Oncology Special Interest Group

Transcription

1 Oncology Special Interest Group Open Meeting Tuesday, 10 November 2015 ISPOR 18 th Annual European Congress MiCo Milano Congressi, Milan, Italy Compounds in Development for Top 10 Therapeutic Areas a Number of compounds in clinical trials or under review by the US Food and Drug Administration. This includes a total of compounds in b Includes all nonimmunological anticancer compounds. c Rare diseases were defined as those affecting or fewer people in the United States. Source: Moses et al. The Anatomy of Medical Research: US and International Comparisons. JAMA. 2015;313(2):

2 Challenges in Oncology Various unaddressed challenges increasing in importance Multiple lines of therapies and treatment sequences Personalized medicine The Precision Medicines Initiative in the US with $215 million for research efforts to accelerate biomedical discoveries Value for money? Not cost-effective at zero price? Discussions at NICE Cancer Drugs Fund ASCO Value Framework, ESMO Magnitude of Clinical Benefit Scale (MCBS) Patient perspective Patient Advisory Committee at the European CanCer Organisation (ECCO) NHS England and Macmillan Cancer Support: Living With and Beyond Cancer Programme following the National Cancer Survivorship Initiative Patient organisations included in various initiatives (e.g. EU Innovative Medicines Initiative GETREAL program) 3 New Challenges in Oncology Adaptive licensing/pathway EMA pilot project initiated in 2014 NEW Drug Development ParadIGmS (NEWDIGS) program (MIT, EFPIA, EMA, MHRA, NORD, NICE, etc.) Centre for the Advancement of Sustainable Medical Innovation (CASMI) with academia and HTA agencies in the UK Limitations of trial data leading to increased interest in real life data EU: Innovative Medicines Initiative GETREAL program (EFPIA, EMA, universities, patient organisations) ASCO Teams With Multinational Software Corporation, SAP, to Develop CancerLinQ to unlock real-world patient care data from millions of electronic health records Immunotherapies ASCO Advance of the year in cancer treatment are 2 immunotherapies and 2 targeted therapies 4 2

3 Oncology Economic Modeling Working Group Oncology Special Interest Group resumed Goal: To identify and address specific oncology outcomes research issues and to develop recommendations to address these issues. One such issue is health economic modelling New developments and changes in medicine challenge current methodology and usual practice in modeling oncology treatments Formation of the Oncology Economic Modeling Working Group under the existing Oncology SIG to : Discuss, collate, and develop best practices in modelling health technologies in oncology Provide a forum for cross-organization collaboration and discussion 5 Aims of the Working Group Advance knowledge and understanding around oncology economic modeling Provide education and resources for researchers interested in the health economic modeling of oncology products Stimulate debate and encourage research Develop best practices in selected aspects of economic modeling, specific to oncology Foster discussions among researchers working in the field in different organisations Foster communication and collaboration between health economists and medical organisations and patient representatives (ISPOR Patient Roundtable) 6 3

4 Oncology Economic Modeling Working Group Noémi Muszbek, MA, MSc, Senior Research Scientist, Modeling and Simulation, Evidera, London, UK Sorrel Wolowacz, PhD, Head, European Health Economics, RTI Health Solutions, Manchester, UK Christopher Graham, Director, Health Economics, RTI Health Solutions, Research Triangle Park, NC, USA Ágnes Benedict, MSc, MA, Executive Director, Center of Excellence, Health Economics, Evidera, Budapest, Hungary 7 Selection of Working Group Topics Topics that present challenges in oncology, require extensive discussions, further research, and would benefit from collaboration with medical and patient organisations Workshop at ISPOR 20th Annual International Meeting, Philadelphia: Modeling in Oncology: The Taming of the Shrews? Presented a broad list of identified topics in oncology modelling Based on our experience, topics at previous ISPOR/ESMO/ECCO Survey conducted to identify most important items to modelling community e.g. How important is it to discuss the impact of personalized medicine on modeling in oncology? Categories: high priority, medium priority, low priority, not relevant 8 4

