Summary of Themes ALZHEIMER S DISEASE WORKING GROUP

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1 Summary of Themes 26 JUNE 2012 Alzheimer s Disease Working Group launch meeting: setting the agenda for change On June 2012, the Alzheimer s Disease Working Group (ADWG) convened for its first meeting in Paris. Please see the Appendix for a list of meeting participants. Participants agreed that the intent of this group is to address critical issues for healthcare systems raised by the rising tide of Alzheimer s disease (AD) and the impact of potential disease-modifying therapies. The group presented and discussed key challenges in improving outcomes in AD, focusing on early diagnosis, developing a standard of care and demonstrating the value of new AD approaches. At the conclusion of the meeting, participants expressed and prioritised opportunities to address challenges in assessing value in AD diagnostics and treatments as well as barriers to preparing a health system for the introduction of disease-modifying therapies in AD. Summary of themes In the face of limited resources, healthcare systems must make specific choices as to the AD populations to be treated and define adequate stopping rules for treatment. Participants acknowledged the significant impact of increasing numbers of AD patients on their countries healthcare systems and the burden it will impose on the available resources for treatment. However, there was little agreement on how therapeutic resources should be prioritised. All agreed that health systems should determine which patients would benefit most from treatments. Participants discussed how patients should be tracked for safety risks, disease progression or stabilisation and at what point treatment should be stopped. They noted that diagnostic tools could play an important role in these decisions. In addition, the group reviewed different healthcare system approaches for addressing AD and chronic diseases, and observed that current incentives for providers and patients may not be aligned to make the most clinically and economically effective choices for society. Consistent criteria are necessary to identify which patients should be offered diagnostic tests and to ensure that tests are reliable and accurate with regard to classifying patients disease severity. The group discussed potential approaches to identifying AD in different patient populations. Participants agreed that a healthcare system should not provide general population screening, but instead should offer diagnostic testing, particularly if there are risk factors present and a patient wants to know whether or not he or she has AD. Some participants specified that if a diagnostic test result would not alter a patient s clinical course, a diagnostic test should not be offered. However, other participants opined that diagnostic testing is still important to ensure that patients and their carers are able to plan appropriately for a future with AD. Even if a disease-modifying therapy were available, participants noted that access to a diagnostic test should depend on the patient s risk factors, including family history, age and lifestyle. Other considerations include the stage of the disease and clinical- and cost-effectiveness of the therapy. Further, the tests themselves ideally should have high positive and negative predictive value. To ensure that tests are developed appropriately, participants suggested that they first be used in specialised centres for AD patients before being used in the general medical community, although this may limit access for some patients. To facilitate this gradual rollout of diagnostic and treatment services, participants suggested establishing education programmes for physicians and other providers. PORTLAND HOUSE, FLOOR 16, STAG PLACE TEL: +44 (0) LONDON SW1E 5RS UNITED KINGDOM efax: +44 (0)

2 A presentation explained potential benefits of early diagnosis and described recent improvements in diagnostic tools, with new criteria recommending application of clinical and biomarker evidence for a diagnosis. Participants outlined different patient cases with varying ages, levels of frailty, comorbidities, risk factors and diagnostic indicators, and debated whether diagnosis should be offered or communicated. Experts commented that the AD community required more exact definitions of the different stages of AD from mild cognitive impairment to prodromal AD to AD dementia for proper diagnosis and patient and general provider education. Participants pointed out that such patient heterogeneity combined with physicians current inconsistent diagnostic labelling created challenges in interpreting clinical trial data when studies use different labels for patients with AD. Regardless of how diagnostics are used in the future, an infrastructure will be required for the diagnostic process and patient autonomy should be respected. To ascribe appropriate value to AD treatments and services, biomarkers and more pragmatic endpoints should be used to predict functional outcomes. Short-term costs and benefits should be weighed with long-term consequences. Participants discussed meaningful patient outcomes and surrogate endpoints for evaluation of AD treatments, within and post clinical trial period. They deliberated methods for integrating patient-reported outcomes and quality of life (QoL) into AD treatment studies. The group agreed that delaying institutionalisation and improvement or stabilisation of cognition were important outcomes for assessment. However they noted that these benefits may accrue only if treatment is given early in the disease course and may not show significance for years. They discussed the difficulty in assessing QoL given AD patients may not be able to evaluate their own stabilisation or improvement. Carers may more reliably assess changes in behaviour and anxiety levels. Participants also noted that QoL and other functional outcomes may not be reliable to show effectiveness of treatments on an individual basis in AD because of the variable course of the disease. Although it may be possible to determine effectiveness from population data, the body of knowledge around progression of AD is not as well-developed or as predictable as it is in other fields, for example, in cardiovascular disease. Payers acknowledged the need for innovative business models and agreements for developers particularly given the long-term investments required by developers to generate this body of knowledge for AD. An expert stated that biomarkers may serve as surrogate endpoints but noted that some may be more appropriate for predicting disease progression while others are more appropriate as diagnostic tools. Consumption of medical and other resources may provide another useful endpoint for evaluating a treatment. Another participant added that consensus models should be developed to demonstrate how these surrogate endpoints may predict long-term clinical and economic outcomes. However, payers cautioned that short-term costs accrued during mild impairment may not be suitable inputs for modelling the long-term costs that result from greater disease severity. From a payer s perspective, cognitive stabilisation or improvement is not valuable unless it is associated with a functional stabilisation or ideally improvement. Because of the long course of AD, demonstration of functional benefit in early stage AD patients may require post-market data and concomitant agreements for market entry that reflect that uncertainty of the treatment value to the Alzheimer s Disease Working Group launch meeting: setting the agenda for change 2

