Getting technologies adopted in the UK healthcare system can be time-consuming and complex. The National Institute for Health and Care Excellence Office for Market Access (NICE OMA) has developed a novel approach to enable greater and more coordinated dialogue between life sciences companies and healthcare system stakeholders on market access issues.

When establishing NICE OMA, interactions were carried out with life sciences trade associations and key healthcare system stakeholders to explore challenges in market access landscape. Feedback highlighted that dialogue with NICE and other stakeholders is often limited and occurs in high-risk situations; indicating a need for greater and more coordinated dialogue between industry and multiple healthcare system stakeholders outside of formal processes.

The approach developed is a safe harbor engagement framework which enables NICE OMA to facilitate interaction between life sciences companies and key healthcare system stakeholders; this collaborative approach promotes shared understanding of aspects that will allow innovative technologies to reach patients faster. It brings together multiple organizations in a safe environment where ideas can be exchanged between participants, allowing organizations to think beyond their own area of interest and to work collaboratively. Companies have used the engagement framework flexibly to engage at different stages along the development to adoption journey. Feedback indicates that companies have benefitted from channeling discussions through NICE to bring together key leaders from different organizations, as well as the neutral facilitation of discussions. Healthcare system partners have gained insights/knowledge that hadn't been apparent beforehand. Patient and clinical representatives have appreciated the opportunity to provide views to a broad range of stakeholders often early in the development of the technology.

The NICE OMA safe harbor engagement framework has been well-received to date. Further feedback will be sought to understand the impact in helping to optimize the market access journey.

A recent article reported a high level of commonality across European Health Technology Assessment bodies’ (HTABs) positions in former parallel scientific advice procedure. Since 2017, the EUnetHTA joint action 3 (JA3) offers a new early dialogue process involving a higher number of European HTABs. The present analysis aims to describe if the JA3 process modified the level of agreement across HTABs.

A descriptive analysis of the written recommendations provided during every JA3 early dialogues coordinated by the French National Authority for Health (HAS) until November 2017 was conducted. The level of commonality for each HTAB position identified was assessed globally and by domain (population, comparator, outcomes, study design and health economics) and classified as follows: “full agreement” if all HTABs had the same position, “partial agreement” if more than half HTABs had the same position and “disagreement” in all other cases.

Four JA3 early dialogues were performed until November 2017: two in oncology, one in neurology and one in metabolic disorders. Between five and nine HTABs from eleven European countries participated. A total of forty-six positions were identified in these four early dialogues: ten on population, five on comparator, fifteen on outcomes, four on study design and twelve on health economics. Of the forty-six positions, full agreement was reached for twenty-eight positions, partial agreement for seventeen positions and only one disagreement was observed. The level of full agreements was highest for questions on comparators (five out of five) and population (nine out of ten) and lower for questions on health economics (six out of 12).

Although the JA3 process substantially increased the number of HTABs participating in the early dialogues, this descriptive analysis suggests that the level of agreement remains very high. This may be facilitated by the high level of dialogue and coordination between HTAB ensured by the EUnetHTA process.

The National Institute for Health and Care Excellence Medtech Innovation Briefings (MIBs) are commissioned by the National Health Service (NHS) England and designed to support NHS and social care commissioners and staff who are considering using new medical devices and other medical, digital or diagnostic technologies. MIBs are fast flexible summaries of single technologies that are intended to be responsive to commissioners needs for information about innovative technologies. MIBs include a description of the technology, how it is used and its potential role in the treatment pathway. They also include a review of relevant published evidence and likely costs. As a relatively new product, the format of MIBs continues to evolve and in 2016 a more streamlined evaluation template was introduced. To ensure MIBs continue to meet users’ needs, a study was conducted to understand the opinions and requirements of core stakeholders and to identify key areas for future development.

An initial cross-sectional online survey with NHS staff who were potential users of MIBs was carried out in December 2015. A second round of online and mail-out surveys were circulated between November 2016 and May 2017 to medical technology manufactures and an additional group of NHS staff. Descriptive analysis was used for all quantitative data and qualitative data was summarized using thematic analysis.

