HTA Uncovered. The introduction of economic evaluations have hitherto not influenced ASMR ratings claimed by manufacturers in France

Transcription

1 Issue No. 8 June 0 HTA Uncovered The introduction of economic evaluations have hitherto not influenced ASMR ratings claimed by manufacturers in France Since the publication of the 008 Social Security Financing Law (Loi de financement de la sécurité sociale, LFSS), the French National Authority for Health (Haute Autorité de Santé, HAS) has been charged with the task to perform medico-economic evaluations to determine the most efficient therapeutic strategies and provide recommendations accordingly. To this end, on July 008 HAS instated the Economic and Public Health Assessment Committee (Commission Evaluation Economique et de Santé Publique, CEESP). the drug is expected to have a significant impact on spending (the threshold has been set at 0 million euros per year by HAS on 8 September 03), and in its application to the CT, the manufacturer claims a major, important, or moderate Improvement of the Medical Benefit (Amélioration du Service Médical Rendu, ASMR level I, II or III) or the equivalent Added Clinical Value (Amélioration du Service Attendu, ASA) Because these ratings have an important impact on subsequent pricing negotiations, ASMR/ASA levels I-III have been sought much more frequently by manufacturers than they have actually been awarded by the CT/CNEDiMTS. Yet, given the recent changes, it has been suggested that applicants might now be inclined to seek an ASMR/ASA IV or V rating more often in order to avoid the economic evaluation process 3. To gauge the impact of the new regulations in France, we investigated trends in ASMR levels requested by pharmaceutical companies before and after the publication and adoption of the new decree. For this purpose, all CT meeting minutes published by HAS between January 0 and March 0 and contained in our HTA Accelerator database were reviewed 4. Four distinct time periods were evaluated:. before publication of the decree (i.e. between 4 January 0 and 3 October 0). between the publication of the decree and the day it took effect (i.e. between 4 October 0 and 3 October 03) Economic evaluations for establishing reimbursement have, however, only become compulsory since a decree to amend the 0 LFSS was passed by the Hollande government in October 0 and took effect one year later. The decree requires manufacturers of medicines or medical devices to submit a cost-effectiveness model, to be evaluated by the CEESP in parallel with the Transparency Committee s (Commission de la Transparence, CT) evaluation process for medicines or the National Committee for the Evaluation of Medical Devices and Health Technologies (Commission nationale d évaluation des dispositifs médicaux et des technologies de santé, CNEDiMTS) for devices, if the following two conditions are met: 3. the first year following the adoption of the decree (from 4 October 03 to 3 October 04) 4. CT meeting minutes published more recently and available at the time of our analysis (from October 04 to 8 March 0) Out of a total of 30 applications, among which 334 related to a single and 8 concerned two indications, 3 were retained for the final analysis (Table ). One hundred and twelve submissions concerned medicines to be included on the list of medicines for hospital, while the list was not specified in 7 applications. These 9 requests were therefore excluded.

2 HTA Uncovered June 0 Type of Period submission 3 4 Total Original submission Extension of indication Reassessment of ASMR Renewal Total Table. Overview of the 3 applications included in the analysis requested by applicants in France. Although manufacturers could avoid the economic evaluation process by claiming an ASMR IV rating, they would most likely also forsake the opportunity to obtain a price premium over comparators as ASMR IV-products should in principle not affect the drug budget. Theoretically, the Transparency Commission could even oblige products to undergo an economic appraisal by awarding an ASMR of I, II or III rating to a medicine regardless of the manufacturer s claim. To our knowledge, this has not yet occurred. As shown in Figure 3, only a minority of products have actually obtained such a beneficial CT evaluation in the last few years. Between January 0 and 8 March 0, an ASMR I, II, or III rating was sought in 83 out of 3 cases (36%). As shown in figure, the proportion of these applications ranged from 7% in period to 44% in period 4. Moreover, this proportion was similar among the various submissions types: 34% for original submissions (47/38), 40% for indication extensions (6/6), 36% for ASMR reassessments (9/), and 33% for renewals (/3). Unsurprisingly orphan drugs formed a clear exception with 7 out of applications (77%) seeking either a major, important, or moderate ASMR rating (Figure ). 6 0 ASMR I ASMR II ASMR III ASMR IV ASMR V NS 00% 80% 60% 40% 0% 0% Period Period Period 3 Period 4 Figure. ASMR levels requested by manufacturers between January 0 and March 0 Based on these results, which do not show a clear evolution over time, it seems that the introduction of compulsory economic evaluations has not had a significant impact on ASMR ratings I II III IV V Not requested/specified Figure. ASMR ratings requested for orphan drugs 00% 80% 60% 40% 0% 0% Figure 3. ASMR levels awarded by HAS between January 0 and March 0 3 Period Period Period 3 Period 4 I II III IV V SMR insufficient Not assessed These results reflect the relatively low marginal cost for most pharmaceutical companies of submitting a cost-effectiveness model, which can be developed in parallel for other countries that require proof of costeffectiveness. It would be interesting to see how the CEPS uses efficiency opinions in its price negotiation with the manufacturer when the Transparency Committee attributes an ASMR IV Pierre Moïse Engagement leader Quintiles Advisory Services

