from Pfizer UK in connection with the development of this manuscript. KH, HT, SLC and JB are employees of Pfizer UK. This study was sponsored by Pfizer UK.

Patient-Reported Outcomes (PRO) are directly reported by the patient without interpretation of the patient's response by a clinician or anyone else and pertains to the patient's health, quality of life, or functional status associated with health care or treatment. It can provide patients’ perspectives regarding treatment benefit and harm, directly measure treatment benefit and harm beyond survival, and are often the outcomes of most importance to patients. This study aims to analyze outcomes reported by Brazilian women diagnosed with breast cancer and rank the most important attributes for these patients.

Observational study composed of interviews and questionnaires applied to a convenience sample of women diagnosed with breast cancer. The instruments were developed taking into account the literature on the topic and the expertise of specialists. The questionnaire was built with close-ended questions using multiple-choice and a Likert scale, in order to rank the attributes and outcomes found in the interviews.

The total sample was composed of 65 women diagnosed with breast cancer. Twelve women were interviewed, in September 2020, to explore the main outcomes and preferences about their treatments, such as the most common side effects and the most impacted aspects of life after diagnosis and breast cancer treatment. Psychological, emotional, and sexual impacts were frequently described as aspects of life affected by the disease and its treatment. Fifty-three women, from all the five Brazilian regions, answered the survey applied in October and November 2020. Following an order of importance ranking, the following outcomes were chosen, respectively: overall survival, progression-free survival; and quality of life. The treatment effects that were considered less important, among this sample, were pain and adverse events.

Thinking about expanding the therapeutic quality of users, it is essential to take into account the experiences of patients. PRO is a trend in current research to achieve this goal, in order to influence the decisions of HTA agencies about the importance of valuing outcomes that affect patients' lives.

This study is the first to analyze and compare the distinctive market access process of new medical technologies focused on technical fee creation in South Korea and Japan. The purpose of this study is to derive implications for each country through analysis and comparison of the market access process and propose improvements of new medical technology adoption program by referring the United States’ incentive program for innovative technology.

Identification and review the published articles and health polices, and reports related to the medical procedure (medical technologies) coding and payment rule in South Korea, Japan and the United States.

In Korea, for the rapid introduction of new medical technologies, a One-Stop Service program (aka parallel review process) is operated that simultaneously conducts regulatory approval and new health technology assessment (nHTA) process. In Japan, the Sakigake designation program aims to give patients better access to innovative pharmaceuticals, medical technologies, and regenerative medicines by streamlining the approval and pricing process but it doesn't provide immediate coverage after approval. Medicare Coverage for Innovative Technology (MCIT) is one of the incentive programs for innovative technology which aims to improve patient access to new medical technologies through rapid market access process in the United States. Medical technologies designated a Breakthrough Device receive immediate Medicare Coverage for 4 years by MCIT.

It is recommended for Korea and Japan to actively implement the accelerated patient access process and grant affordable premium prices for the innovative medical technologies. MCIT can be considered as a breakthrough for innovative medical technology adaption.

Myelofibrosis (MF) is a rare (annual incidence estimated to be 1/100,000 in Europe), chronic hematologic disorder associated with morbidity and mortality as well as the risk of evolution to acute myeloid leukemia. Ruxolitinib (Jakavi®, Novartis) is the first JAK 1/2 inhibitor approved by the FDA and EMA in 2011 in treating MF. Ruxolitinib is considered a high-cost and life-time treatment. UK-based estimates of the cost of treatment are in the region of GBP43,000/year/patient (in 2013). Against the background of the challenge of treatments for rare diseases reaching cost-effectiveness thresholds, this study identified, collected, and appraised the available evidence on the cost-effectiveness of ruxolitinib in the treatment of MF.

A systematic approach was taken to conducting the literature review. Databases searched included PubMed, EMBASE, MEDLINE, and the Cochrane Library based on search terms informed by PICO: myelofibrosis, ruxolitinib, best available therapy/standard of care, and cost-effectiveness/cost-utility/pharmacoeconomics. The search was limited to studies published in the English language. A narrative synthesis was used to evaluate studies and the CHEERS checklist to explore the quality of reporting of the cost-effectiveness analysis.

