Very few practical frameworks exist to guide the formulation of recommendations at hospital-based health technology assessment (HTA) units. The objectives of our study were: (i) to identify decision criteria specific to the context of hospital-based health technologies and interventions, (ii) to estimate the extent to which the expert community agrees on the importance of the identified criteria, (iii) to incorporate the identified criteria into a decision-aid tool, and (iv) to illustrate the application of a prototype decision-aid tool.

Relevant decision criteria were identified using existing frameworks for HTA recommendations, our past experience, a literature search, and feedback from a survey of diverse stakeholders.

Based on the survey results, twenty-three decision criteria were incorporated into the final framework. We defined an approach that eschewed a scoring system, but instead relied on a visual means for arriving at a final recommendation, by juxtaposing the importance rating for each criterion against the results of the health technology assessment. For a technology to be approved, a majority of criteria considered important should also have received favorable findings.

We created a simple and practical decision-aid tool that incorporates all decision criteria relevant to a hospital-based HTA unit. With its ease of use and accessibility, our tool renders the subjective decision-making process more structured and transparent.

ABC-VEN analysis is an easy method of clinical and economic analysis on the costs of drug coverage and an important tool for monitoring and ensuring the rational use of medicines. However, this methodology is difficult to apply in assessing the viability of medical equipment procurement (MEP) in hospital. Using a combined model of ABC analysis and Multiple criteria decision analysis (MCDA) may be more appropriate to apply to MEP.

We created five standardized criteria, which present the main results of assessment of the viability of MEP for implementing new health technologies (HTs). These criteria address the following: 1) Novelty/innovation; 2) Comparative clinical effectiveness and safety; 3) Relevance (demand); 4) Economic effectiveness; and 5) Payback period. Based on these criteria we determine the threshold values of priority for MEP: 1) High priority; 2) Medium priority; 3) Low priority.

Using the ABC model and five standardized criteria, we analyzed all proposals from the Hospital units for implementing new HTs connected with MEP for 2018. In total, proposals contained 11 items of ME, among them three items were in group A (27%), two items were in group B (18%), and six items were in group C (55%). All items were high priority for procurement with the exception of one item from group B with medium priority. Items with low priority were not revealed which can be considered as a direct indicator of the operational effectiveness of Hospital-based HTA Unit. Excluding ME with a medium priority from the procurement plan would reduce Hospital costs by 13.5 percent.

Combined ABC and MCDA analysis in the process of assessment the viability of MEP can give the opportunity to make comparative assessment of different types of ME based on standardized criteria; determine the priority for procurement of new ME; and avoid the influence of subjective factors of the managerial decision-making process in hospital.

Cardiac surgery has seen substantial scientific progress over recent decades. Health economic evaluations have become important tools for decision makers to prioritize scarce health resources. The present study aimed to identify and critically appraise the reporting quality of health economic evaluations conducted in the field of cardiac surgery.

A literature search was performed to identify health economic evaluations in cardiac surgery. The consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement was used to assess the quality of reporting of studies.

A total 4,705 articles published between 1981 and 2016 were identified; sixty-nine studies fulfilled the inclusion criteria. There was a trend toward a greater number of publications and reporting quality over time. Six (8.7 percent) studies were conducted between 1981 and 1990, nine (13 percent) between 1991 and 2000, twenty-four (34.8 percent) between 2001 and 2010, and thirty (43.5 percent) after 2011. The mean CHEERS score of all articles was 16.7/24; for those published between 1980 and 1990 the mean (SD) score was 10.2 (±1.4), for those published between 1991 and 2000 it was 11.2 (±2.4), between 2001 and 2010 it was 15.3 (±4.8), and after 2011 it was 19.9 (±2.9). The quality of reporting was still insufficient for several studies after 2000, especially concerning items “characterizing heterogeneity,” “assumptions,” and “choice of model.”

The present study suggests that, even if the quantity and the quality of health economics evaluation in cardiac surgery has increased, there remains a need for improvement in several reporting criteria to ensure greater transparency.

Antibiotics impregnated calcium sulfate (AI-CaSO4) is an innovative practice to ensure local diffusion of antibiotics especially in the treatment of prosthesis or medical implants infections. A recent introduction of AI-CaSO4 at CHU de Québec-Université Laval (CHU de Québec) was followed by a rapid increase in use and costs. A hospital-based health technology assessment (HTA) was then requested to assess the clinical relevance of AI-CaSO4 in surgical site infection (SSI) management.

