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  • 1. Towards Planning for Optimal Access to Effective Therapies for Rare and Ultra-Rare Conditions: A Scoping Study Mike Paulden, Tania Stafinski, Devidas Menon and Chistopher McCabe University of Alberta, Edmonton, AB
  • 2. Acknowledgements: CIHR Emerging Team Grants Funding Promoting Rare-disease Innovations through Sustainable Mechanisms (PRISM) Collaborators Network Rare and Ultra-Rare Conditions: A Scoping Study
  • 3. Rare and Ultra-Rare Conditions: A Scoping Study US Orphan Drugs Act (1983) Designed to facilitate development and commercialization of drugs to treat rare diseases (< 200,000 Americans) - 7 year market exclusivity for companies - Tax credits equal to half of the development costs - Grants for drug development - Fast-track approvals of drugs Orphan Drug Approvals 1973 to 1983: 10 products Orphan Drug Approvals 1983 to 2013: > 400 Background
  • 4. Rare and Ultra-Rare Conditions: A Scoping Study Personalised Medicine (PM) Common diseases sub-divided on molecular, proteomic or genetic characteristics Emergence of PM-defined orphan and ultra-orphan diseases and associated treatments - Kalydeco is a treatment for Cystic Fibrosis G551D mutation (affects 4% of CF population) which costs > $300k per year Background
  • 5. Rare and Ultra-Rare Conditions: A Scoping Study Health system budgets are increasingly stretched: Aging populations driving increasing demand Increasingly expensive therapies for common & rare diseases Global economic downturn reducing tax revenues Health systems are establishing formal value based reimbursement mechanisms CDR & pCODR, NICE, HAS, iQWIG Decisions criticised by patients, clinicians and manufacturers for implicit and explicit value frameworks used Background
  • 6. Rare and Ultra-Rare Conditions: A Scoping Study Cost of orphan drugs often > $50,000 per annum Mathematically exceeds conventional value threshold given neutral equity position Accentuates the focus on the value in value-based coverage decision making for orphan drugs Critical to establish the values underlying decision making Background
  • 7. Rare and Ultra-Rare Conditions: A Scoping Study To scope the landscape of proposed approaches for assessing the value of orphan and ultra orphan drugs in order to identify: Specific value propositions Decision process propositions Purpose of our research
  • 8. Rare and Ultra-Rare Conditions: A Scoping Study We conducted a scoping review Scoping reviews map out key concepts and the main sources of evidence in a complex area (Mays et al., 2001) Identifying knowledge gaps Potential research opportunities Methods
  • 9. Rare and Ultra-Rare Conditions: A Scoping Study Six steps of a scoping review: 1. Identifying the research question 2. Searching for relevant studies 3. Selecting studies 4. Charting or extracting the data 5. Tabulating and summarizing the results 6. Consulting with relevant experts on the results (Arksey and OMalley, 2005) Methods
  • 10. Rare and Ultra-Rare Conditions: A Scoping Study Research question was formulated with input from PRISM Investigators and Network: What is known about societal values for new therapies for rare and ultra-rare diseases and conditions? Societal values were defined as any statements regarding how health care resources should be prioritized to reflect public choices or social preferences Rare and ultra-rare diseases were defined as any conditions that had been self-described as such 1. Identifying the research question
  • 11. Rare and Ultra-Rare Conditions: A Scoping Study A comprehensive search strategy for identifying as many relevant published and unpublished papers Search parameters not limited to a particular study design For feasibility reasons, language/date restrictions applied (English language papers between Jan 1990 and Oct 2013) The search strategy was applied to the following databases: PubMed (MEDLINE and non-MEDLINE sources), EMBASE, Web of Science, Scopus, CINAHL, and EconLit 2. Searching for relevant studies
  • 12. Rare and Ultra-Rare Conditions: A Scoping Study Pre-specified inclusion and exclusion criteria were used to create a screening checklist, which was applied to discrete citations or abstracts by two independent researchers Any papers addressing both of the following were included: 1. A specific rare or ultra-rare disease, or either more broadly 2. Values or decision factors that should be taken into account during funding deliberations and treatment decision making Multi-country comparisons of access to, or utilization of, specific therapies or drug review processes were excluded 3. Selecting studies
  • 13. Rare and Ultra-Rare Conditions: A Scoping Study Records identified through database searching (n = 3,699) Additional records identified through other sources (n = 24) IdentificationScreeningEligibilityIncluded Records after duplicates removed (n = 3,030) Records screened (n = 3,030) Full-text articles assessed for eligibility (n = 401) Records excluded (n = 2,629) Studies included in synthesis (n = 43) Full-text articles excluded: opinion pieces, news articles, descriptive or utilization studies (n =358) Studies discussing potential value-bearing attributes (n=35) Studies discussing potential process-related factors/frameworks (n=8)
  • 14. Rare and Ultra-Rare Conditions: A Scoping Study Information independently charted by both researchers via a data charting form to identify key aspects or concepts Author(s) Type of paper Country where the paper originated Purpose of the paper Definition of rare or ultra-rare applied The type(s) of therapies addressed Decision factors or values-based statements considered Methods, approaches, and information sources used Findings and conclusions 4. Charting or extracting the data
  • 15. Rare and Ultra-Rare Conditions: A Scoping Study Information from completed forms was tabulated to assess the nature and distribution of papers comprising the review Content analysis of the tabulated information was conducted to identify themes: Decision factors Preferences Value propositions 5. Tabulating and summarizing the results
  • 16. Rare and Ultra-Rare Conditions: A Scoping Study A consultation exercise was carried out with key stakeholder communities: patients, providers, industry, and government through PRISM Network Network members reviewed the draft results and contributed references, factors or arguments that had not been captured Feedback received was incorporated into the draft results through a similar approach to that applied to the papers 6. Consulting relevant experts
  • 17. Rare and Ultra-Rare Conditions: A Scoping Study Prevalence (rarity) of disease (CoD) Severity (seriousness) of disease (CoD) Identifiability (CoD) Is the disease life-threatening or chronically debilitating? (CoD) Evidence of treatment efficacy/effectiveness (CoT) Magnitude of treatment benefit (CoT) Safety profile of treatment (CoT) Is treatment innovative? (CoT) Societal impact of treatment (CoT & CS-E) Impact of treatment upon distribution of health (CS-E, CoT, CoD) Price (cost) of treatment (OC) Budget impact of treatment (OC) Cost-effectiveness of treatment (Synthesis of Value & OC) Availability of alternatives (CoD) Feasibility of diagnosis (Necessary condition for meaningful problem) Feasibility of treatment (Necessary Condition for meaningful problem) Commercial considerations (CS-E) Legal considerations (CS-E) Results: Identified decision factors
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