Technology Assessment Bodies Research Articles

HTA60 Health Disparities in Health Technology Assessment: An Opportunity for Improvement

Equity in the provision of healthcare and access to health resources is vital, and payers/health technology assessment bodies (HTAs) are in a position to support equitable access to technology within their patient populations.

Expanding the Role of Early Health Economic Modelling in Evaluation of Health Technologies Comment on "Problems and Promises of Health Technologies: The Role of Early Health Economic Modeling".

In this commentary, we discuss early stage assessments of innovative medical technologies both in terms of methods applied as well as their use in healthcare decision-making. We argue that cost-effectiveness alone may be too reductive if taken as the only decision rule, and it would benefit from being used within a broader evaluation framework. We discuss innovative methods which may contribute to better estimate the potential costs and consequences of a technology in the absence of solid clinical data, as frequently the case in early assessments. Finally, we comment on the potential synergies which may take place should early economic models be used not only by technology developers alone but as a negotiating base during early dialogues with health technology assessment (HTA) bodies.

VP07 Cost-Effectiveness Of HTA Fees

IntroductionHealth technology assessment (HTA) bodies evaluate the clinical and/or economic impact of new therapies to inform public reimbursement decision-making. This research evaluates the value for money of current or proposed fees for HTA in countries with mandatory cost-effectiveness HTA bodies relative to their respective public drug expenditure.MethodsHTA appraisal fees were identified from publicly-available websites: National Institute for Health and Care Excellence (NICE), Canadian Agency for Drugs and Technologies in Health (CADTH), Institut National d'Excellence en Santé et Services Sociaux (INESSS), and Pharmaceutical Benefits Advisory Committee (PBAC). Annual national public drug expenditure (ANPDE) were sourced from the National Health Service England, Canadian Institute for Health Information, and the Pharmaceutical Benefit Scheme.ResultsNICE is proposing to charge GBP 126,000 (EUR 142,582) for a single technology or highly specialized technology appraisal, CADTH charges CAD 72,480 (EUR 48,576) for a Schedule A submission, INESSS charges CAD 38,921 (EUR 26,089) for the first evaluation of a new drug or new indication, and PBAC charges AUD 136,716 (EUR 87,576) for a Major Lodgment. The ANPDE in England: GBP 16 billion (EUR 18.1 billion), Canada: CAD 14.5 billion (EUR 9.7 billion), Quebec: CAD 4 billion (EUR 2.7 billion) and Australia: AUD 8.7 billion (EUR 5.6 billion). The appraisal cost to drug expenditure ratio for these countries/regions were: 126,984, 200,055, 102,772, and 63,636, respectively.ConclusionsHTA submissions in the United Kingdom, Canada and Australia require financial contributions from manufacturers. These contributions bear little relation to the market size and cumulatively exceed EUR 300,000 (assuming no resubmissions). By adopting charging/cost recovery models, HTA bodies are aiming to reinvest the proceeds to increase the efficiency and capacity of appraisals, expediting patient access. However, these fees may be burdensome, especially for SMEs with promising therapies for orphan/rare diseases, and they may thus have the potential to deter/delay their submissions.

OP93 Collaboration Between Health Technology Assessment And Procurement: A Rapid Mixed-Methods Study

IntroductionThe Irish Health Service (HSE) Health Technology Assessment Group (HTAG) aims to maximise the impact of its work by collaborating with HSE Procurement, formalised through an evidence-based Memorandum of Understanding (MOU). This study aims to inform the MOU.MethodsA sequential mixed-methods study design was used. A rapid review of the literature identified no substantive body of evidence on collaboration between independent national health technology assessment (HTA) and procurement bodies. Personnel involved in HTA or procurement were invited by email to complete a survey, take part in an interview, or both. The quantitative and qualitative data were analysed using descriptive statistics and thematic analysis, respectively. Findings were integrated using a conceptual framework that examined the complementarity of HTA and procurement processes relevant to an MOU.ResultsThirteen surveys were completed (response rate was 13 percent). Eleven interviews (five Ireland, two Canada, three UK, one New Zealand) were conducted between August and November, 2017. No formalised collaboration between independent national HTA and procurement bodies was identified. However in New Zealand, HTA and procurement are an integrated function of the Pharmaceutical Management Agency (PHARMAC). In other jurisdictions, successful ad hoc collaborations occurred where there was a clear need expressed by Procurement for additional evidence required for decision-making, and where HTA personnel tailored their research approaches accordingly. Key themes to successful collaboration were relationships, communication, clear roles, rigorous research and ‘system support’. Good individual relationships and ready access/communication promoted successful outcomes. Successful outcomes included improved clinical practice, and major cost savings. Collaboration may be focussed on: innovative or established devices; specific types of HTA/research products; specific categories/specialties; or specific procurement departments.ConclusionsAll participants considered collaboration to be beneficial but requiring good relationships and ‘system support’. Furthermore, successful collaboration requires clarity regarding the purpose, parties involved, their roles, responsibilities, modes of communication, information to be shared, and the expected outcomes.