The narrative synthesis included five studies conducted in the UK, Portugal, Chile, Canada, and Finland. All cost-effectiveness analyses used data from the same two large, randomized controlled, double-blind, phase III studies (COMFORT-I and -II). Ruxolitinib was compared to the best available therapy (BAT), including hydroxyurea, no medication, and prednisone/prednisolone. Perspectives and included costs varied among analyses. Markov models and discrete state cohort models were used to evaluate the cost-effectiveness and clinical benefit was measured in quality-adjusted life years (QALY) or life years (LY) gained.

These analyses estimated the base-case incremental cost-effectiveness ratios (ICER) per QALY of (converted into USD, if appropriate, at the historic average annual exchange rate) GBP44,905 in the UK (2013; USD 70,226), EUR40,000 in Portugal (2016; USD44,272), USD54,500 (2016), CAD61,444 in Canada (2012; USD61,474), and EUR42,367 in Finland (2015; USD42,027). Based upon the cost-effectiveness thresholds applied in each of these countries, ruxolitinib was found to be universally cost-effective, albeit with price adjustments as part of the wider pricing and reimbursement processes used in these countries.

Ruxolitinib was found to be cost-effective in treating MF informed by different types of models and from different perspectives; however, there was some uncertainty around available data due to limited data sources.

The Scottish Medicines Consortium (SMC) provides advice on which new medicines should be accepted for routine use by the NHS in Scotland. To help increase the accessibility of the advice, SMC produces public information summaries, which have been published on the SMC website since 2018. We conducted an evaluation to investigate if the public summaries are achieving their purpose and subsequently help inform improvements from a user perspective. The objectives were to determine how the public summaries are being used; what users like and what could be improved; and if they have achieved a greater understanding of decisions.

The first stage of the evaluation involved surveying patient groups (organizations that represent the interests of patients, families and carers) to investigate how they use the public summaries. We then conducted workshops with patient groups and Public Partners (members of the public that volunteer with Healthcare Improvement Scotland) to gather perspectives on the content, language and layout of a selection of public summaries.

The survey responses (n = 14) illustrate that the public summaries are being used in a variety of ways. The majority (n = 10) of patient groups reported using the public summaries to help explain SMC decisions to the people they support.

The workshops highlighted that participants found the public summaries clear and helpful. In general, patient groups felt the level of detail and language used in the public summaries improved their understanding of SMC decisions compared to other sources of information, such as the press release or Detailed Advice Document.

There were a number of suggested improvements, including changing the layout (so the SMC decision appears first) and providing definitions for some technical terms. Where actionable, these recommendations have been implemented.

Working in partnership with patient groups and Public Partners has enabled SMC to further strengthen public summaries, and patient engagement more broadly. Improvements have ensured that SMC's decisions are communicated clearly, helping to increase accessibility.

The use of information technology within health systems has emerged over the years and the current pandemic has further catalyzed this development. As a result, various definitions of eHealth have emerged. Our objective was to provide an overview of definitions available on the internet to complement the traditional (“white”) literature search.

We adapted methodological guidelines from the Cochrane Handbook and management sciences to mirror the search in bibliographic databases. A comprehensive Google search was performed in July 2020 to retrieve uniform resource locators (URL's) of webpages containing terms for eHealth within four words of synonyms for the word “definition”. The DataScraper extension of the Google Chrome browser was used to collect all URL's. Webpages were eligible if they contained an original or adopted English-language definition of eHealth or contained a direct link to a definition or a document containing a definition. All document types were eligible. The analysis was performed 7 months after the data collection.

Out of the 270 unique URL's, 37 (13.7%) were no longer accessible and 51 (18.9%) were links to academic publications (“white” literature). The language was not English for five webpages (1.9%) and 113 (41.9%) did not contain a definition of eHealth or other related terms. Other related terms were defined in 29 webpages (10.7%), among which “electronic health record” occurred most frequently (18/29, 62.1%). eHealth was defined in 35 (13.0%) webpages, out of which 45.7 percent (16/35) cited an existing source and 54.3 percent (19/35) provided an original definition.

