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Recognizing the Importance of QoL Measurements of Oncology Trials — Executing / Informing: The Building of Your Strategic Plan Scott Megaffin March 23, 2016 Property of Churchill Pharmaceuticals, LLC – March 2016 9th Annual Oncology Commercialization & Market Access CBI Conference 1 Check us out: www.churchillpharma.com Property of Churchill Pharmaceuticals, LLC – March 2016 2 Presentation Objectives A well conducted QoL Deliverable will: 1. Meet evidence requirements for reimbursement and market access 2. Generate the HEOR evidence for labeling throughout product life cycle 3. Communicate full brand value to payers and clinicians Hint: This is a test Property of Churchill Pharmaceuticals, LLC – March 2016 3 Executive Summary • Background: o Define your commercialization opportunity (Big Data) o Understand the literature (The Oncology Maze) • Determine strategic and tactical objectives • Inform: 1. Health Economics 2. Outcomes Research • Execute: QoL Strategy with data / validation Property of Churchill Pharmaceuticals, LLC – March 2016 4 Key commercialization milestones • Year -2: Commercialization plan; know your literature; establish the QoL plan • Year -1: Phase III trial data; submit NDA • Launch year: Phase III trial data published - editorials, commentaries; opinion leader perspective • Launch +0/+1: Approval; brand as standard of care for indication QoL strategy supports and informs commercialization timeline Property of Churchill Pharmaceuticals, LLC – March 2016 5 Case example: Rigosertib (ONTX) • Second-line monotherapy for patients: – Relapsed after hypomethylating agents (HMAs) – Failed to respond to HMA – Intolerant to HMA • Uses novel mechanism of action in MDS • Delays progression to acute myeloid leukemia (AML) • Phase III ON 01910 Na. Trial In Myelodysplastic SyndromE (ONTIME) trial (rigosertib vs. supportive care) ongoing Property of Churchill Pharmaceuticals, LLC – March 2016 6 Case example: Understanding the data • Group of related cytopenias: – Can include anemia, dysplastic changes – Three risk classification schemes: French-American-British, World Health Organization (WHO), International Prognostic Scoring System (Bennett 1982, Vardiman 2002, Greenberg 2012) • Incidence difficult to quantify: – Estimates range from 20,000 to 80,000 new cases per year in US (Cogle 2011) – Accuracy of estimate highly dependent upon data source (e.g, cancer registry, claims data) • Median age at diagnosis < 60 years (Vardiman 2002) • Long term prognosis is poor: 3 to 6 months, depending upon risk Understanding data means understanding the impact of QoL Property of Churchill Pharmaceuticals, LLC – March 2016 7 Case example: Understanding disease treatment • Largely incurable (Nimer 2008) • Best supportive care (BSC) includes: – Blood transfusions – Iron chelation therapy, especially if transfusion-dependent – Anemia management (NCCN 2013) • After diagnosis, most patients receive BSC only, due to: – Advanced patient age – Toxicities of current medications (Greenberg 2010) • Antineoplastic treatments • Only curative therapy is hematopoietic stem cell transplantation; few are candidates Treatment options limited, QoL becomes mixed with quantity Property of Churchill Pharmaceuticals, LLC – March 2016 8 Case example: Defining - QoL, in population • Little may be known about quality of life (QoL) – Small studies cited fatigue, transfusion dependence and management of iron overload as factors contributing to low QoL (Steensma 2008, Thomas 2012, Payne 2008) • Value to patients of being “transfusion independent” is high • No studies specifically measured QoL in second-line MDS QoL for MDS was not well defined, no studies in 2nd Line MDS treatment Property of Churchill Pharmaceuticals, LLC – March 2016 9 Case example: Knowledge Gap - Cost of care • Cost of BSC and treatment $50,000 per year (Greenberg 2008, Wang 2012) • Few studies examined cost effectiveness of HMAs – Those that had suggested treating MDS as cost effective • Cost estimation hampered by same issues as incidence estimation (e.g., incomplete data, wide variation in estimates) • No studies specifically measure cost in second-line MDS No studies compared cost effectiveness for second-line MDS Property of Churchill Pharmaceuticals, LLC – March 2016 10 HEOR: Aligning the tactics Goal: Improve acceptance of YOUR COMPOUND in YOUR Malignancy treatment 1. Claims analysis to improve incidence estimates 2. Conceptual model to understand treatment decision-making 3. Claims analysis to characterize burden of illness 4. Cost effectiveness analysis of current second-line treatment 5. Validation - Patient survey for quality of life 6. Cost effectiveness analysis of treatment your compound 7. Decision model, either: progression – survival (?) Property of Churchill Pharmaceuticals, LLC – March 2016 11 Validated Tools Property of Churchill Pharmaceuticals, LLC – March 2016 12 Presentation Summary The successful QoL Deliverable will: • Meet evidence requirements for reimbursement and market access • Generate the HEOR evidence for labeling throughout product life cycle • Communicate full brand value to payers and clinicians Property of Churchill Pharmaceuticals, LLC – March 2016 13 Thank you . . . and Rock Chalk!! Property of Churchill Pharmaceuticals, LLC – March 2016 14 References Bennett JM, Catovsky D, Daniel MT, et al. Proposals for the classification of the myelodysplastic syndromes. Br J Haematol. 1982;51:189-199. Cogle CR, Craig BM, Rollison DE, List AF. Incidence of the myelodysplastic syndromes using a novel claims-based algorithm: high number of uncaptured cases by cancer registries. Blood. 2011;117:7121-7125. Greenberg PL, Tuechler H, Schanz J, et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012;120:2454-2465. Greenberg PL. Current therapeutic approaches for patients with myelodysplastic syndromes. Br J Haematol. 2010;150:131-143. Greenberg PL, Cosler LE, Ferro SA, Lyman GH. The costs of drugs used to treat myelodysplastic syndromes following National Comprehensive Cancer Network Guidelines. J Natl Compr Canc Netw. 2008;6:942-953. National Comprehensive Cancer Network (NCCN). NCCN clinical practice guidelines in oncology: myelodysplastic syndromes, version 2.2013. http://www.nccn.org/professionals/physician_gls/pdf/mds.pdf. Accessed February 11, 2013. Nimer SD. Myelodysplastic syndromes. Blood. 2008;111:4841-4851. Payne KA, Rofail D, Baladi JF, et al. Iron chelation therapy: clinical effectiveness, economic burden and quality of life in patients with iron overload. Adv Ther. 2008;25:725-742. Prébet T, Gore SD, Esterni B, et al. Outcome of high-risk myelodysplastic syndrome after azacitidine treatment failure. J Clin Oncol. 2011;29:3322-3327. Steensma DP, Heptinstall KV, Johnson VM, et al. Common troublesome symptoms and their impact on quality of life in patients with myelodysplastic syndromes (MDS): results of a large internet-based survey. Leuk Res. 2008;32:691-698. Thomas ML, Crisp N, Campbell K. The importance of quality of life for patients living with myelodysplastic syndromes. Clin J Oncol Nurs. 2012;16 (Suppl):47-57. Vardiman JW, Harris NL, Brunning RD. The World Health Organization (WHO) classification of the myeloid neoplasms. Blood. 2002;100:2292-2302. Wang R, Gross CP, Frick K, et al. The impact of hypomethylating agents on the cost of care and survival of elderly patients with myelodysplastic syndromes. Leuk Res. 2012;36:1370-1375. Property of Churchill Pharmaceuticals, LLC – March 2016 15