VP74 Orphan Black Box: Explanatory Principles

IntroductionOrphan legislations over the past thirty years have successfully increased the number of drugs receiving marketing authorization for rare diseases. However, for a therapy to be accessible to most patients, it requires not only marketing authorization, but market access via public reimbursement. In many major markets, the pricing and reimbursement of new therapies is based on an assessment by a national Health Technology Assessment (HTA) body, for which economic value is typically a key consideration. This research evaluates the outcome of HTAs of orphan drugs in Europe.MethodsHTA decision data (to 31/08/2017) was extracted from Gemeinsame Bundesausschuss (G-BA), Haute Autorité de Santé (HAS), National Institute for Health and Care Excellence (NICE), Pharmaceutical Benefits Advisory Committee (PBAC), and Scottish Medicines Consortium (SMC) websites. EC-approval data was extracted from the European Medicines Agency (to 31/08/2017).ResultsOnly a small minority of drugs for orphan diseases received full recommendations for their licensed indication(s) by NICE (3/35, 9%), SMC (8/66, 12%) and PBAC (1/44, 2%). 37% (26/70) of drugs assessed received positive HTA outcome by HAS (ASMR I-III). In Germany, all approved orphan drugs (100/100) received automatic additional benefit post regulatory approval by G-BA.ConclusionsThere have been significant challenges for manufacturers in converting regulatory approval of orphan drugs into commercial success and optimised market access. Attaining positive HTA appraisals for these drugs, which have been approved under expedited regulatory pathways on a less than fully mature dataset, whilst also having high prices, due to small patient populations, limiting commercial returns, may necessitate increased utilisation of alternative reimbursement mechanisms.

OP91 Developing A Celtic Connections Regional Health Technology Assessment Alliance

IntroductionThe Irish, Scottish and Welsh national Health Technology Assessment (HTA) bodies (Health Information and Quality Authority, Health Technology Assessment Group, Scottish Health Technologies Group, Health Technology Wales) have recently (2018) established a ‘Celtic connections’ regional HTA alliance on non-medicine technologies. The primary purpose is to add value by realizing potential economies of scale and scope in non-medicine HTA efforts.MethodsA Memorandum of Understanding (MoU) was agreed to: formalize collaboration and partnership working; improve shared understanding of work programs and processes; collaborate on and co-produce evidence reviews of mutual interest; increase both the volume and range of technology topics for which advice is developed in each nation; promote knowledge exchange; and enhance professional and personal development for each agency's staff.ResultsEarly benefits include: collaboration on one technology topic resulting in the production of bespoke guidance in three countries; an update of a partner's rapid review; identification of a further potential topic collaboration (sacral nerve stimulation); a six month senior staff secondment; and reciprocal observer membership on each country's national committees. Other general benefits have included: reduced duplication of effort; improved quality assurance through ‘critical friend’ peer review; enhanced access to methodological advice and a broader range of stakeholders; and development of a forum for discussion and peer support.ConclusionsThe alliance offers real potential to optimize use of the scarce resources for non-medicine technologies across the three countries and increase evidence review and guidance volume through adapting or co-producing outputs. Longer term benefits are anticipated to include: improved knowledge exchange; advancing skills of staff; building and broadening capacity through shared learning and access to a wider professional peer group; improved staff recruitment and retention; production of joint publications and other modes of dissemination; and increased profile for each country's work.

