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Access to orphan drugs despite poor quality of clinical evidence
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Access to orphan drugs despite poor quality of clinical evidence (2010)Access to orphan drugs despite poor quali…Relative efficacy of drugs: an emerging issue between regulatory agencies and third-party payers (2010)Relative efficacy of drugs: a…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…Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products (2006)Are rare diseases still orpha…A journey of hope: lessons learned from studies on rare diseases and orphan drugs (2006)A journey of hope: lessons le…Orphan drugs and the NHS: should we value rarity? (2005)Orphan drugs and the NHS: sho…Drugs for exceptionally rare diseases: do they deserve special status for funding? (2005)Drugs for exceptionally rare …Two decades of orphan product development (2002)Two decades of orphan product…The results of direct and indirect treatment comparisons in meta-analysis of randomized controlled trials (1997)Combination of direct and indirect evidence in mixed treatment comparisons (2004)Strengthening the reporting of observational studies in epidemiology (STROBE) statement: guidelines for reporting observational studies (2007)What principles should govern the use of managed entry agreements? (2011)Generating comparative evidence on new drugs and devices after approval (2020)Generating comparative evidence on new drugs and devices before approval (2020)QALYfying the value of life. (1987)Explaining the Identifiable Victim Effect (1997)A rational framework for decision making by the National Institute For Clinical Excellence (NICE) (2002)Public healthcare resource allocation and the Rule of Rescue (2008)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)Multiple Criteria Decision Analysis (MCDA) for evaluating new medicines in Health Technology Assessment and beyond: The Advance Value Framework (2017)Setting Health Care Priorities in Oregon (1991)Intergenerational Equity: An Exploration of the ‘Fair Innings’ Argument (1997)QALY maximisation and people's preferences: a methodological review of the literature (2004)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)Does NICE have a cost‐effectiveness threshold and what other factors influence its decisions? A binary choice analysis (2004)National Institute for Clinical Excellence and its value judgments (2004)Pricing and reimbursement of orphan drugs: the need for more transparency (2011)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)Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries (2015)Translation of rare disease research into orphan drug development: disease matters (2009)A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study (2010)Availability of and Access to Orphan Drugs (2010)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)Rare essentials drugs for rare diseases as essential medicines (2006)Incentives for orphan drug research and development in the United States (2008)Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs? (2017)
this work references cited by second hop

References

  • 2008Why rare diseases are an important medical and social issue621 cited
  • 2007Assessing the economic challenges posed by orphan drugs279 cited
  • 2005Orphan drugs and the NHS: should we value rarity?229 cited
  • 2005Drugs for exceptionally rare diseases: do they deserve special status for funding?220 cited
  • 2006A journey of hope: lessons learned from studies on rare diseases and orphan drugs185 cited
  • 2010Relative efficacy of drugs: an emerging issue between regulatory agencies and third-party payers174 cited
  • 2002Two decades of orphan product development138 cited
  • 2006Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products88 cited

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