Thirty-nine medical professionals and fourty-two manufacturer representatives participated in the surveys. More than half of clinicians were aware of MIBs and thought that raising awareness and visibility should be a future priority. Manufactures regarded MIBs as having a positive or mixed impact on innovation, access, or uptake by the healthcare system.

Stakeholders are using MIBs in a variety of ways and there was and a range of suggestions for their future development particularly regarding moving from single technology evaluation to simultaneous assessment of similar technologies.

The Romanian healthcare system has been struggling to use a more transparent approach in evaluating health care technologies for more than 10 years. No systemic and satisfactory approach to evaluate health technologies was implemented until the present. The objective of the presentation is to describe the characteristics of the HTA system used by the Romanian healthcare authority as well as the consequences of the drug assessments by using the actual Romanian health technology assessment (HTA) evaluation framework, from the initiation in May 2014 to the end of year 2017.

The drug reimbursement context and the healthcare legislation regarding HTA evaluation were studied. A critical appraisal of the scorecard was conducted, taking into consideration general principles of the health technology assessment. A descriptive analysis covering the assessment drug reports issued by the National Agency for Drug and Medical Devices (NADMD) issued between May 2014 and December 2017 was presented, together with the decision made by the Ministry of Health and the Romanian government.

During the analyzed period of time, more than 10 updates of the reimbursement list were implemented by the Ministry of Health. By November 2017, more than 180 drugs (new INN, new indications or fix dose combinations) were included in the reimbursement system with conditional or unconditional reimbursement; more than 230 reports were assessed by the NADMD. While the new drugs reimbursed between May 2014 and November 2017, in the most part demonstrated cost savings, a lot of new innovative drugs proposed to be evaluated were rejected since the drugs had no comparators on the Romanian market and their costs were considered to have a negative impact on the healthcare budget.

The rapid HTA assessment has many strengths, by using a proper scorecard. Limitations and weakness of the actual scorecard were identified, mainly regarding the lack of a basic budget impact analysis which must include at least the direct healthcare cost, as well as the imported results of different healthcare environments that are not matching the Romanian context. Opportunities to implement a more rapid and accurate HTA evaluation are identified since the scorecard could be updated in order to address the HTA general principles.

The National Committee for Health Technology Incorporation (CONITEC) has a structured process for the incorporation, disinvestment, or alteration of different health technologies in the Brazilian public health system and provides technical support for the decision-making process. Since its creation, CONITEC has received several submissions for the incorporation of medicines and the update of clinical practice guidelines for multiple sclerosis (MS). Nowadays, more than twelve different therapies are currently available to treat MS and the Brazilian clinical practice guideline, which was last updated in 2015, offers six medicines to treat MS that are divided into first, second, and third line treatments. The purpose of this study was to describe CONITEC's assessments of applications for incorporation, disinvestment, or alteration of medicines for MS.

A case study method was used to evaluate information, retrieved from CONITEC's database, about the health technology reports developed by CONITEC's Executive Secretariat in response to applications received in the period from 2012 to 2017.

Ten technical reports on health technologies for MS were produced by CONITEC during the study period. This number represented four percent of the external submissions for incorporation of technologies for several clinical conditions in the public health system. Six medicines were evaluated. The highest number of submissions were for incorporation (n = 6), followed by alteration of treatment lines (n = 3), and disinvestment (n = 1); fifty percent of the submissions were not recommended. The main reasons for rejection were low or unproven efficacy, high budget impact, and inadequacy of the proposal based on the evidence presented. CONITEC's favorable recommendations caused a profound change in the current clinical practice guideline and had a significant impact on the health system.

MS is considered a rare disease in Brazil, but there is significant pressure from society to provide better treatment options that will impact the MS scenario in the health system. The recent CONITEC assessments have led to a revolution in the treatment of MS in Brazil, which is now in the process of being updated.

A statewide health technology assessment (HTA) program was implemented to increase equity of access and support robust assessment of technologies, with a focus on those that are high-cost, high-risk, or have state-wide impact.

