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Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)
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Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL) (2017)Recommendations from the European Working…American Society of Clinical Oncology Statement: A Conceptual Framework to Assess the Value of Cancer Treatment Options (2015)American Society of Clinical …Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries (2015)Access to Orphan Drugs: A Com…A standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) (2015)A standardised, generic, vali…A Systematic Review of Stated Preference Studies Reporting Public Preferences for Healthcare Priority Setting (2014)A Systematic Review of Stated…Reconciling uncertainty of costs and outcomes with the need for access to orphan medicinal products: a comparative study of managed entry agreements across seven European countries (2013)Reconciling uncertainty of co…Managed entry agreements for pharmaceuticals: the European experience (2013)Managed entry agreements for …SOCIETAL VIEWS ON NICE, CANCER DRUGS FUND AND VALUE‐BASED PRICING CRITERIA FOR PRIORITISING MEDICINES: A CROSS‐SECTIONAL SURVEY OF 4118 ADULTS IN GREAT BRITAIN (2012)SOCIETAL VIEWS ON NICE, CANCE…Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments? (2012)Paying for the Orphan Drug Sy…Pricing and reimbursement of orphan drugs: the need for more transparency (2011)Pricing and reimbursement of …Estimating the budget impact of orphan medicines in Europe: 2010 - 2020 (2011)Estimating the budget impact …Characteristics of Clinical Trials to Support Approval of Orphan vs Nonorphan Drugs for Cancer (2011)Characteristics of Clinical T…Orphan drug: Development trends and strategies (2010)Orphan drug: Development tren…A comparative study of European rare disease and orphan drug markets (2010)A comparative study of Europe…Why rare diseases are an important medical and social issue (2008)Why rare diseases are an impo…Assessing the economic challenges posed by orphan drugs (2007)Assessing the economic challe…Drugs for exceptionally rare diseases: do they deserve special status for funding? (2005)Drugs for exceptionally rare …How to Value Orphan Drugs? A Review of European Value Assessment Frameworks (2021)How to Value Orphan Drugs? A …Implementing reflective multicriteria decision analysis (MCDA) to assess orphan drugs value in the Catalan Health Service (CatSalut) (2019)Implementing reflective multi…Comparing access to orphan medicinal products in Europe (2019)Comparing access to orphan me…The European challenges of funding orphan medicinal products (2018)The European challenges of fu…Rare diseases under different levels of economic analysis: current activities, challenges and perspectives (2018)Rare diseases under different…Multi-Criteria Decision Analysis (MCDA) Models in Health Technology Assessment of Orphan Drugs—a Systematic Literature Review. Next Steps in Methodology Development? (2018)Multi-Criteria Decision Analy…Economic Modeling Considerations for Rare Diseases (2018)Economic Modeling Considerati…Design and use of questionnaires: a review of best practice applicable to surveys of health service staff and patients (2001)QALY maximisation and people's preferences: a methodological review of the literature (2004)Response Rates and Nonresponse Errors in Surveys (2012)Evidence-informed frameworks for cost-effective cancer care and prevention in low, middle, and high-income countries (2014)Attributes and weights in health care priority setting: A systematic review of what counts and to what extent (2015)Emerging Use of Early Health Technology Assessment in Medical Product Development: A Scoping Review of the Literature (2017)Priority setting of health interventions: the need for multi-criteria decision analysis. 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(2017)Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group (2018)Cost-Effectiveness Analysis and the Consistency of Decision Making (2001)National Institute for Clinical Excellence and its value judgments (2004)Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy (2023)Randomized, Controlled Trials, Observational Studies, and the Hierarchy of Research Designs (2000)GRADE guidelines: 3. Rating the quality of evidence (2011)Economic burden of cancer across the European Union: a population-based cost analysis (2013)Updating the American Society of Clinical Oncology Value Framework: Revisions and Reflections in Response to Comments Received (2016)Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13 (2017)The high price of anticancer drugs: origins, implications, barriers, solutions (2017)Net Health Benefits (1998)Projections of the Cost of Cancer Care in the United States: 2010-2020 (2011)The Financial Toxicity of Cancer Treatment: A Pilot Study Assessing Out-of-Pocket Expenses and the Insured Cancer Patient's Experience (2013)Essential medicines for universal health coverage (2016)Financial Hardships Experienced by Cancer Survivors: A Systematic Review (2016)Does NICE have a cost‐effectiveness threshold and what other factors influence its decisions? A binary choice analysis (2004)Preferred reporting items for systematic reviews and meta-analyses: the PRISMA statement (2009)Preferred reporting items for systematic reviews and meta-analyses: the PRISMA Statement. (2009)Socio-economic burden of rare diseases: A systematic review of cost of illness evidence (2014)Overcoming Challenges Facing Advanced Therapies in the EU Market (2016)Orphan drug development: an economically viable strategy for biopharma R&D (2012)The Roles Of Academia, Rare Diseases, And Repurposing In The Development Of The Most Transformative Drugs (2015)Trends in utilization of FDA expedited drug development and approval programs, 1987-2014: cohort study (2015)Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years (2010)The supportive care needs of parents with a child with a rare disease: results of an online survey (2016)Alone in a Crowd? Parents of Children with Rare Diseases’ Experiences of Navigating the Healthcare System (2018)
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References

  • 2015American Society of Clinical Oncology Statement: A Conceptual Framework to Assess the Value of Cancer Treatment Options916 cited
  • 2015A standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS)817 cited
  • 2008Why rare diseases are an important medical and social issue621 cited
  • 2007Assessing the economic challenges posed by orphan drugs279 cited
  • 2015Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries278 cited
  • 2011Pricing and reimbursement of orphan drugs: the need for more transparency261 cited
  • 2011Characteristics of Clinical Trials to Support Approval of Orphan vs Nonorphan Drugs for Cancer230 cited
  • 2005Drugs for exceptionally rare diseases: do they deserve special status for funding?220 cited
  • 2012SOCIETAL VIEWS ON NICE, CANCER DRUGS FUND AND VALUE‐BASED PRICING CRITERIA FOR PRIORITISING MEDICINES: A CROSS‐SECTIONAL SURVEY OF 4118 ADULTS IN GREAT BRITAIN208 cited
  • 2012Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?171 cited
  • 2013Managed entry agreements for pharmaceuticals: the European experience167 cited
  • 2010Orphan drug: Development trends and strategies137 cited
  • 2011Estimating the budget impact of orphan medicines in Europe: 2010 - 2020

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133 cited
  • 2013Reconciling uncertainty of costs and outcomes with the need for access to orphan medicinal products: a comparative study of managed entry agreements across seven European countries122 cited
  • 2014A Systematic Review of Stated Preference Studies Reporting Public Preferences for Healthcare Priority Setting121 cited
  • 2010A comparative study of European rare disease and orphan drug markets121 cited
  • Cited by

    • 2019Comparing access to orphan medicinal products in Europe88 cited
    • 2018Multi-Criteria Decision Analysis (MCDA) Models in Health Technology Assessment of Orphan Drugs—a Systematic Literature Review. Next Steps in Methodology Development?76 cited
    • 2019Implementing reflective multicriteria decision analysis (MCDA) to assess orphan drugs value in the Catalan Health Service (CatSalut)65 cited
    • 2018The European challenges of funding orphan medicinal products64 cited
    • 2018Economic Modeling Considerations for Rare Diseases63 cited
    • 2021How to Value Orphan Drugs? A Review of European Value Assessment Frameworks62 cited
    • 2018Rare diseases under different levels of economic analysis: current activities, challenges and perspectives58 cited