Volume 24, Issue 1 SPRING/SUMMER 2018 newsletter of the myelodysplastic syndromes foundation MDS NEWS HIGHLIGHTS FROM THE GUEST EDITOR’S DESK I MDS: Regulatory Aspect, Cost Effectiveness and Costs of Care Co-authors: David T. Bowen, MD (UK), Trever Burgon, PhD (USA) ADVANCING RESEARCH & PATIENT CARE SÃO PAULO 2018 PLAN TO ATTEND 2018 LATIN AMERICAN MDS FOUNDATION SYMPOSIUM LATIN AMERICAN MDS FOUNDATION October 31, 2018 • São Paulo, Brazil SYMPOSIUM São Paulo, Brazil October 31st 2018 IN THIS ISSUE TABLE OF CONTENTS FROM THE GUEST EDITOR’S DESK 2 PATIENTS AND CAREGIVERS LIVING WITH MDS FORUMS MEETING HIGHLIGHTS & ANNOUNCEMENTS 29 MDSF MEMBERSHIP ASH 2018: MDS Breakfast Symposium 5 30 IN THE NEWS PATIENT SUMMARY: 2017 ASH Symposium 6 35 2018 Latin American MDS Foundation AML CORNER 37 Symposium, Brazil 11 OUR PATIENT STORIES RESEARCH 41 OUR CAREGIVER STORIES MDS/MPN International Working Group 12 50 International Working Group for 12 CONTRIBUTIONS TO THE FOUNDATION Prognosis in MDS Gifts 54 INTERNATIONAL NURSE LEADERSHIP BOARD 14 Memorial Donations 55 MDS RESOURCES: LITERATURE HIGHLIGHTS 16 Living Endowments 55 MDS CENTERS OF EXCELLENCE 21 15TH INTERNATIONAL SYMPOSIUM ON MDS FROM THE FOUNDATION 26 60 www.mds-foundation.org FROM THE GUEST EDITOR’S DESK GUESTXXXXX EDITORIAL Guest Editorial – Regulatory Aspect, Cost Effectiveness and Costs of Care Where We Have Come From, What interests the authors Where We Are is how healthcare systems European Perspective (with focus on UK) could assess the ‘value’ In the UK, there is a semi-transparent of a given treatment, and process for cost effectiveness analysis, with only the details of the company consequently how to use Patient Access Scheme (typically a simple David T. Bowen, MD discounted price) always marked Consultant Haematologist that assessment to decide ‘commercial in confidence’. An objective Leeds Teaching Hospitals analysis of clinical efficacy, survival United Kingdom whether the intervention benefit and quality of life benefit creates an Trever Burgon, PhD (drug) is affordable Incremental Cost Effectiveness Ratio Vice President (ICER) and a cost per Quality-Adjusted QURE Healthcare within the given Life Year (QALY) which must be below San Francisco, California, USA agreed thresholds for affordability by the healthcare system. NHS to permit market access into the NHS. The licensing and market entry of active Because the NHS has a fixed annual drugs to treat MDS patients (azacitidine, The focus of our Editorial will be the budget, any new expenditure within this decitabine, lenalidomide) during the 2000s cost of care for MDS and the analyses and budget inevitably displaces other NHS represented important progress in processes that inform and optimise market activity. Other countries also conduct CEA improving outcomes for those patients who access. In Europe these analyses, and an example of variation from country achieve a clinically meaningful response. collectively called health technology to country for CEA analyses, in this case These agents have different licensed assessment (HTA), are typically for azacitidine, can be seen in the Table. indications and price points between the comparative effectiveness (CE) or cost Countries that have no formal process US and European countries. These effectiveness analysis (CEA). The US has a for cost effectiveness analysis will differences are good examples of the much different process wherein the FDA negotiate price directly with the company different ways the US Federal Drug first decides whether a new drug can be based either on a formal comparative Administration (FDA) and the European marketed and then the federal health effectiveness analysis and a framework for Medicines Agency (EMA) interpret insurance program, Medicare, decides the pricing negotiation that follows this efficacy and safety data, and the whether it will pay for the drug, with analysis (e.g. France/Germany), or if no consequent benefit:risk ratio assessment Medicare ultimately covering most drugs CEA process operates in that country, that determines final market authorisation approved by the FDA. In stark contrast to based on other discount models, some of and indication. European countries, Medicare is forbidden which involve comprehensive registry data MDS is an expensive condition to by law to directly negotiate price with drug collection as a mandatory commitment, manage. Average 5-year per patient makers, although private payers can do so. such as Italy. treatment costs for MDS are higher than What interests the authors is how For many years, NICE has been any of the 18 most prevalent cancers in the healthcare systems could assess the ‘value’ considered the ‘bad guy’, reviewing new US.1,2 These costs are driven not only by of a given