Local hospital networks and clinicians refer technologies to the South Australia Policy Advisory Committee on Technology (SAPACT) for assessment. Independently produced, comprehensive HTA reports are developed using internationally recognized evidence and critical appraisal methodologies. Clinical and economic systematic analyses are utilized, with extensive clinical consultation, to develop recommendations for new technologies and their role in models of care. Feasibility of adoption and local implementation are considered, including existing service delivery and appropriate training and credentialing. For approved technologies, SAPACT may also develop audit criteria and seek implementation reports on clinical outcomes.

The HTA framework has been successfully adopted across South Australia Health, increasing the incorporation of evidence-based decision making in the use of high-cost and high-risk health technologies. Over 35 evidence evaluations for high-risk and high-cost health technologies have been conducted for a broad range of treatment interventions. SAPACT develops and utilizes HTA decision-making criteria for transparency of Committee considerations. The program recommends adoption or rejection of technologies, or it may request a re-submission due to safety concerns or a lack of proven effectiveness. SAPACT has also granted temporary approval through adoption under clinical evaluation to inform investment decisions. A key component is working with clinicians to define specific treatment criteria and patient selection. SAPACT continues to strengthen relationships with all stakeholders, increase patient input through the development of public summary documents for technologies, and improve monitoring and reporting of clinical outcomes.

The HTA program has been very productive and positively received. The success of the program is underpinned by its engagement with clinicians, hospital networks, and consumers. The completion of SAPACT HTA reviews and the publication of the SAPACT decision-making criteria have increased the credibility of decisions, supporting enhancements in patient care and cost efficiency for the state government.

We examined relationships between measures of total knee arthroplasty (TKA) “appropriateness” constructs and surgeon TKA recommendations in people with knee osteoarthritis (OA). Although TKA is highly effective, fifteen to thirty percent of recipients report dissatisfaction and/or little or no symptom improvement. More appropriate selection of surgical candidates may improve both patient outcomes and healthcare resource use, but no validated appropriateness criteria exist currently in Canada.

Patients 30 years of age or older with knee OA referred for surgical consultation at two large joint arthroplasty centres in Alberta, Canada were invited to participate. Participants completed a standardized pre-consult questionnaire, which included the following sociodemographics and validated measures of appropriateness constructs for TKA: knee symptoms; non-surgical management; patient readiness for and expectations of TKA; and net patient benefit. Post-consultation, surgeons were asked to confirm knee OA and their recommendation. We used multivariable logistic regression to examine the relationship between measures of appropriateness constructs and receipt of surgeon TKA recommendation.

Of 3,009 patients approached, 2,360 completed the questionnaire and 2,064 (sixty-nine percent) were eligible at surgical consultation (mean age 65.7 years, standard deviation 9.1; fifty-nine percent were women); 1,495 (seventy-two percent) were recommended for TKA. The likelihood of receiving a TKA recommendation was independently associated with: knee symptoms (odds ratio [OR] per unit increase in pain intensity, 1.19 (95% confidence interval [CI]: 1.11–1.27)); prior non-surgical OA management (OR for prior knee injection, 1.53 (95% CI: 1.21–1.94)); readiness for surgery (OR if definitely/probably willing to undergo TKA, 3.03 (95% CI: 1.99–4.59)); and TKA expectations (OR outcome “very important”: ability to perform daily activities, 1.40 (95% CI: 1.04–1.88); straighten the knee/leg 1.42 (95% CI: 1.13–1.80); participate in exercise/sports 0.75 (95% CI: 0.58–0.98)).

In our cohort of patients with confirmed knee OA who consulted a surgeon for TKA, appropriateness constructs were significantly associated with receipt of a TKA recommendation. Research is ongoing to evaluate the predictive validity of these measures for patient-reported outcomes associated with TKA.

More than 50 HTA agencies evaluate the clinical value of medicines in Europe, resulting in duplication of work for HTA agencies and manufacturers, and lengthy and variable time to reimbursement for patients across Europe. A consistent, single European relative clinical scientific benefit assessment of medicines could become a key element in ensuring patients get equitable and timely access across Europe. The European Network for Health Technology Assessment (EUnetHTA) is responsible under Joint Action 3 (JA3, 2016–2020) to pilot more than 30 Relative Effectiveness Assessments (REAs) of medicines. The first EUnetHTA JA3 REA pilots are now being completed and Roche, with its participation in the REA pilot for alectinib, has gathered relevant experience.