A systematic review of the effectiveness and adverse effects of AI-CaSO4 was performed in indexed databases and grey literature. The local context analysis included different methodologies: 1) interviews with pharmacists, surgeons and operating room managers, 2) data extraction from electronic patient records (EPR), 3) procurement database on CaSO4, and 4) interdisciplinary working group including orthopedic and vascular surgeons, pharmacists, infectiologists, and hospital managers.

Available evidence suggest that AI-CaSO4 could contribute in the treatment of osteomyelitis whereas no conclusion can be drawn for other medical indications in both treatment and prevention of SSI. A review of 113 surgical procedures showed that AI-CaSO4 was rapidly adopted after only one year and used for various medical indications in neuromodulation, orthopedic and vascular surgery. Osteomyelitis treatment accounted for less than 3% of cases. AI-CaSO4 was mainly used in prevention of SSI (65%) and surgical revisions (74%). Furthermore, local safety issues were raised by a lack of standardization for the preparation and under recording of antibiotics use with AI-CaSO4.

The current state of knowledge does not support the widespread use AI-CaSO4 at CHU de Québec. This study highlights the importance of adapting HTA approach to the local context to influence decision-making especially in the context of innovating practice in order to insure the relevance, safety and sustainability of care.

The aim of this study was to describe the development and activities of the Hospital-Based Health Technology Assessment (HB-HTA) Unit in the Hospital of the President's Affairs Administration, one of the first examples of the implementation of HB-HTA into the practice of Kazakhstani hospitals.

Details of the development of the Unit were obtained from the hospital's administrative records. The Unit's own records were used to describe the reports prepared and the clinical areas that were covered. Responses to recommendations in the Unit's reports were obtained from hospital administration and individual departments. Estimates of savings and payback periods were based on data from the hospital information system, and data submitted by manufacturers and distributors of medical equipment.

Fifty-one rapid- and mini-HTA reports were prepared by the Unit from 2015 to 2017. Seventeen health technologies (33 percent) were not recommended for implementation in hospital practice. Refusal to implement sixteen of these technologies saved approximately 1,053,500 USD. Of the thirty-four recommended health technologies, twenty-four were implemented to treat or diagnose 1,376 patients, and eight others were included in plans for 2018–20. Of the twenty-four implemented health technologies, twelve did not require additional investments. The payback period of investments for the other twelve implemented technologies is not more than 3 years for six, less than 5 years for four, and more than 10 years for two technologies.

Establishment of the HB-HTA Unit in the hospital created the basis for making informed managerial decisions; identifying key directions for strategic development; and improving hospital management.

Traditional decision rules have limitations when a new technology is less effective and less costly than a comparator. We propose a new probabilistic decision framework to examine non-inferiority in effectiveness and net monetary benefit (NMB) simultaneously. We illustrate this framework using the example of repetitive transcranial magnetic stimulation (rTMS) and electroconvulsive therapy (ECT) for treatment-resistant depression.

We modeled the quality-adjusted life-years (QALYs) associated with the new intervention (rTMS), an active control (ECT), and a placebo control, and we estimated the fraction of effectiveness preserved by the new intervention through probabilistic sensitivity analysis (PSA). We then assessed the probability of cost-effectiveness using a traditional cost-effectiveness acceptability curve (CEAC) and our new decision-making framework. In our new framework, we considered the new intervention cost-effective in each simulation of the PSA if it preserved at least 75 percent of the effectiveness of the active control (thus demonstrating non-inferiority) and had a positive NMB at a given willingness-to-pay threshold (WTP).

rTMS was less effective (i.e., associated with fewer QALYs) and less costly than ECT. The traditional CEAC approach showed that the probabilities of rTMS being cost-effective were 100 percent, 39 percent, and 14 percent at WTPs of $0, $50,000, and $100,000 per QALY gained, respectively. In the new decision framework, the probabilities of rTMS being cost-effective were reduced to 23 percent, 21 percent, and 13 percent at WTPs of $0, $50,000, and $100,000 per QALY, respectively.

This new framework provides a different perspective for decision making with considerations of both non-inferiority and WTP thresholds.

It is important to capture all health effects of interventions in cost-utility analyses conducted under a societal or healthcare perspective. However, this is rarely done. Household spillovers (health effects on patients’ other household members) may be particularly likely in the context of technologies and interventions to change behaviors that are interdependent in the household. Our objective was to prospectively collect outcome data from household members and illustrate how these can be included in a cost-utility analysis of a behavior change intervention in chronic obstructive pulmonary disease (COPD).