PP47 Defining The Needs And Preferences Of Patients With Dry Eye Disease

Introduction:Dry eye disease (DED) is a common condition that significantly impacts patients’ quality of life. Previous studies have explored the impact of DED on patients’ lives qualitatively; however, patients’ preference structures have not been thoroughly explored quantitatively.Methods:A targeted literature review and social media listening project guided design of a discussion guide for in-depth patient interviews (n=12). These, in turn, guided construction of a quantitative questionnaire administered to moderate to severe DED patients, 40 per country in Australia, Germany, United States and United Kingdom (total n=160). Patients’ preference structures were explored through an online survey using a self-explicated conjoint methodology, because of its high respondent-friendliness. Additionally, we administered the EQ5D-5L instrument to determine the health states/utilities of patients. Reaction to a hypothetical novel treatment was further obtained to check for convergent validity with the self-explicated conjoint. Finally, we asked respondents to rate the ease and relevance of the questionnaire to them.Results:Qualitative research uncovered important patient perspectives that were built into the quantitative survey. For example, patients seek medical advice when their symptoms are not improving. Patients’ lives are most affected by sensitivity to light, itchy and tired eyes and an inability to perform computer/screen work; however, of most concern/worrying to them is that their DED will get worse and they go blind. Results from the quantitative preference research will also be shared and its implications for future clinical trials in DED outlined. The results of the patient research and preference study are to be shared with health technology assessment (HTA) bodies and regulators through the early dialogue scientific advice process.Conclusions:A process of using qualitative research to determine what matters to patients and then quantification through respondent-friendly preference research can identify outcomes that are most patient-relevant, to inform future drug development strategies.

OP37 Can Local Ultra-Orphan Patient Evidence Shape Global Understanding?

INTRODUCTION:Written patient evidence is submitted to the National Institute for Health and Care Excellence (NICE) by patient organizations and their nominated patient experts. We reviewed the impact that patient evidence had on the committee's decision making. This local learning can help generate global opportunities for Health Technology Assessment (HTA) bodies and patient groups to further develop their understanding and methodology about how patient evidence can support HTA decision making for ultra-orphan conditions.METHODS:There were two phases.Phase one was an online questionnaire about the impact of patient evidence on the committee's decision making for ultra-orphan HTA evaluations. It was sent to the committee chair, lay committee members and selected other committee members.Phase 2 developed the initial questionnaire findings using in-depth interviews with the committee chair and the lay members. These gained further understanding of the impact of patient evidence and the themes raised.RESULTS:Key findings showed patient evidence was helpful to understand the: •Burden of disease•Patient population•Likely uptake of new medicines•Impact on carers.For ultra-orphan conditions, where other forms of evidence are scarcer, patient evidence is fundamental to understanding patient needs, the impact of the disease, patient population and preferences.CONCLUSIONS:Patient evidence was useful for the committee in different ways; it provided the committee with new evidence and it helped the committee understand and interpret the evidence submitted by others. Both are key to committee decision making. It was clear that due to the very small patient population, patient groups knew the patient population, their stage of disease, and their preferences in detail.The findings will be used to inform an updated patient submission template for ultra-orphan HTAs, and supporting guide. These will be available on the NICE website and offered to the HTAi Interest Group on Patient and Citizen Involvement so they can be shared globally.

VP02 Are Journal Editors A Barrier To Publication Of Real World Evidence?

INTRODUCTION:Health technology assessment (HTA) bodies often rely heavily upon evidence from peer-review publications to inform their recommendations. Since journal editors attitudes likely influence the types of study designs that are accepted through the peer-review process and on to publication, journal editors serve as gatekeepers to translation of evidence, including real word evidence (RWE), into practice. Given past skepticism of RWE study designs, journal editors perceptions of and possible biases toward RWE are important to understand as they may impact dissemination and, therefore, uptake of research findings. The objective of this study was to examine current views of journal editors regarding the: (i) value of RWE studies and (ii) challenges editors face in managing, reviewing, and publishing RWE manuscripts.METHODS:Journal editors representing general medicine (GM), specialty medicine (SM), health policy/services research (HSR) were invited to participate in a telephone interview, a survey, and an in-person, roundtable discussion.RESULTS:In total, seventy-nine journals were approached, resulting in: 15 interviews (GM = 2; SM = 5; HSR = 8), 17 survey responses (GM = 2; SM = 6; HSR = 9) and 8 roundtable participants. RWE was viewed favorably by interviewed editors (n = 15). Characteristics of high-quality RWE manuscripts included: research question novelty/relevance, rigorous methodology, alignment of data with question, and the extent data-source advantages are optimized. Similar manuscript review processes and challenges were voiced for RWE and other study designs. HSR editors were more likely than SM or GM editors to participate, potentially indicating these researchers are more comfortable or interested in RWE. A possible study limitation was that editors favorable toward RWE may have been more likley to participate.CONCLUSIONS:Peer-review journal editors appear to have favorable views regarding RWE studies and can be accelerators to dissemination of RWE findings. However, they do report that studies and processes could be improved. One suggested improvement included a checklist for editors to speed rejections and improve communications with authors.