The goal of this analysis is to summarize and reflect upon the experience with one of the first EUnetHTA REA assessments in JA3. The authors also propose potential process improvements.

The experience with the alectenib REA shows that EUnetHTA processes have improved compared to JA2. The timing of the assessment has been aligned with the EU regulatory marketing authorization process by shortening the duration of the scoping phase. More EUnetHTA members than in JA2 seem to be committed to use the reports in national HTA, pricing and reimbursement processes. At the same time, the REA pilots have identified other areas that could benefit from further refinement, for example the active engagement of patient group representatives and clinical experts, rules and principles related to the handling of confidential information.

Based on the limited number of REA pilots for medicines it is too early to draw final conclusions on the state of EU-level collaboration. But first signals indicate a positive development compared to REA pilots conducted in JA2. Interim evaluations are recommended to assess progress, and capture learnings for future pilots.

Nusinersen is an orphan drug for spinal muscular atrophy (SMA) recently approved for marketing. Its high cost, striking but limited evidence of efficacy, and strong demand by media and patient organizations have generated a health policy conflict. We analyze the flaws of available evidence on nusinersen and its budget impact at a pediatric hospital, and report a collaborative strategy for drug procurement and financing.

Nusinersen is the highest-cost drug assessed by our hospital-based health technology assessment (HB-HTA) program so far. At the time of our assessment, only interim-analysis data of the pivotal randomized trial submitted to Federal Drug Administration (FDA) for approval and the European Medicines Agency (EMA) report containing unpublished final results were available. These secondary sources and other published phase II results were appraised. As a referral hospital, we concentrate most of the 300 SMA patients in our country. Hospital budget impact estimation included drug and hospitalization costs for the first and following years. The HTA report was submitted to the Ministry of Health to address this financing issue.

The available evidence of efficacy raised serious methodological and clinical uncertainties. First-year treatment cost per patient was estimated in ARS 13,008,688 (USD 752,000, 10 percent of pharmacy annual drug budget). Hospital budget impact (70 eligible patients) was ARS 910,608,160 (USD 52,000,000; 18 percent of total annual hospital budget). Our recommendation was to contact central level authorities to resolve both drug financing and patient access by negotiating a shared-risk approach for an expanded access program, allowing further data collection for reassessment after 12 months. This, in turn, fostered mutual collaboration and consensus within the health system where several lawsuits were demanding drug coverage. Negotiation with the industry was initiated by the Ministry.

This case is a clear example of forthcoming ultra-high-cost drugs unaffordable by hospital budgets. Their acquisition opportunity cost is a health policy matter requiring to display collaborative coping strategies with Ministries and other stakeholders including industry.

The European Union (EU) directive (2014/24) on public procurement strives to stimulate innovation and seeks for methodologies to implement a quality/cost based approach to search for the most economically advantageous tendering (MEAT). MedTech Europe launched the MEAT value-based-procurement (VBP) framework and tool which considers product's value from different perspectives/dimensions. Results from the first EU pilot, testing the feasibility to use the MEAT framework at a university hospital, are presented.

The framework and tool were tested using two types of technologies: high volume (underpads, diapers) and highly specialized (transcatheter aortic valve implantation). Companies were invited to participate following standard procurement rules. For each dimension, criteria, metrics and weights were defined, using multidisciplinary hospital teams. In parallel, companies were asked to do the same. Product performance scores were obtained from companies’ information. Challenges to implement MEAT in real life were identified through face-to-face meetings.

The process was well perceived by companies and hospital. Nevertheless, the level of information provided by companies was heterogeneous (quantity and quality). A match in the cost and outcome criteria was observed between hospital and companies; but relative weights assigned differed. Value propositions and robustness of information provided by companies varied across technologies and size of companies. Implementing the MEAT VBP framework and tool need extra time and knowledge.

