Overview of the Budget Impact Analysis of Drugs in Hungary and Internationally

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within European HTA are ongoing and reflect the growing move towards full engagement rather than a tokenistic or tick box approach that was traditionally practised. PHP290 Review of Health Technology Assessment (HTA) Requirements for Rare Diseases Across European Countries Garcia Sanchez JJ, Sattar S, Hill CA MAP BioPharma Limited, Cambridge, UK

Objectives: To review HTA requirements currently in place for treatments for rare diseases in selected European countries (EU15 plus Nordics and Poland), to identify and evaluate differences between country requirements.  Methods: HTA requirements specific to treatments for rare diseases were obtained from HTA agencies in the selected countries. Structured desk research was conducted to identify further reimbursement considerations relating the market access of such treatments such as managed access agreements. Where available, HTA requirements were categorised into those applicable to orphan or ultra-orphan drugs. Requirements and the rationale for their application were compared against each other and each country’s rare disease plan.  Results: Definitions of orphan (prevalence ≤ 5:10,000) and ultra-orphan drug (prevalence ≤ 1:50,000) were consistent in most countries. Methods used by HTA agencies to enable access to orphan and ultra-orphan drugs included: development of specific HTA criteria (e.g. Highly Specialised Technology Appraisals – England), policies to facilitate adoption of orphan or ultra-orphan products (e.g. allowance for higher incremental cost-effectiveness ratios in England and Scotland), budget impact with specific thresholds (e.g. Germany, Italy), fast-track access (e.g. Italy, England), acceptance of lower levels of clinical trial evidence (e.g. Scotland, Germany) and higher levels of uncertainty in clinical data (e.g. Germany, Sweden). Most countries provided market exclusivity for ten years. Two countries offered financial incentives including tax exemptions (France) and reduced rebates (Spain).  Conclusions: This review highlights the significant differences in the HTA requirements for treatments of rare diseases applied across Europe. Some countries provide adjusted HTA requirements for rare diseases, but many countries still do not offer sufficient flexibility despite the clear need for an adjusted process that factors in the inherent uncertainties in clinical data and the high-cost of treatments for rare diseases. This results in different appraisal recommendations for the same drug and inequitable patient access in different European countries. PHP291 Key Drivers of HTA Decisions for “Life-Changing” Drugs Jaksa A1, Peress E2, Viana R2 1Context Matters, New York, NY, USA, 2Novartis, Basel, Switzerland

Objectives: There are mechanisms in place to reward innovative and “life-changing” drugs. Most of these mechanisms exist in the regulatory process, but what about the reimbursement process? Are “life-changing” drugs rewarded by health technology assessment agencies with positive reimbursement decisions?  Methods: A “life-changing” was defined as providing significant improvement in benefit over existing therapies. The French ASMR score of 1 and 2 and the German additional benefit score of “major” and “considerable” identified “life-changing” drugs. Drugs that were historically known to be breakthrough therapies (e.g., Gleevec and Herceptin) were also included. Assessments from NICE, SMC, CADTH/pCODR, and PBAC were analyzed. Reimbursement decisions, clinical and economic rationales for the decision, mortality, and quality of life (QoL) data were extracted.  Results: 102 assessments were included. 77% of “life-changing” drugs were issued positive decisions though this varied by agency (range 57% [PBAC] - 100% [pCODR]). 82% of clinical rationales were positive. Drugs/indications that received negative clinical rationales were more likely to be issued negative decisions. Drugs that showed statistically significant improvement in mortality or QoL were not more likely to get a positive decision than drugs that did not show improvement. Economics were a key driver of reimbursement decisions. 45% of economic rationales were positive. If the agency’s economic rationale was negative (e.g., higher cost or worse cost-effectiveness than comparator) the drug always received a negative decision.  Conclusions: 75% of the assessments for “life-changing” drugs/indications resulted in positive decisions and the majority of the agencies noted the positive clinical profiles of these “life-changing” drugs. However, cost-effectiveness was still a primary driver in reimbursement decisions. For example, drugs that demonstrated improvements in mortality or QoL were not more likely to receive a positive reimbursement decision. The presentation will further explore these themes through case studies and will compare decision rates of “life-changing” drugs to other drugs. PHP292 Overview of the Budget Impact Analysis of Drugs in Hungary and Internationally Eder B1, Nagy B2 Institute of Pharmacy and Nutrition, Budapest, Hungary, 2ELTE University, Budapest, Hungary

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Objectives: In the context of reimbursement inclusion decisions on pharmaceuticals, several aspects are taken into account. One of these is the budget impact analysis (BIA), which estimates the financial consequences of the social insurance inclusion of new therapies within a specific health-care system context. The aim of our poster was to make recommendations related to the Hungarian pharmacoeconomic guideline based on an international review of BIA.  Methods: The methodology of this study included a systematic review and analysis of the relevant national and international literature as well as publicly available materials of HTA agencies.  Results: Recommendations regarding BIA appear typically as part of pharmacoeconomic guidelines. Nevertheless, their content, extent and structure show notable differences. As a result of the research, we identified 12 domains, 6 of which play a major role in the Hungarian guideline. In the case of the other 6 domains, the Hungarian guideline needs to be developed further (model design, consistency with the cost effectiveness analysis, compared scenarios, estimation of the affected population, reduction of uncertainty, validation).  Conclusions:

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It is recommended that development proposals regarding the national guideline are to be pasted in the current structure. Furthermore, it is worth considering as to what type of supporting materials might prove helpful in the preparation/appraisal of the BIA. PHP293 Understanding Health Technology Assessment (HTA) Bodies in Major Latin-American (LATAM) Markets: Systematic Evaluation in 5 Latam Countries Nanavaty M, Gala S, Nyandege A, Ramesh V, Mwamburi M Market Access Solutions LLC, Raritan, NJ, USA

Objectives: The importance of HTA bodies in healthcare decision-making in Latin American markets has increased significantly in recent years. Rapidly growing markets, political changes, state of healthcare infrastructure add to the complexity of patient access to medicines. HTA bodies in LatAm countries vary significantly in characteristics, attributes, methodologies employed, and referencing practices as do the payer reimbursement architypes. The objective was to characterize the different HTA bodies used in LatAm markets.  Methods: We conducted a systematic review of HTA bodies’ documentation of 5 LatAm markets (Brazil, Chile, Colombia, Cuba and Mexico) and of peer-reviewed publications through PubMed. Over 350 characteristics, attributes assessed and methodologies used by each HTA were assessed and mapped.  Results: Documentation for all 5 markets had limited information available on HTA websites or in peer-reviewed publications. Cuba, Mexico and Brazil are among the longest established HTA bodies in LatAm. The HTA body in Brazil is a department of the national health ministry, while in Chile and Colombia HTA bodies are in-regulatory agencies. All markets consider clinical evidence including clinical trials, systematic literature reviews, meta-analyses and economic analysis. Specifically, Chile, Colombia and Mexico require budget-impact analyses. No markets utilize an explicit ICER thresholds, 2 markets publish reports online, 1 market considers rare diseases, 2 markets consider unmet needs, and all 5 markets are a part of Red ETSA, a commission that promulgates the integration of policies, information diffusion and evidence generation. Brazil, Colombia and Mexico have a Memorandum of Understanding with National Institute for Health and Care Excellence (NICE, UK).  Conclusions: HTA bodies in LatAm are influenced by payer architypes in respective countries. HTA bodies rely on HTA reviews from other countries and regional inter-communication regarding evidence-based decision-making than most other regions in the world. These markets are currently developing policies regarding cost-effectiveness evidence and rare diseases. PHP294 Multi-Criteria Decision Analysis to Evaluate the Femtosecond Laser-Assisted Cataract Surgery in Children Giorgia Tedesco G1, Francesco Faggiano F1, Buzzonetti L2, Petroni S2, Di Pinto G1, Chessa G2, Pietro Derrico P1, Matteo Ritrovato M1 1Bambino Gesù Children’s Hospital, Rome, Italy, 2Bambino Gesù Children’s Hospital, Fiumicino (Roma), Italy

Objectives: The aim of this study is to describe the application of Decisionoriented HTA method to assess the femtosecond laser-assisted cataract surgery (FLACS) compared to conventional cataract surgery (CCS).  Methods: This study reviews the spectrum of evidence regarding the feasibility, safety, technical and organizational features, costs and economic factors of FLACS compared to CCS, by using doHTA methodology. The information is summarized in a hierarchical decision tree by means of Key Performance Indicators (KPI), subsequently weighted through pairwise comparisons. For each pair of elements in the pairwise comparison, a verbal judgment by professionals involved in the assessment was gathered, and then converted in the numerical ratings. Lastly, FLACS and CCS were ranked against lowest indicators of decision tree. A global score was determined delivering a consequent ranking between the alternatives.  Results: According to the professionals judgments, “safety” along with “clinical effectiveness” make up almost 60% of the whole evaluation, while the weights associated to “costs and economic evaluation” dimension was 14.63%, followed by “technical characteristics of technology” (14.28%) and “organizational aspects” (10.51%) domains. FLACS seems to overcome CCS with several important developments such as increased precision of anterior capsulotomy, reduced ultrasound power requirement during phacoemulsification, decreased collateral tissue damage. Notwithstanding such clinical improvements, FLACS is more expensive than its comparator; the relative costs were also analyzed in the cost-minimization analysis.  Conclusions: The Hospital’s Top Management has decided to consider the doHTA evaluation as a whole. Indeed, although FLACS had the highest capital investment cost, its safety, clinical and technical features led to its high score. Moreover, due to the peculiar features of doHTA (i.e. the quantitative integration of different aspects of the assessment and their synthetic representation), the Top Management has provided a timely response between both alternatives: the implementation of FLACS seems to be attainable. PHP295 Results of HTA in Brazil: The Performance of Conitec in its First Five Years Rabelo RB1, Canuto Santos VC1, Petramale C2, DA Silveira LC1 1Department of Management and Incorporation of Health Technology, National Committee for Health Technology Incorporation (CONITEC), Brazilian Ministry of Health, Brasília, Brazil, 2Department of Management and Incorporation of Health Technology, Ministry of Health of Brazil, Brasília, Brazil

Objectives: Since the creation of the National Committee for Health Technology Incorporation in the Brazilian Health System (CONITEC), a new phase started to the public Brazilian Health System (SUS): a continuous updating of the system based on health technology assessment. CONITEC was created by federal law in 2012 and it is responsible for advising the Ministry of Health regarding the incorporation or disinvestment of health technologies. The whole process involves a