The digital era raises both challenges and opportunities in conducting a grey literature search. We found that an augmented Google-based search can identify valuable references that traditional literature searches cannot detect. Term definitions (and their context) found in the grey versus bibliographic databases will be compared to assess their alignment with health economists perspectives.

Sacral neuromodulation is a well-established therapy for urinary and fecal incontinence. Currently available sacral neurostimulators require replacement every three to five years due to battery depletion. New rechargeable sacral neurostimulators with a potential 15-year battery life now have regulatory approval in Australia. However, the initial outlay for the device is higher than for the predicate devices. Our objective was to assess the economic value of rechargeable devices, compared with recharge-free implants, and to identify the patients most likely to benefit from the introduction of this novel technology in Australia.

The Medicare database was reviewed to quantify populations likely to derive benefits from rechargeable technology. Cost minimization and budget impact analyses were conducted from a payer perspective. Cost inputs were derived from Medicare and the Private Hospital Data Bureau. Two scenarios were modeled comparing the three and five-year battery life of the recharge-free devices with 15 years for a rechargeable device. Sensitivity testing was conducted based on potential uptake and dropout rates (due to death, dementia, etc.).

Rechargeable neurostimulators were found to be dominant (cost-saving) in all modeled scenarios, facilitated by a reduction in the frequency of battery replacement procedures and their associated risks for patients. Rechargeability also facilitated higher power settings for optimal symptom control, without trading off device longevity. Younger patients are expected to derive the greatest benefit from the extended battery life as data showed that 40 percent of the implantations were for patients younger than 65 years. The key uncertainty in this analysis was the limited real-world data on patient selection and preferences, which may influence uptake and dropout rates.

Rechargeable sacral neurostimulators deliver cost savings to the healthcare system due to their extended battery life. Fewer replacement surgeries are an important patient-relevant outcome, especially for younger populations. This finding is important because it demonstrates the economic value of rechargeability to payers and provides robust evidence supporting therapy access for privately insured patients in Australia.

Patients with type 1 diabetes (T1D) require the administration of insulin to maintain glycemic control. Currently, two modes of subcutaneous insulin delivery have gained wider acceptance: multiple daily injections (MDI) and continuous subcutaneous insulin infusion (CSII). Randomized controlled trials have shown that CSII is associated with a slightly lower glycated hemoglobin (HbA1c) level when compared with MDI.

The case study on diabetes by the H2020 Next Generation Health Technology Assessment project aims to link evidence from randomized controlled trials to real-world data to estimate the impact of health technology on specific subgroups of patients, as a first step in building prediction models to personalize treatment strategies. This work aims to assess whether patients with T1D can be stratified according to the use of health technology for insulin delivery and associated glycemic control from real-world data.

We used a longitudinal prospective data repository of T1D patients from 83 clinics in the United States (T1D exchange). A data-driven two-step clustering analysis was done on adult individuals (n = 8,034) with more than five years of disease duration. Clusters were based on body mass index (BMI), sex, age at diagnosis, diabetes duration, HbA1c level, and insulin delivery method. The optimal number of clusters was estimated based on silhouette width.

We identified the following four clusters of T1D patients characterized by differences in gender and insulin delivery method: men and women with insulin injections or pens and men and women with CSII. Individuals that used CSII had lower HbA1c levels, a higher BMI, and longer diabetes duration than those using Injections or pens.

This preliminary work identified subgroups of T1D patients linked to insulin delivery methods. Future research includes the study of complications associated with different clusters and additional data sources. While the data were sourced from the T1D Exchange, the analyses, content, and conclusions presented have not been reviewed or approved by the T1D Exchange.

Mental health services for adults have been developed to provide community-based interventions. There is a recognized unmet need in some of the most complex patients that may not be adequately met by existing mental health services provision. Research is warranted to consider the best model of service delivery for this group of service users. The aims of this research were to examine the profile and history of service users defined as having complex needs as well as their service use and associated costs in the Cheshire and Wirral Partnership NHS Foundation Trust (CWP).

A diverse group of stakeholders were invited to provide feedback on the content and design of the proforma for data collection from the medical records of service users. The rationale of the data collection was described to ensure relevant patient-level cost information was collected to identify and quantify the relevant resources consumed, to inform the evaluation of direct medical costs, direct non-medical costs, and indirect costs for each patient. The proforma was designed to also permit comparisons of clinical and service use outcomes for evaluation of patient health and non-health outcomes associated with alternative care pathways.