3 payers. Adding complexity to the topic of demonstrating value, a presentation shared a model that showed overall costs to social and health systems may increase with the availability of a diseasemodifying therapy. The model did highlight a significant shift of costs from long-term care to shortterm direct healthcare as patients perform at higher functional levels for longer time periods. This higher functioning in patients lessens the burden on carers, thereby decreasing indirect costs of AD. AD care should be integrated across medical and social services, and various interventions should be assessed for efficacy. Financing mechanisms for such care need to be rethought. Participants noted the value of integrated care for AD patients. Such care would include pharmacological and non-pharmacological therapies, lifestyle interventions and potentially patient navigators to guide patients and carers through the healthcare system to receive the appropriate resources. Social care experts discussed the importance of evaluating different aspects of AD patient care through randomised controlled trials and debated whether placebo arms should be included in trials. Several participants noted the need to rethink financing for chronic disease states such as AD, e.g. increasing co-pays, as current systems do not adequately fund the increasing need for patient care. Participants placed priority on issues related to early diagnosis and valuation of AD treatments. Participants expressed high ambitions to make progress on some of these challenges going forward and identified the following areas for focus of the Working Group. Early diagnosis Developing a clinical hierarchical decision tree for identifying the right patients at the right time for the right treatments Creating criteria for diagnostics test use and validation Establishing a framework for the introduction of a disease-modifying therapy Valuation of AD treatments Identifying endpoints and outcomes that are most relevant for study in early-stage AD, and understanding linkages amongst them Developing new business models or innovative agreements to help guide the introduction of new medicines Understanding how can we prepare health systems for disease-modifying agents, including infrastructure for diagnostics and coordination of care The meeting closed with reflections from participants on the collective next steps of the ADWG. Tapestry working together with the group will clarify and refine these priority areas, define principles and approaches to guide recommendations, and continue to coordinate with other ongoing AD initiatives to ensure our outcomes remain complementary. The Tapestry team will distribute a more detailed summary of the meeting and the path forward for the ADWG in the post-meeting summary report to be distributed in July. Alzheimer s Disease Working Group launch meeting: setting the agenda for change 3

4 Appendix: Alzheimer s Disease Working Group Participants Health Technology Assessors Payers Regulators Advisers Antje Behring and Wiebke Klipper* G-BA (Germany) (Observer) Jeni Bremner* European Health Management Association (EHMA) Karl Broich* EMA / BfArM (Germany) Anna Bucsics* Main Association of Austrian Social Security Institutions (Austria) Wing Cheng / Fredrik Nilsson* TLV (Sweden) Bo Claesson SKL (Sweden) Luisa Muscolo AIFA (Italy) Susan Morgan MHRA (Medicines & Healthcare Products Regulatory Agency) (UK) Jan van Emelen MLOZ (Mutualites Libres Onafhankelijke Ziekenfondsen) (Belgium) Julius van Dam formerly Menzis (The Netherlands) Subject matter experts Ron Akehurst* Sheffield University (UK) Sube Banerjee King s College London, National Dementia Strategy (UK) Kaj Blennow Gothenburg University (Sweden) Jeffrey Cummings* Cleveland Clinic (US ) Bruno Dubois Hôpital Pitié Salpêtrière (France) José Luis Molinuevo Barcelona Clinical Hospital Pasqual Maragall Foundation (Spain) Louise Robinson* National Primary Care Lead; DeNDRoN Network (UK) Anders Wimo Karolinska Institutet Sweden Patient and policy advocates Clive Ballard King s College London, Alzheimer s Society (UK) Andrew Chidgey* Alzheimer s Society (UK) Marc Wortmann Alzheimer's Disease International *Participant was unable to attend 14 June 2012 launch meeting. Continued overleaf Alzheimer s Disease Working Group launch meeting: setting the agenda for change 4

5 Sponsor representatives from Bristol Meyer Squibb (BMS) Andrew Friedman* Executive Director Neuroscience - Global Regulatory Strategy Gil L Italien Executive Director Global Health Outcomes Research Jane Tiller Vice President Neuroscience & Global Development Lead - Alzheimer's Disease Sponsor representatives from General Electric (GE) François Nicolas Director PET Neurology Steve Sandor Market Access Leader PET Neurology Sponsor representatives from Johnson & Johnson (J&J) Chris Leibman Senior Director Head Market Access/Health Economics Luc van Oevelen* European New Product Leader Alzheimer s Steve Wooding Head Market Access EMEA *Participant was unable to attend 14 June 2012 launch meeting. The views expressed in this document represent those of the Alzheimer s Disease Working Group, convened by the European Healthcare Innovation Leadership Network, a group of leading stakeholders from the public and private sectors committed to improving healthcare and economic well-being in the European Union and its Member States. This document is not intended to represent the particular policies or positions of the Network s individual participants or their affiliated organisations. This material is prepared by and copyrighted by Tapestry Networks. It may be reproduced and redistributed, but only in its entirety, including all copyright and trademark legends. Alzheimer s Disease Working Group launch meeting: setting the agenda for change 5