Data were collected from patients’ household members (n = 153) alongside a randomized controlled trial of a COPD self-management intervention. The impact of the intervention on household members’ EQ-5D-5L scores (primary outcome), was evaluated. Analyses were then carried out to estimate household members’ quality-adjusted life-years (QALYs) and assess the impact of including these QALYs on cost-effectiveness.

The intervention had a negligible spillover on household members’ EQ-5D-5L scores (−0.007; p = .75). There were also no statistically significant spillovers at the 5 percent level in household member secondary outcomes. In the base-case model, the inclusion of household member QALYs in the incremental cost-effectiveness ratio (ICER) denominator marginally increased the ICER from GBP 10,271 (EUR 13,146) to GBP 10,991 (EUR 14,068) per QALY gained.

This study demonstrates it is feasible to prospectively collect and include household members’ outcome data in cost utility analysis, although the study highlighted several methodological issues. In this case, the intervention did not impact significantly on household members’ health or health behaviors, but inclusion of household spillovers may make a difference in other contexts.

To analyze the current development of HTA in China and to identify areas for improvement, we mapped the level of HTA development in China and compared it with the level of HTA development in ten other countries using a survey instrument.

We launched a nationwide survey targeting different stakeholders. For this purpose, we used a validated instrument that enables mapping HTA development in a country using eight domains. The views of the respondents regarding the overall level of HTA development and for each domain were compared with the results of a mapping study that included ten countries.

In total, we received 222 responses, 33 from policy-makers, 158 from researchers, and 31 from industry, as well as health provider representatives including 8 from hospitals, centers for disease control and prevention. We calculated the mean score for the level of HTA development. The overall HTA development for China was scored at 76.4 (out of a maximum of 146). Although the total score for China was comparable to the mean score of 75.6 among the ten countries, China scored significantly lower than the mean score of 117.0 among the three developed countries. In addition, China scored significantly lower in the domain of institutionalization compared to the other ten countries.

China needs to tackle the issue of low HTA institutionalization to strengthen the foundation of HTA development. Future government initiatives that institutionalize HTA, for example, establishing a national HTA system or consortium, will improve the development of HTA in China.

Patients with chronic obstructive pulmonary disease (COPD) who experience acute exacerbations usually require treatment with oral steroids or antibiotics, depending on the etiology of the exacerbation. Current management is based on clinician's assessment and judgement, which lacks diagnostic accuracy and results in overtreatment. A test to guide these decisions in primary care is in development. We developed an early decision model to evaluate the cost-effectiveness of this treatment stratification test in the primary care setting in the United Kingdom.

A combined decision tree and Markov model was developed of COPD progression and the exacerbation care pathway. Sensitivity analysis was carried out to guide technology development and inform evidence generation requirements.

The base case test strategy cost GBP 423 (USD 542) less and resulted in a health gain of 0.15 quality-adjusted life-years per patient compared with not testing. Testing reduced antibiotic prescriptions by 30 percent, potentially lowering the risk of antimicrobial resistance developing. In sensitivity analysis, the result depended on the clinical effects of treating patients according to the test result, as opposed to treating according to clinical judgement alone, for which there is limited evidence. The results were less sensitive to the accuracy of the test.

Testing may be cost-saving in primary care, but this requires robust evidence on whether test-guided treatment is effective. High quality evidence on the clinical utility of testing is required for early modeling of diagnostic tests generally.

In 2007, Taiwan began conducting health technology assessments (HTA) to support the National Health Insurance Administration (NHIA) in its reimbursement decisions for drugs, medical devices, and medical services.

In this study, the development, missions, and procedures of the implementation of HTA in Taiwan are briefly introduced. Moreover, the value of HTA is examined by reviewing the outcomes and impacts of recent HTA-related research projects, which are classified into five categories: (i) pharmaceutical products, (ii) medical procedures, (iii) medical devices, (iv) health policy, and (v) social care.

Overall, the 10-year implementation of HTA has not only supported the government's decision making but also enhanced patient care. Furthermore, patient evidence has been highlighted, and patient care pathways have been transformed through patient involvement in HTA.

In conclusion, HTA's value has been determined by both government and social aspects in Taiwan.