Stakeholder feedback comprised representatives from the CWP, patients, commissioners, the Local Authority, and housing. Relevant data for extraction from patient medical records were identified and a proforma was developed. The following items were identified for inclusion: baseline demographic data, service user data (family background, contact with the criminal justice system, social history), and clinical data (diagnosis, treatment, hospital visits, and other health service use).

A proforma was developed with diverse stakeholder involvement to inform data collection on the resource use and cost impact associated with alternative care pathways in the National Health Service and other sectors of the economy. Based on the proforma developed and data extracted, an exploration of patient health and non-health outcomes associated with alternative care pathways will be conducted to inform service evaluation and to promote patient centric care.

The National Institute of Health and Care Excellence (NICE) issued a supplementary advice in 2009 stating that treatments for patients with short life expectancies (<24 months) can exceed the cost-effectiveness threshold of GBP30,000 (EUR34,668) per additional quality-adjusted life-year (QALY), as long as the treatment is indicated for small patient populations and there is sufficient evidence that it extends life (≥ three months), compared with current National Health Service (NHS) treatments. This study investigated how often NICE reimburses treatments that meet end-of-life (EOL) criteria.

Health technology assessments (HTAs) conducted by NICE from 2009 to 2020 were reviewed for approved oncology drugs. Terminated appraisals were excluded. Data regarding EOL criteria in these submissions were then gathered. The HTA decisions were divided into the following categories: EOL criteria met; EOL criteria not met; and EOL criteria not applicable. A chi-square analysis was performed.

A total of 316 reviews were assessed in the final sample, of which 71 percent (n = 223) of decisions were positive. Out of the positive decisions, 43 percent (n = 96), 25 percent (n = 55), and 32 percent (n = 72) of decisions were in the EOL criteria met, EOL criteria not met, and EOL criteria not applicable groups, respectively. The chi-square analysis showed a significant correlation between HTA decisions and EOL criteria (p = 0.0008). These results were consistent when the “EOL criteria not applicable” group was excluded (p = 0.001). When the analysis was performed between the “EOL criteria met” and “EOL criteria not met”, along with “EOL criteria not applicable” groups, it showed a possible correlation (p = 0.05).

This study showed that in oncology, NICE reimburses treatments that meet EOL criteria more often than treatments that attempt, but fail, to meet the EOL criteria.

Patient access schemes (PAS) are agreements that may enable patients to access drugs or other treatments that may not be cost effective under normal circumstances. The aim of this study was to determine whether the use of PAS by the National Institute for Health and Care Excellence (NICE) and Scottish Medicines Consortium (SMC) for recommended drugs can lead to greater access to medications for rare diseases.

Reimbursement data for rare diseases between 2004 and 2021 from health technology assessment (HTA) agencies, namely the SMC (Scotland) and NICE (England), were included. The reviews with positive HTA decisions were considered, while those with negative decisions were excluded. Several observations were made from these data and reported.

Among the total positive reviews (n = 81), 43 included PAS. The inclusion of PAS in manufacturer submissions was more frequent for NICE than for the SMC (79% and 40% percent, respectively). Most of the drugs with PAS were included in the HTA guidance from both agencies. The positive NICE reviews contingent on PAS consisted of 20 drugs. For the same set of drugs, the SMC recommended 14 with PAS and one without PAS; five drugs were not assessed. Adalimumab was recommended by NICE with a PAS (base-case incremental cost-effectiveness ratio of GBP12,336 [EUR14,256]; GBP13,676 [EUR15,804]) and by the SMC without a PAS (base-case incremental cost-effectiveness ratio of GBP22,519 [EUR26,023]). Hence, without a PAS, the drug was costlier per quality-adjusted life-year for the National Health Service (NHS) Scotland.

PAS submissions for rare diseases are more frequent for NICE than for the SMC. With the PAS discounts, the overall cost of the drugs is reduced, resulting in cost effectiveness. The SMC approved some drugs for which NICE required a PAS to improve the economic argument. Hence, the use of PAS for these drugs could lead to potential cost-savings to the NHS Scotland.