3 HTA Uncovered June 0 Growing need for Health Technology Assessment (HTA) in Japan Population trends in Japan are precipitating the growing burden of healthcare expenditures in the country. Japan has had a negative population growth rate in recent years, due in part to declining birth rates, while the country continues to have one of the highest life expectancies in the world. By 060, over 40 percent of the population in Japan will be over 6. Healthcare expenditure through its universal public healthcare system outpaces economic growth; over ten percent of Japan s GDP is spent on healthcare, and average total health expenditure is increasing at a rate of.7 percent annually 6. As the second largest pharmaceutical market in the world 7, Japan s per capita pharmaceutical expenditure grows at an average annual rate of three percent 8, which will become an increasing cost burden in line with the aging population. Results of HTA pilot program are expected this spring Historically, Japan has tried unsuccessfully to implement HTA as a cost containment measure 9. The Japanese Association of Medical Technology Assessment was established in 98, but its development has been hindered by a lack of funding and clear direction. A five-year strategic plan was passed in 007 in an effort to streamline review of pharmaceuticals and medical devices. In 009, the FDA and Ministry of Health, Labour and Welfare (MHLW) announced closer regulatory collaboration to discuss the contents of individual submissions. The purpose of which, was for regulators to help design clinical trials which can be accepted by both agencies, in an attempt to eliminate redundant clinical trials. Subsequently, in the last couple of years Japan started an HTA pilot program with five products from participating companies. Companies will provide a set of pharmacoeconomic data to the government s MHLW working group, who then conduct analysis and come up with a recommendation. These results are anticipated to be made publically available soon (spring 0), and HTA guidance around possible implementation is expected to be introduced in April 06. Based on results of the pilot program, the current pricing scheme will be adjusted. The scope of drugs subject to the HTA review is still under determination. The successful introduction of HTA will largely depend upon consensus among all stakeholders in terms of methodology and interpretation of the results. Whilst much remains to be determined, the implementation of HTA will undoubtedly have a significant impact on pharmaceutical development in Japan Yujiro Tsutsumi Vice President Commercial Solutions Quintiles Japan Current pricing & reimbursement is cost-focused To date, there has been little incentive for the pharmaceutical industry in Japan to conduct formal economic analyses due to the organization of the current healthcare system, which is centralized through the MHLW. In 99, the MHLW recommended including health economic evidence in the dossier, however, it was not mandated 4. The current pricing mechanism is a unique aspect of Japan s healthcare system. The concept of HTA has been embedded in this pricing scheme, meaning the price is set based on comparing existing products in the same drug category either in Japan or comparable markets, with no room for negotiation 4. Whilst no negotiation is stipulated officially, manufacturers attempt to influence the choice of comparator and any price premiums. If no comparator is available, a cost-plus methodology is used. If there is a premium deserved for a drug, that premium is based on an assessment of innovativeness and safety advancement 0. Every two years, the MHLW undertakes a pricing revision for all drugs. In 07, an additional exceptional price revision will be carried out to neutralize the effects of an anticipated increase in consumption tax from 8% to 0%. These continuous price revisions have a significant impact on the pharmaceutical industry, which currently consider HTAs as a vehicle for price reductions. Success of the future HTA implementation in Japan will largely depend on consensus among stakeholders in terms of methodology, which is an ongoing discussion. While uncertainties remain, the goal from a regulatory perspective is that implementation of HTA in Japan will contain costs while still promoting innovation in drug development.