Heated humidified high-flow nasal cannula (HHHFNC) is gaining popularity as a mode of respiratory support. We updated a systematic review and meta-analyses examining the efficacy and safety of HHHFNC compared with standard treatments for preterm infants. The primary outcome was the need for reintubation for preterm infants following mechanical ventilation (post-extubation analysis) or need for intubation for preterm infants not previously intubated (analysis of primary respiratory support)

We searched PubMed, MEDLINE, Embase, and the Cochrane Library for randomized controlled trials (RCTs) of HHHFNC versus standard treatments. Meta-analysis was conducted using Review Manager 5.3.

The post-extubation analysis included ten RCTs (n = 1,201), and the analysis of primary respiratory support included ten RCTs (n = 1,676). There were no statistically significant differences for outcomes measuring efficacy, including the primary outcome. There were statistically significant differences favoring HHHFNC versus nasal cannula positive airway pressure (NCPAP) for air leak (post-extubation, risk ratio [RR] 0.29, 95 percent confidence interval [CI] 0.11 to 0.76, I2 = 0) and nasal trauma (post-extubation: 0.35, 95 percent CI 0.27 to 0.46, I2 = 5 percent; primary respiratory support: RR 0.52, 95 percent CI 0.37 to 0.74; I2 = 27 percent). Studies, particularly those of primary respiratory support, included very few preterm infants with gestational age (GA) <28 weeks.

HHHFNC may offer an efficacious and safe alternative to NCPAP for some infants but evidence is lacking for preterm infants with GA ≤28 weeks.

This study was conducted to evaluate the cost-effectiveness of sunitinib versus interferon-alfa for the treatment of advanced and/or metastatic renal cell carcinoma (RCC) in Singapore.

A partitioned survival model with three health states (progression-free, progressive disease, and death) was developed from a healthcare payer perspective over a 10-year time horizon. Survival curves from the pivotal trial of sunitinib versus interferon-alfa were extrapolated beyond the trial period to estimate the underlying progression-free survival and overall survival parametric distributions. Health state utilities were derived from the literature and direct costs were sourced from local public healthcare institutions. The sunitinib dose in the model reflected local prescribing practices whereby a combination of 50 mg (28 percent) and 37.5 mg (72 percent) strengths are used.

The base-case analysis comparing sunitinib versus interferon-alfa resulted in an incremental cost effectiveness ratio (ICER) of SGD191,061 (USD139,757) per quality-adjusted life-year gained. Sensitivity analysis demonstrated that the ICER was most sensitive to variations in the utility value assumed for the progression-free health state and the price of sunitinib.

In the absence of any price reduction, sunitinib had an exceedingly high ICER and was not considered a cost-effective use of healthcare resources in Singapore's context for the first-line treatment of advanced RCC. The findings from our evaluation will be useful to inform local healthcare decision making and resource allocations for tyrosine kinase inhibitors when appraised alongside comparative clinical effectiveness data and payer affordability considerations.

Procurement's important role in healthcare decision making has encouraged criticism and calls for greater collaboration with health technology assessment (HTA), and necessitates detailed analysis of how procurement approaches the decision task.

We reviewed tender documents that solicit medical technologies for patient care in Canada, focusing on request for proposal (RFP) tenders that assess quality and cost, supplemented by a census of all tender types. We extracted data to assess (i) use of group purchasing organizations (GPOs) as buyers, (ii) evaluation criteria and rubrics, and (iii) contract terms, as indicators of supplier type and market conditions.

GPOs were dominant buyers for RFPs (54/97) and all tender types (120/226), and RFPs were the most common tender (92/226), with few price-only tenders (11/226). Evaluation criteria for quality were technical, including clinical or material specifications, as well as vendor experience and qualifications; “total cost” was frequently referenced (83/97), but inconsistently used. The most common (47/97) evaluative rubric was summed scores, or summed scores after excluding those below a mandatory minimum (22/97), with majority weight (64.1 percent, 62.9 percent) assigned to quality criteria. Where specified, expected contract lengths with successful suppliers were high (mean, 3.93 years; average renewal, 2.14 years), and most buyers (37/42) expected to award to a single supplier.

Procurement's evaluative approach is distinctive. While aiming to go beyond price in the acquisition of most medical technologies, it adopts a narrow approach to assessing quality and costs, but also attends to factors little considered by HTA, suggesting opportunities for mutual lesson learning.

Ultra-orphan therapies (prevalence: <1:50,000) can have trouble meeting Health Technology Assessment (HTA) clinical- and cost-effectiveness criteria, set by HTA bodies to inform reimbursement decision-making, due to low patient numbers limiting the supporting clinical evidence generated and high per-patient prices. Since 2013, National Institute of Health and Care Excellence (NICE) appraise Highly Specialised Technologies (HST) (“for use in the provision of services for rare and very rare conditions”) using a distinct appraisal framework. This research compares NICE HST appraisal outcomes with corresponding guidance by other HTA bodies.