MEAT VBP value technologies ahead of price, leading to the most economic advantageous purchasing. Nevertheless its implementation in real life is limited.

According to the Vietnamese Cardiovascular Association, one-fifth of Vietnam's population is suffering from cardiovascular disease (CVD) – now the leading cause of death in the country that accounts for about one-third of total deaths every year. Yet affordable and convenient solutions to monitor and detect CVDs remain limited and not available nation-wide. This study aimed to investigate the usability of a portable dry-electrode electrocardiography (ECG) device, paired with a mobile phone, in supporting ECG service delivery in Vietnam.

An evaluation study was designed to combine a portable dry-electrode ECG device to measure and a mobile phone to receive and record ECG signals. Healthy young college students were invited to participate in the study. Three rounds of ECG measurement were administered for each of the participants. Usability of the device was assessed through the reliability of the measures and feasibility of use during intervention. Standard error of measurement (SEM) and intra-class correlation coefficient (ICC) estimations were used for reliability, while structured questionnaire administered before and after measures was used for feasibility assessments.

A total of 234 participants enrolled in the study. No major difference was found in SEMs between trials one and two (4.96 percent, 90% CI: 4.61 − 5.37) and two and three (4.14 percent, 90% CI: 3.85 − 4.48). A slight improvement was observed in ICC of trials two and three (0.95, 90% CI 0.94 − 0.96) in comparison to one of trials one and two (0.94, 90% CI: 0.92 − 0.95). The SEM and average ICC of all trials were 3.41 (90% CI: 3.17 − 3.69) and 0.96 (90% CI: 0.95 − 0.96) respectively. Forty-five percent of participants thought the device would be suitable for their parents while 69 percent thought the device would benefit their grandparents the most.

High consistency of measures demonstrated that the device is reliable to provide ECG service delivery. The study also showed great potential of device usage in primary health care of Vietnam.

There has been a move towards the development of disease-modifying agents in Alzheimer's disease (AD) and it is likely that early disease-modifying treatments will initially be offered to people who have positive AD markers and mild cognitive impairment (MCI). Consequently, disease-modifying drugs will have distinctive features as compared to currently licensed symptomatic treatments, which makes the implications of these new agents for regulatory and health technology assessment (HTA) processes unclear.

The ROADMAP (Real-world Outcomes across the AD spectrum for better care: Multi-modal data Access Platform) project provides the foundation for a European data platform for real-world evidence in AD, and established an expert advisory group (EXAG) consisting of regulatory and HTA experts. This presentation will summarize the key lessons from the first year of ROADMAP's EXAG and identifies the next steps that are required to prepare Europe's healthcare systems for a disease-modifying drug.

The EXAG identified a need for: (i) establishing the rationale for the selection of priority outcomes in pre-clinical AD and MCI; (ii) establishing accepted outcomes for defining prevention of AD or delayed AD onset; (iii) exploring modern technology that could assist in testing cognition that could easily be used in clinical practice; and (iv) establishing caregiver-relevant outcomes (e.g. quality of life, loss of income, carer time) that are important to capture; and found that not all evidence to support modelling assumptions can be generated through RCTs, making the case for using real-world evidence.

Many of the challenges that the EXAG identified can be solved by generating better real-world data in AD. There is a clear need to agree on the outcomes that will facilitate and inform regulatory and HTA decision-making. Once the gaps in the availability of outcomes in AD will be closed, we will be one step closer towards being ready for a disease-modifying drug.

Timely recommendation by Health Technology Assessment (HTA) agencies for drug reimbursement is critical to ensure patient access to medicines of therapeutic value. In this study, HTA performance was examined in terms of their outcome and timing by looking at how 103 drugs, which gained regulatory approval from 2013 to 2015, were assessed by HTA agencies from 2014 to 2016.

Products must have received regulatory approval from one of the following regulatory agencies: EMA (Europe), Health Canada (Canada) and TGA (Australia). The first HTA recommendations were then collected from PBAC (Australia), CADTH (Canada), HAS (France), IQWiG (Germany), SMC (Scotland) and TLV (Sweden). The HTA decisions were classified as positive, positive with restrictions, negative and multiple.