VP216 Health Technology Assessment's Balance Between Additional Data, Adoption, And Patient Access

INTRODUCTION:Historically, many Health Technology Assessment (HTA) bodies were developed with a focus on addressing rapidly rising drug costs and the unique need to evaluate each drug as a de novo entity. The degree to which the unique needs for evaluating technologies vis a vis drugs are reflected in distinct HTA methods and activity is to date understudied.METHODS:We examined HTA's reviews of two technologies: WATCHMAN™, a device to reduce the risk of stroke in certain patients and Alair™, a procedure-based treatment for severe asthma. Both technologies have been extensively reviewed by HTA bodies and payers in many countries. These HTA reviews are compared to a convenience sample of these HTA's bodies reviews of drugs and qualitative differentiators between these two categories explored.RESULTS:The differences and similarities (for example, in rigor and necessity of evidence) between US Section 510(k) clearances, US premarket approval (PMA), and US new drug application (NDA) regulatory pathways have not been clearly understood by HTA or reflected in their methodologies employed. Additionally, emergent methodologies such as Bayesian statistical analyses may encounter challenges within technologies reviews. HTA bodies may not be cognizant of development timelines or the timelines of comparators. Finally, HTA bodies may overestimate device adoption rates.CONCLUSIONS:The differences in evidence requirements for regulatory approval between US 510(k), US PMA, and US NDA pathways have not been reflected in different methodological approaches within HTA bodies reviews. Opportunities and novel methods are needed for HTA bodies to derive imputed comparisons between technologies that may have inherently incongruent timelines. Finally, HTA bodies could benefit from methods to more accurately estimate projected adoption curves. Challenges exist using frameworks, paradigms, and methodologies initially established for, and commonly used for, pharmaceuticals on device evaluations; leaders of HTA methods can improve the situation by providing guidance and recommendations for more appropriate HTA methods to evaluate devices.

Development of Innovative Medicines for European Patients and Impact of Brexit

European patients may benefit from innovative medicines only at the end of a complex process with a sequence of positive decisions on different levels by different stakeholders. The decision of the industry to invest in a usually global clinical development must be followed by a European marketing authorization decision and a mostly national decision on price and reimbursement until finally patients and their physicians can make an individual treatment decision. Development strategies must consider the evolution of scientific and procedural requirements. Current trends are characterized by an enhanced cooperation of regulators and health technology assessment-bodies. The increasing availability of innovative personalized or precision medicines is reflected in the new procedural tools like European Medicines Agency’s priority medicines scheme and adaptive pathways concept. The UK decision to leave the EU will have consequences for their contribution to the European regulatory and health technology assessment network. Current strategies for the successful development of innovative medicines may need adjustments to address both scientific and political changes.

Health protection in times of economic crisis: Challenges and opportunities for Europe

STOA, the European Parliament's technology assessment body, and the European Observatory on Health Systems and Policies recently organised a workshop on the impacts of the economic crisis on European health systems. Evidence of the impact of the recent financial crisis on health outcomes is only just beginning to emerge. Data suggests that this latest recession has led to more frequent poor health status, rising incidence of some communicable diseases, and higher suicide rates. Further, available data are likely to underestimate the broader mental health crisis linked to increased rates of stress, anxiety, and depression among the economically vulnerable. Not only does recession affect factors that determine health, but it also affects the financial capacity to respond. Many European governments have reduced public expenditure on health services during the financial crisis, while introducing or increasing user charges. The recession has driven structural reforms, and has affected the priority given to public policies that could be used to help protect population health. The current economic climate, while challenging, presents an opportunity for reforming and restructuring health promotion actions and taking a long-term perspective.

