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Drugs for exceptionally rare diseases: do they deserve special status for funding?

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References

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2006A journey of hope: lessons learned from studies on rare diseases and orphan drugs185 cited
2018Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group131 cited
2017Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs?113 cited
2018Evaluating and Valuing Drugs for Rare Conditions: No Easy Answers109 cited
2017Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)103 cited
2010Societal views on orphan drugs: cross sectional survey of Norwegians aged 40 to 67103 cited
2014Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework99 cited
2006Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products88 cited
2010Access to orphan drugs despite poor quality of clinical evidence82 cited
2008The use of cost-effectiveness by the National Institute for Health and Clinical Excellence (NICE): no(t yet an) exemplar of a deliberative process

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2015Can the EVIDEM Framework Tackle Issues Raised by Evaluating Treatments for Rare Diseases: Analysis of Issues and Policies, and Context-Specific Adaptation77 cited

2010A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study76 cited

2008Media influence on Herceptin subsidization in Australia: application of the rule of rescue?57 cited

2011Disrupting the Community—Saving Public Health Ethics from the EU Internal Market19 cited