4 HTA Uncovered June 0 Implementation and future landscape of HTA in Japan Although these uncertainties remain, Japan s HTA system and processes will undoubtedly be unique. While many industry stakeholders are involved in the pilot project, the formal HTA process will rely on academia to conduct assessments. Assuming that the same criteria from the pilot phase will be implemented in 06, then drugs will be subject to assessment based upon: () expected peak sales and cost benefit, () same drug subject to HTA in other countries, and (3) sufficient dataset available for local HTA assessment. Due in part to cultural differences, HTA is being discussed in isolation of other Asian countries and Japan is unlikely to take strong leadership for HTA in the Asian Pacific region. Rather, Japan is looking to European HTA systems to identify best practices and guidance, carefully considering each system and how they could apply to the setting in Japan. Japan s Central Social Insurance Medical Council (Chuikyo), is discussing potential Key Performance Indicators (KPIs) for evaluation criteria, with Quality Adjusted Life Years (QALYs) in the forefront. Beyond QALYs, Chuikyo is considering the benefits and drawbacks of measuring therapeutic effect based on overall survival, efficacy, and laboratory values. In the context of costeffectiveness analysis, Chuikyo is discussing whether to consider direct costs as well as indirect costs (e.g. loss of productivity, wider societal care). From a cultural perspective, QALYs are counter to the widely held belief that life is priceless and cannot be converted into a metric. The complexity of implementing HTA and measuring cost-effectiveness, while considering Japanese culture, cannot be overstated, and the Chuikyo has not yet made a decision on what metrics will be used. References. Legifrance - Décret n 0-6 du octobre 0 relatif aux missions médico-économiques de la Haute Autorité de santé. JORF n 03 du 4 octobre 0 page. Available from: The first draft of the decree did not include medicines for use in hospitals nor medical devices 3. Moïse P, Katz S. A new Era for Pricing and Reimbursement in France. HTA Uncovered. Issue No.3 November Given the uncertainty of the decree s application to medical devices when it was first introduced in 0 it was decided to not review decisions by the CNEDiMTSWorld Bank. Total health expenditure by country as % of GDP, Data is available from the World Bank website: es. Kamae I (00). Value-based approaches to healthcare systems and pharmacoeconomics: South Korea, Taiwan, Thailand and Japan. Healthcare and Pharmaecoeconomics in Asia, 00; 8(0): Bradshaw S (04). 04 Japanese HTA guidelines: Industry uncertainty persists. World Pharmaceuticals. 7. Health at a Glance 0: OECD Indicators. Growth in real per capita pharmaceutical expenditures, Available from: 8. Hisashige A (009). History of healthcare technology assessment in Japan. International Journal of Technology Assessment in Health Care, : Supplement ; Grainger D (0). Considerations of Health Technology Assessment in Japan. PhRMA.. HTA Accelerator: facts and figures Among all HTA evaluations published between January 04 and May 0, 36% of products obtained a positive while 30% received a negative recommendation. The most important restrictions, which applied to % of therapies, limit the use of these products to a specific patient subgroup or treatment line only. Figure 4. Outcome of HTAs published between January 04 and May 0

5 HTA Uncovered June 0 Events and publications Meet us at the following conferences PharmAccess Leader Forum, October 0, Berlin, Germany European Pricing & Reimbursement, 3 October 0, Regents Park, London, United Kingdom ISPOR 8th Annual European Congress, 7- November 0, Milan, Italy Recordings of presentations can be found on: Publications Real-world evidence generation for evolving stakeholder needs Louise Parmenter, Anke van Engen Seizing the future in oncology Improving the clinical development of immunotherapies Brad Smith, Chris A. Learn, Eric Groves Value is the Target: a stakeholder survey of perceptions on value in healthcare An integrated approach to biosimilar development & commercialization Deepa Dahal, John Doyle, and Kamali Chance Oncology drug development and value-based medicine John J. Doyle and Brian Huber Contact us Quintiles Advisory Services Toll free: Direct telephone: International: On the web: advisoryservices@quintiles.com Anke van Engen Office: + 3 (0) Mobile: + 3 (0) anke.vanengen@quintiles.com Kavi Littlewood Office: + 3 (0) Mobile: + 3 (0) kavi.littlewood@quintiles.com Further information Available from: Available from: Quintiles blog Emerging market strategies: four options for small and midsize pharma - Evangelos Tryfonidis FDA approves first biosimilar - Nigel Rulewski What should we be doing with electronic health record data? Karen Knecht How outcomes evaluations can increase NHS access to innovative drugs - Paul Sutton Available from: Copyright 0 Quintiles. All rights reserved