All NICE HST technology guidance was screened (1 January 2013–6 November 2018) alongside corresponding guidance by Gemeinsamer Bundesausschuss (G-BA), Haute Autorité de Santé (HAS), Scottish Medicines Consortium (SMC), and National Centre for Pharmacoeconomics (NCPE).

NICE have published eight HST guidances all with positive recommendations after a median of 21 months (range: 7–38) after European Marketing Authorization (MA). An additional eight HST have guidance in-development despite having European MA for a median of 12 months (range: 2–46) with 5/8 having draft guidance issued, all being “not recommended”. Of the 18 HSTs with NICE guidance published/in-development, 29 percent (2/7), and 33 percent (2/6) have been assessed with positive outcomes (definition: “recommended”/”accepted”/“conditional”/”restricted”) by SMC, and NCPE, respectively vs. 100 percent (9/9) by G-BA (definition: any additional benefit), and 50 percent (5/10) by HAS (definition: ASMR I-III). Median delays between European MA and positive appraisal outcomes were seven (G-BA), nine (HAS), 12 (NCPE), and 19.5 months (SMC).

Although all NICE HST final guidances to date have been positive, few technologies have completed this process after substantial delays from MA. Other cost/QALY HTA bodies (i.e. excluding the G-BA and HAS clinical-assessment HTAs) have shown low appraisal and recommendation rates for these technologies; therefore, ultra-orphan technologies may require a distinct appraisal process/framework but the HST may not (yet) represent best-practice.

Indirect comparisons via a common comparator (anchored comparisons) are commonly used in health technology assessment. However, common comparators may not be available, or the comparison may be biased due to differences in effect modifiers between the included studies. Recently proposed population adjustment methods aim to adjust for differences between study populations in the situation where individual patient data are available from at least one study, but not all studies. They can also be used when there is no common comparator or for single-arm studies (unanchored comparisons). We aim to characterise the use of population adjustment methods in technology appraisals (TAs) submitted to the United Kingdom National Institute for Health and Care Excellence (NICE).

We reviewed NICE TAs published between 01/01/2010 and 20/04/2018.

Population adjustment methods were used in 7 percent (18/268) of TAs. Most applications used unanchored comparisons (89 percent, 16/18), and were in oncology (83 percent, 15/18). Methods used included matching-adjusted indirect comparisons (89 percent, 16/18) and simulated treatment comparisons (17 percent, 3/18). Covariates were included based on: availability, expert opinion, effective sample size, statistical significance, or cross-validation. Larger treatment networks were commonplace (56 percent, 10/18), but current methods cannot account for this. Appraisal committees received results of population-adjusted analyses with caution and typically looked for greater cost effectiveness to minimise decision risk.

Population adjustment methods are becoming increasingly common in NICE TAs, although their impact on decisions has been limited to date. Further research is needed to improve upon current methods, and to investigate their properties in simulation studies.

To assess the societal cost-effectiveness of the Transmural Trauma Care Model (TTCM), a multidisciplinary transmural rehabilitation model for trauma patients, compared with regular care.

The economic evaluation was performed alongside a before-and-after study, with a convenience control group measured only afterward, and a 9-month follow-up. Control group patients received regular care and were measured before implementation of the TTCM. Intervention group patients received the TTCM and were measured after its implementation. The primary outcome was generic health-related quality of life (HR-QOL). Secondary outcomes included disease-specific HR-QOL, pain, functional status, and perceived recovery.

Eighty-three trauma patients were included in the intervention group and fifty-seven in the control group. Total societal costs were lower in the intervention group than in the control group, but not statistically significantly so (EUR-267; 95 percent confidence interval [CI], EUR-4,175–3011). At 9 months, there was no statistically significant between-group differences in generic HR-QOL (0.05;95 percent CI, −0.02–0.12) and perceived recovery (0.09;95 percent CI, −0.09–0.28). However, mean between-group differences were statistically significantly in favor of the intervention group for disease-specific HR-QOL (−8.2;95 percent CI, −15.0–−1.4), pain (−0.84;95CI, −1.42–−0.26), and functional status (−20.1;95 percent CI, −29.6–−10.7). Cost-effectiveness acceptability curves indicated that if decision makers are not willing to pay anything per unit of effect gained, the TTCM has a 0.54–0.58 probability of being cost-effective compared with regular care. For all outcomes, this probability increased with increasing values of willingness-to-pay.