A Systematic Review of Economic Evaluations in Second and Later Lines of Therapy for the Treatment of Non-Small Cell Lung Cancer

Non-small cell lung cancer (NSCLC) is associated with high morbidity and mortality. Surgery is generally accepted as the first-line treatment in patients with advanced/metastatic NSCLC, followed by radiotherapy and chemotherapy as second-line treatments. Docetaxel or erlotinib are generally recommended as the first-line chemotherapy option. The objective of this review was to identify previously published economic evaluations in NSCLC for second- and later-line treatments in order to (i) determine common modelling approaches and (ii) establish the relative cost effectiveness of these treatments. An overview of model critique was also produced to identify common criticisms from health technology assessment (HTA) bodies on the models submitted. MEDLINE, Embase, EconLit, MEDLINE in Process(®) and NHS Economic Evaluation Database (NHSEED) were searched (database start-October 2011), along with proceedings from eight major conferences (2007-2011). National Institute for Health and Clinical Excellence (NICE), Scottish Medicines Consortium (SMC), Pharmaceutical Benefits Advisory Committee (PBAC) and Canadian Agency for Drugs and Technologies in Health (CADTH) websites and the International Network of Agencies for Health Technology Assessment (INAHTA) database were also searched for appraisals in second- or later-line NSCLC. All published studies and HTA appraisals that reported economic evaluations of interventions used in current clinical practice as second- or later-line treatment in patients with advanced/metastatic NSCLC were included. Only studies in English were considered for inclusion. Studies which met the eligibility criteria after the screening of full-text articles were extracted by a reviewer and checked by a second party. Where multiple publications were identified describing a single study, the extracted data were compiled into one entry. A total of 29 studies were included which clearly evaluated second-line or later-line regimens. Most studies were either cost-effectiveness or cost-utility evaluations. Three-state transition Markov models were frequently used in cost-effectiveness and cost-utility evaluations. The model inputs were well reported and commonly consisted of data from pivotal trials. Sensitivity analyses were conducted in the majority of studies and covered variables such as cost, effectiveness, hospitalization and treatment duration. Therapies (docetaxel, pemetrexed and erlotinib) are for the most part cost-effective/cost-saving second-line therapies compared with best supportive care (BSC). Six erlotinib HTAs, across NICE, SMC, and PBAC, and four pemetrexed HTAs, one by NICE and three by SMC, were identified. The CADTH website did not provide sufficient detail on the appraisals and was excluded. Certain aspects of the models and model assumptions, e.g. efficacy inputs, were criticized or determined unjustifiable by the NICE, SMC and PBAC appraisal committees. Erlotinib and pemetrexed were considered to be cost effective versus docetaxel by NICE and SMC in the final submissions. PBAC considered erlotinib to be cost effective versus BSC following a price reduction in 2008. Three-state Markov models are often used to conduct economic analysis in NSCLC and are regarded as appropriate to HTA agencies. Docetaxel, erlotinib and BSC are suitable comparators that should be considered for use in the model in the UK and Australia. Further, manufacturers should carefully select underlying assumptions used in the model, for both costs and clinical inputs, where the latter is derived from direct head-to-head trial data.

The Changing Face of Risk Governance: Moving from Precaution to Smarter Regulation

STOA, the European Parliament's Technology Assessment body, which I had the honour to chair, has a mission to: (i) assess the impact of introducing and promoting new technologies, and (ii) identify the relevant policy options. In the context of its work, STOA has to address many different issues. One of the issues deserving STOA's attention is the changing face of risk governance: “Moving from precaution to smarter regulation”.This is currently being debated on various levels, including that of the WTO (World Trade Organization), where science-based decision-making has always played an important role. The JRC (Joint Research Centre) is very active in this field and has developed, among others, a science-based approach to risk assessment in the area of nanotechnology. With the comitology procedure becoming more and more important, the European Parliament and its members have to assume their role with respect to risk management and explain it to the public.