Eighty-four drugs were approved in Europe before Australia and Canada. Of the studied HTA agencies, PBAC had the highest percentage of products recommended within a year from regulatory approval (93 percent). In addition, Australia had the shortest median time between first regulatory submission by any of the three agencies and HTA recommendation (553 days) as compared to Europe (616 days) and Canada (722 days). This can be attributed to the TGA/PBAC parallel process. However, Australia has the highest proportion of products receiving a negative PBAC recommendation (62 percent).

The majority of drugs were first submitted for reimbursement in Europe, but the time from regulatory submission to HTA decision was the fastest in Australia. This can be attributed to the TGA/PBAC parallel review process, which showed its benefit in reducing the overall time. A parallel review process is also available in Canada; however, it is not utilized as frequently by companies as in Australia.

In an effort to speed the assessment of new medicines while maintaining the quality of the regulatory review, facilitated regulatory pathways (FRPs) have been introduced in many countries. In this study, the effects of FRPs (expedited and conditional reviews) were investigated in terms of their influence on HTA outcomes and timing.

HTA recommendations issued between 2014 and 2016 were collected from CADTH (Canada), HAS (France), IQWIG (Germany), SMC (Scotland) and TLV (Sweden) for 90 internationalized medicines (new active substances approved between 2012 and 2016 by all regulatory agencies in the five jurisdictions). The HTA decisions were then classified into the following categories: positive, positive with restrictions, negative and multiple.

Of this cohort of internationalized medicines that received an HTA recommendation, 31 percent in Canada and 28 percent in Europe were approved via a FRP. With the exception of Scotland, expedited medicines were more likely to be appraised within a year from regulatory approval and had a shorter median time between regulatory approval to HTA recommendation than standard medicines. The largest difference was seen in Sweden, where medicines were 66.5 days faster than standard pathways when it underwent the expedited pathways. Compared to standard pathways, there were generally a higher proportion of positive and positive with restrictions recommendations when expedited pathways were used. Germany reported the largest proportional difference (31 percent) between the two pathways.

Medicines being designated for an expedited review pathway show a reduced time from regulatory approval to HTA decision. This finding suggests there is an alignment between regulators and HTA agencies on which medicines require expedited HTA pathways; however, from this data it cannot be assessed whether the reduced time from approval to HTA decision is attributed to the company strategy, HTA review time or both. Further investigation is required.

Several early access schemes (EAS) exist, which aim to accelerate patient access to new, potentially life-saving therapies. While some information exists on key schemes and their modalities, the determinants that drive adoption of a new medicine under an EAS remain unclear. We aimed to map eligibility criteria for inclusion of new medicines into the different EAS available across countries.

Health technology assessment (HTA) stakeholders across 23 countries globally were invited via email to complete a web-survey with questions on (i) items that define product eligibility for EAS designation, (ii) standards for minimum level of evidence, monitoring, and additional evidence generation for early access products, and (iii) funding arrangements for these products across settings and types of schemes. Anonymized responses were analysed using descriptive statistics.

Fourteen responses from 10 countries (including Belgium, England, France, Japan and Mexico, among others) demonstrated that “unmet clinical need” was paramount for EAS designation across all countries and types of schemes. The next most important factors were “phase-III trials underway” and “serious condition” for Compassionate Use Programme (CUP) and Named Patient Programme (NPP) inclusion (21 percent and 20 percent of respondents, respectively). “Measures in place to monitor risk” was key for CUP and NPP designation (43 percent and 27 percent of respondents, respectively), followed by “innovative product designation” for CUP and “scientific opinion” for NPP eligibility (14 percent and 23 percent of respondents, respectively). “No specific monitoring requirements” exist in Germany and Austria, whereas “reporting of adverse events” is crucial in France, England, Japan and Spain. NPP eligible products are mainly funded at a negotiated price and CUP designated products are largely provided by manufacturers free-of-charge (i.e. England, Scotland, Germany).