5 Potential Topics Introduction of new treatment types/modalities (e.g. Molecularly targeted therapies, immunotherapies) Personalized medicine Patient perspective Multiple lines of treatment and complex treatment sequences Small incremental benefit Fast changing treatment algorithms in some cases that render trial comparators irrelevant Off-label drug use Pressure on health care budgets with emphasis on oncology drug costs Focus on biologic pathways Modelling effectiveness of subsequent treatments Caregivers costs and utilities Difficulties in measuring costs and utilities at the end of life Pressure on early access Modelling changes in quality of life, disease management, and treatment pattern post- progression Measuring the consequences of toxicity on QoL and costs Alternative model structures Oncology landscape changes on establishing comparative effectiveness Association between PFS and OS and the surrogacy of PFS Extrapolation of survival endpoints and treatment effects Confounding effect of cross-over design in trials Decision on utility instruments (oncology specific or generic) Increasing role of oncology specialist services in determining access 9 Most Important Issues According to Percentage of 'High Priority' Answers 10 5

6 Most Important Issues According to Total Score of Answers Minimum: 0 Maximum: Working Group Topics Discussion topic: Modeling changes in quality of life, disease management and treatment pattern after disease progression in oncology Selected to have high priority Fulfills all the criteria Presents challenges in oncology Requires extensive discussions, further research Would benefit from collaboration between health economists and medical and patient organisations Knowledge resource topic: Survival extrapolation Had the highest proportion of High priority answers Despite the articles, guidelines published in the last years, practice varies and is still an area of active debate and methodological development 12 6

7 Discussion Topic: Background Modelling changes in quality of life, disease management and treatment pattern after disease progression in oncology In the classic 3 health state oncology Markov model, post-progression QoL and costs assumed constant over time Various accepted assumptions on how costs and QoL change throughout disease progression E.g. dependent on progression status, linear change over time Data usually not collected in trials; alternative sources challenging New approaches (e.g. disease specific utilities) Literature is limited, often same publication used in various models Costs: Only few recent published studies on BSC specifically (Lester 2013, lung cancer; Henk 2014, mrcc; Remak 2003, BC) Utilities: Review of technology appraisals at NICE: utility values predominantly came from previous studies (80.7%) (Muszbek et al. 2014) 13 Discussion Topic: Aims and Output Aims Demonstrate the paucity and importance of accurate information about HRQoL, treatment patterns, and costs after disease progression in the economic evaluation of cancer treatments Assess the limitations and challenges of data collection using standard sources and methods Provide recommendations for future research Collaborate with multiple stakeholders to inform and complement recommendations for future research Output Manuscript developed in collaboration with medical organisations and patient representatives 14 7

8 Discussion Topic: Process Sign-up with Theresa Tesoro or via the website Series of discussions through webmeetings with leadership team, and discussions Background work (e.g. targeted literature review) Based on background work, discuss: Available information Importance of accurate information Limitations and challenges in collecting accurate information Recommended approaches, research topics Use of case studies Present findings and result of discussions at ISPOR Oncology SIG Open Meeting On ISPOR SIG webpage, list of articles, links to ISPOR presentations, summary Manuscript (e.g. to Value in Heath) 15 Knowledge Resource Topic: Background and Aim Extrapolation of Survival Curves Background In recent years various guidelines, reviews, articles published on extrapolation Hoyle 2011, Guyot 2012, Latimer 2013, NICE DSU Technical Support document 2013, Ishak 2013, Grieve 2013 Aim and output To have a top-level, user friendly ISPOR resource that: Provides an overall summary of key terms and concepts Guides researchers to key publications Similar to current Oncology Outcomes Research Resources (OORR) Drop-down menus of main terms with short explanation and references 16 8

9 Knowledge Resource Topic: Process The members can decide on a list of key terms Everyone can volunteer to write a paragraph about one or more terms, or can volunteer to review Keep it very top level, not go into discussions Collating reviewed definitions and reference list Sent to review team Update Definition of terms do not change much in time Significant references When new methodology is published or guidance is released 17 Plans for Future Topic Selection and Development of ISPOR Website Resource Potentially a new topic every year One discussion Revising current resource topic or selection of new one 18 9

10 Questions and Comments? 19 Thank You for Your Attention! 20 10