The TTCM may be cost-effective compared with regular care, depending on the decision-makers willingness to pay and the probability of cost-effectiveness that they perceive as acceptable.

The National Institute for Health and Care Excellence (NICE) in England has a separate appraisal process for drugs for very rare conditions, i.e. Highly Specialised Therapies (HST). In April 2017, the HST process has been changed to incorporate a quantitative approach: automatically fund treatments with incremental cost-effectiveness ratio (ICERs) up to GBP 100,000 (EUR 113,008 based on the 2018 average GBP / EUR exchange rate) per quality-adjusted life year (QALY). For treatments with an ICER above GBP 100,000 per QALY, NICE will consider treatments that offer a substantial magnitude of improvement, with additional QALY weighting. We investigated the impact of this more quantitative approach on the likelihood of a HST receiving a positive recommendation.

All HST appraisals and draft guidance documents were reviewed (up to November 2018) and data were extracted on ICERs, incremental QALY gain, budget impact, and recommendations. The extracted data from each HST were assessed based on the interim HST guidance.

Eighteen products have been or are currently going through the NICE HST process. Of these, 8/18 (44%) have received a positive recommendation, while 5/18 (28%) have received a draft negative guidance, and for 5/18 (28%) products, no recommendations have been published. For the products with a positive outcome, 5/8 (63%) had incremental QALY gain of at least 10, qualifying these products for additional QALY weighting. For the products that received a draft negative recommendation, the negative decision was related to the cost-effectiveness estimates being higher than GBP 100,000 per QALY (5/5 reported) in all cases, while none of these products were eligible to receive a ‘QALY modifier’.

It has become more difficult for HSTs to get recommended by NICE under the new guidance, which requires cost-effectiveness analyses, whereas previously there was no official ICER threshold. The additional weighting of QALYs may be insufficient to meet an ICER threshold of GBP 100,000 per QALY for many products.

The aim of this study was to provide a comprehensive overview of the evolution of health technology assessment (HTA) in Malaysia over the past decade.

We described the evolution of HTA program in Malaysia based on review of administrative data, publicly available information and quantitative description of impact evaluation.

Health Technology Assessment HTA was formalized in Malaysia in 1995 as a central structure within the Ministry of Health, Malaysia in 1995. Expansion of activities demonstrated over the years including Horizon Scanning of health technologies and implementation of evidence-based Clinical Practice Guidelines. Improvement on the processes in terms of types of report, quality, monitoring, and impact evaluation as well as accessibility was also carried out. Examples of impact/influence of the reports have also been demonstrated.

HTA program in Malaysia has evolved over the past decades. Its role in policy formulation and decision making of health technologies has become more significant over the years and is foreseen to be bigger in the future. As a trusted source of evidence, HTA in Malaysia will continue to strengthen the health system by advocating informed decision making and value-based medicine. As other countries in this region is trying to establish their own HTA processes and procedures, this review on the evolution of the HTA program in Malaysia might give some insights on developing a sustainable HTA program.

Patient involvement in drug evaluation decision making is increasing. The aim of the current study was to develop a multi-criteria decision analysis (MCDA) framework that would enable the inclusion of the patient perspective in the selection of appropriate criteria for MCDAs being used in the value assessments of oncologic drugs.

A literature review was conducted to identify and define criteria used in drug assessments from patient perspectives. The Evidence and Value: Impact on Decision Making methodology was used to develop a MCDA framework. Identified criteria were discussed by a sample of oncology patient association representatives who decided which criteria were important from patient perspectives. Selected criteria were rated by importance. The preliminary MCDA framework was tested through the assessment of a hypothetical oncology treatment. A discussion was carried out to agree on a final pilot MCDA framework.

Twenty-two criteria were extracted from the literature review. After criteria discussion, sixteen criteria remained. The most important criteria were comparative patient reported outcomes (PRO), comparative efficacy and disease severity. After the discussion generated by the scoring of the hypothetical oncology treatment, the final pilot MCDA framework included seven quantitative criteria (“disease severity”, “unmet needs”, “comparative efficacy / effectiveness”, “comparative safety / tolerability”, “comparative PROs”, “contribution of oncological innovation”) and one contextual criterion (“population priorities and access”).

The present study developed a pilot reflective MCDA framework that could increase patient's capability to participate in the decision-making process by providing systematic drug assessments from the patient perspective.