treatment, and consequently how drugs too slowly and then declining market the expensive drugs mentioned above, but to use that assessment to decide whether access because the drugs are not also by costly supportive care (e.g., the intervention (drug) is affordable within considered a cost effective use of NHS transfusions, growth factors, iron the given healthcare system. We present resources by the defined, and still widely chelators) and high use for other health perspectives from the European approved, criteria. NICE has recently care services (e.g., inpatient admissions (predominantly UK) and from the US created a CEA process contemporaneous and emergency department visits). healthcare systems. with EMA’s market authorisation delib- 2 GUEST EDITORIAL Cost per QALY Managed access in model Analysis NICE (UK)3 €55,945 Yes; simple discount Weighted average for CCR based on ‘real world’ data for BSC vs LDAC vs IC Spain4 €34,673 No Cost effectiveness Canada5 €58,074 No ‘Cost utility’ eration. There is now also an emerging greater initial scrutiny regarding cost- coordination. This program includes public understanding that much of the effectiveness. MDS patients and seeks to incentivize responsibility for facilitating market access Interestingly, unwarranted clinical providers and health systems to deliver in the UK (and elsewhere) rests with variation in MDS care, more so than the the most cost-effective care, contingent realistic pricing from the pharmaceutical price of any specific drug, may be one of on achieving specific quality measures. industry. One example is a small, recently the biggest drivers of high costs. In the US, This can certainly include drug selection, established pressure group, JustTreatment, 2-year MDS related costs vary but also efforts to reduce complications that claims some success in lobbying Pfizer significantly by geographic region, from and costly hospitalizations. to reduce the cost to the UK NHS for $40,793 per patient in New Mexico to Initiatives aimed at measuring ‘value’ palbociclib as treatment of adjuvant breast $78,156 in Detroit, Michigan.6 Even are far from perfect, but these value cancer therapy, such that NICE was then though some areas spent nearly twice as frameworks are increasingly used in able to approve market access. much on care, this spending was not oncology. One such framework is a correlated with different patient (somewhat arbitrary) points-based system US Perspective populations or better survival. There is a combining efficacy and toxicity Much ink has been spilt by others huge opportunity to better understand what assessment with simple cost, exemplified detailing the unique challenges and drives value and cost-effectiveness in MDS by the ESMO and ASCO models. Value opportunities in the US market, where across the continuum of care for the frameworks have strikingly been used to comparative effectiveness and cost disease. Published cost-effectiveness call into question the true value of many of effectiveness analyses play a less research in MDS remains low. A search of the oncology drugs licensed by FDA influential role in establishing the price and the Pubmed database reveals nearly 4,000 between 2000–20157, and will likely accessibility of new drugs. Because of the journal articles published in 2016 on ‘cost- intensify scrutiny on new drugs coming lower cost-effectiveness bar, more effectiveness’, but only 15 papers that to market. therapies are generally available to US included MDS in the analysis. Where We Are Going? patients more rapidly, but usually at a much This cost variation data suggests that higher cost. there may be a significant opportunity to The future is bright in oncology therapy, However, the US marketplace is increase the overall value and cost- but not without costs. A tranche of gradually taking a harder look at the cost- effectiveness of care by standardizing remarkably effective oncology therapies is effectiveness and ultimate value of health care around the best available evidence- now reaching the market, but these care good and services in oncology. For based practices, and integrating new therapies continue to test the upper example, Medicare now publishes a therapies into those treatment pathways thresholds for cost around the world. dashboard highlighting the drugs that as they become available. In the US, both Comparative/cost-effectiveness analyses account for the highest total Medicare Medicare and commercial insurers are in oncology can be challenging for a spending and per user spending. launching new programs to encourage number of reasons, including: Lenalidomide is featured prominently in and reward more efficient, standardized 1. Early licensing of promising new the top 10 of both spending areas (7th in care. Medicare,