Rare and Orphan Diseases

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Insight
February 2024

Rare Disease Day 2024: Why Do We Care About Rare?

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Insight
December 2022

ISPOR Europe Round-up: “Gene Therapies: Where High Promise Meets High Uncertainty, How Should HTA Methodologies Appropriately Value and Enable Access?”

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

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

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

Insight
September 2018

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

Establishing a Reasonable Price for an Orphan Drug

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

Market Access

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

Multi-Criteria Decision Analysis to Value Orphan Medicines

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

Health Policy and Regulation

Access Mechanisms for Orphan Drugs: A Comparative Study of Selected European Countries

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Huntington’s Chorea

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Rare Disease Day 2024: Why Do We Care About Rare?

ISPOR Europe Round-up: “Gene Therapies: Where High Promise Meets High Uncertainty, How Should HTA Methodologies Appropriately Value and Enable Access?”

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

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

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

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

Establishing a Reasonable Price for an Orphan Drug

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

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

Multi-Criteria Decision Analysis to Value Orphan Medicines

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

Access Mechanisms for Orphan Drugs: A Comparative Study of Selected European Countries

Huntington’s Chorea

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