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Category: Innovation for Small Populations

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Ethical and Economic Issues in the Appraisal of Medicines for Ultra-Rare Conditions

Henderson N., Errea M., Skedgel C. and Jofre-Bonet, M.
  • #Consulting report,
  • #Innovation for Small Populations,

How Should We Measure Quality of Life Impact in Rare Disease? Recent Learnings in Spinal Muscular Atrophy

Sampson, C. and Garau, M.
  • #Consulting report,
  • #Innovation for Small Populations,

Multi-Criteria Decision Analysis to Value Orphan Medicines

Sussex, J., Rollet, P., Garau, M., Schmitt, C., Kent, A. and Hutchings, A.
  • #Consulting report,
  • #Innovation for Small Populations,

Establishing a Reasonable Price for an Orphan Drug

Berdud, M., Drummond, M.F., and Towse, A.
  • #Consulting report,
  • #Innovation for Small Populations,

Appraising Ultra-Orphan Drugs: Is Cost-Per-QALY Appropriate? A Review of the Evidence

Towse, A. and Garau, M.
  • #Consulting report,
  • #Innovation for Small Populations,

Orphan Drugs Policies: A Suitable Case for Treatment

  • #Blog,
  • #Innovation for Small Populations,

MoCA Initiative and its Value Framework: An Opportunity to Improve Decision Making for Orphan Drugs in Europe?

Martina Garau and Anna Bucsics
  • #Blog,
  • #Innovation for Small Populations,

Recent Statistics on Orphan Approvals in Scotland and England

Martina Garau
  • #Blog,
  • #Innovation for Small Populations,

Assessment of the Impact of Orphan Medicinal Products on the European Economy and Society

Mestre-Ferrandiz, J., Garau, M., O'Neill, P. and Sussex, J.
  • #Consulting report,
  • #Innovation for Small Populations,

Comparing Access to Orphan Medicinal Products (OMPs) in the United Kingdom and other European countries

Zamora, B., Maignen, F., O’Neill, P., Mestre-Ferrandiz, J. and Garau, M.
  • #Consulting report,
  • #Innovation for Small Populations,

A Case Study Analysis: Challenges in the NICE Evaluation of Multi-Indication Medicines for Rare and Ultra-Rare Diseases

Henderson, N., Firth, I., Errea, M., Skedgel, C. and Jofre-Bonet, M.
  • #Consulting report,
  • #Innovation for Small Populations,

An analysis of orphan medicine expenditure in Europe: is it sustainable?

Jorge Mestre-Ferrandiz, Christina Palaska, Tom Kelly, Adam Hutchings, Adam Parnaby 
  • #Blog,
  • #Innovation for Small Populations,
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