Eligibility criteria/requirements and funding arrangements for early access vary considerably across settings and their respective EAS. Information from a larger sample of countries is required for an all-encompassing mapping of the early access products’ characteristics.

The lack of institutional mechanisms in the Philippine Health Insurance Corporation (PhilHealth) for rationalizing spending has led to a less than optimal allocation of financial resources. The study's objective is an explicit and systematic priority setting process of selecting new interventions for PhilHealth through identification of relevant literature evidence on the themes under study, then subjecting these to stakeholder and expert consultations.

The qualitative study followed a problem solving approach to policy analysis. Bardach's Eightfold Path, supplemented by a World Health Organization (WHO) guideline on policy analysis, provided the framework. Eightfold path recommends that the analysis proceed by (i) defining the problem, (ii) assembling the evidence, (iii) constructing the alternatives, (iv) selecting the criteria for identifying the best alternative, (v) projecting the outcomes, (vi) confronting the tradeoffs, (vii) making the decision, and (viii) disseminating the results.

A six-step priority setting process to facilitate the assessment of new interventions for PhilHealth coverage was developed. The process is governed by seven accountability-based principles and four explicit criteria to evaluate interventions. Additionally, the study provided proof-of-concept for conducting local cost-effectiveness and budget impact analyses as key inputs to a national systematic priority-setting process.

This study recommended four criteria and a seven-step process for priority setting to be adopted and an overarching set of principles that will guide the conduct of such activities. The proposed priority-setting process was approved by the PhilHealth. The same process was adopted by the Department of Health in the draft administrative order for health technology assessment. This study stimulated research projects for economic evaluations of health interventions.

Decisions about which health and social services to include in the publicly funded services basket are complex. Several criteria need to be taken into account in decision-making (DM), as well as ethical, economic and organizational issues. Nowadays a global consensus supports the view that citizens’ values and preferences must guide DM. To elicit these values and concerns regarding publicly funded services, the Quebec Health and Welfare Commissioner recently conducted a vast public consultation on the population viewpoints. Parts of this consultation targeted criteria for DM, approaches to assess new or current services and perspectives on appropriateness of care.

Various consultation methods were used in complementary steps: a representative population survey (n=1850), six regional focus groups (n=62), a call for briefs (n=52) for groups that wished to share their views, consultation meetings (n=35) with diverse stakeholders and a call for personal accounts (n=2633). It also held five deliberation sessions (18 citizens and 9 experts) over the course of the project on major related issues.

The need to ensure the appropriateness of covered services was one of the strongest themes emerging from the consultation. Citizens want that the appropriateness evaluation be carried out under certain conditions: transparently, in explicit DM processes, using criteria that are clear and adaptable according to the disease or problem. The whole evaluation process needs to be well documented, showing clearly the data used and rejected, so that they can understand the decision and see on what basis it is supported. Among the usual criteria for DM, those related to cost are less valued whereas others are considered incomplete.

Citizens have clear viewpoints and expectations regarding DM criteria and processes for resource allocation. Decision-makers must take them into account to ensure that the basket of insured services is representative of social values and preferences.

Health technology has no or low added value when it is harmful and/or is deemed to deliver limited health gain relative to its cost, representing inefficient health resource allocation. A joint effort by the Health Technology Assessment International (HTAi) interest group (IG) on disinvestment and early awareness, the IG on ethics, the EuroScan network and the International Network of Agencies for Health Technology Assessment (INAHTA) is aiming to design a toolkit that could aid organizations and individuals considering disinvestment activities. We synthesized state of the art methods for identifying candidate technologies for disinvestment, and propose a framework for executing this task.

We searched systematic reviews on disinvestment and compared the methods used for identifying potential candidates. A descriptive analysis was performed including sources of evidence used and methods for selection / filtration.

Ten systematic reviews were retrieved, and the methods of 29 disinvestment initiatives were compared. A new framework for identifying potential candidates was proposed which comprises seven basic approaches based on the wide definition of evidence provided by Lomas et al.; 11 triggers for disinvestment were adapted from Elshaug's proposal, and 13 methods for applying these triggers that were grouped in embedded and ad-hoc methods.

Identification methods have been described in the literature, and have been tested in different contexts. Context is crucial in determining the ‘not to do’ practices as they are described in different sources.

In recent years, unlike developing countries, developed countries have seen an increase in the survival of women diagnosed with breast cancer and this has been attributed to early detection through screening and best treatments such as adjuvant systemic therapies with medications like Tamoxifen. The burden of breast cancer in Africa, including Ghana, lies among premenopausal women, with mean age of diagnosis being 46 years. However, survival among these women is low due to reasons such as inability to afford treatment including Tamoxifen, an older but cheaper and effective adjuvant therapy. This study therefore sought to assess the cost effectiveness of Tamoxifen compared to nothing for the adjuvant treatment of early breast cancer among pre- and peri-menopausal women in Ghana to inform funding decisions.

A Markov model was developed using TreeAge pro to incorporate effectiveness, costs and utility data. Effectiveness of Tamoxifen, rate of events and utility weights were derived from published literature. Resource utilization and costs were estimated from Ghanaian clinical expert, national health insurance scheme tariffs and medicines. The analysis was conducted from the perspective of the payer.

Patients on Tamoxifen incurred additional costs compared to those who received nothing. The key driver of costs was the cost of Tamoxifen. However, these costs were offset by the QALY gained: 3.51. The incremental cost effectiveness ratio (ICER) was GHC 666.15 (USD 150) per QALY gained. In line with the effective measure commonly used in developing countries, the ICER per DALYs averted was GHC 219.96 (USD 50). The results were sensitive to variations in the utility weights and the cost of Tamoxifen. There were no significant differences between the ICERs of premenopausal and peri-menopausal women in a subgroup analysis.

Compared to no treatment, Tamoxifen therapy is highly cost-effective for the adjuvant treatment of breast cancer among pre- and peri-menopausal women in Ghana. The results can be applied to other African countries with similar resource use and treatment protocols

Botulinum toxin type A (BoNT-A) is used in the management of lower limb spasticity in children, which affects more than 2.5 million children worldwide. BoNT-A aims to improve active function and to prevent or delay future musculoskeletal complications. The objective was to evaluate the relative efficacy and safety of different BoNT-A to manage spasticity in children, in the absence of head-to-head evidence.

A systematic literature review was conducted in March 2016 to identify all relevant randomized controlled trials. The evidence base was synthesized by means of Bayesian network meta-analyses. Scenario analyses included standardized mean differences (SMD). The endpoints were Modified Ashworth Scale (MAS), Tardieu scale-spasticity grade and Goal Attainment Scale (GAS) (SMD only) at twelve weeks post-injection, and any adverse events.

Thirty-eight studies were identified, ten of which met the inclusion criteria for quantitative synthesis. For MAS, abobotulinumtoxinA 15 U/kg/leg was significantly better compared to onabotulinumtoxinA 4 U/kg/leg (−0.99 [−1.49; –0.50]), onabotulinumtoxinA 4 U/kg/leg + casting (−0.81 [−1.30; –0.32]) and numerically (although not statistically significantly) better than onabotulinumtoxinA 8 U/kg (−0.70 [−1.64; 0.22], Pbetter=93%). For GAS, abobotulinumtoxinA 15 U/kg/leg was numerically better than onabotulinumtoxinA 12 U/kg/leg. On Tardieu scale-spasticity grade, abobotulinumtoxinA was comparable to other treatments. AbobotulinumtoxinA 15 U/kg/leg showed the highest SUCRA value on MAS and GAS. On tolerability, abobotulinumtoxinA was found to have comparable or fewer adverse events than onabotulinumtoxinA 4 U/kg/leg.

Our analyses suggest that abobotulinumtoxinA offers a comparable or favourable efficacy on tone (measured by MAS), spasticity (Tardieu scale-spasticity grade), functional outcomes (GAS) and tolerability versus onabotulinumtoxinA, in the management of children with lower limb spasticity. The results must be interpreted in the context of the heterogeneity of the evidence base and sparse evidence base.