Rare and Orphan Diseases
Rare Disease Day 2024: Why Do We Care About Rare?
29 February 2024
Rare Disease Day is observed on the last day of February every year, and today – February 29th – is truly the rarest day.
ISPOR Europe Round-up: “Gene Therapies: Where High Promise Meets High Uncertainty, How Should HTA Methodologies Appropriately Value and Enable Access?”
8 December 2022
OHE, in collaboration with Pfizer, hosted an educational symposium entitled “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
1 September 2021
Medicines for rare and ultra-rare conditions face distinct economic and ethical challenges compared to medicines for more common conditions. These challenges are implicitly acknowledged by the…
Ethical and Economic Issues in the Appraisal of Medicines for Ultra-Rare Conditions
1 January 2020
In light of concerns that not all medicines for ultra-rare (also known as ultra-orphan) conditions are appraised under the same NICE process, a new OHE Consulting…
How Should We Measure Quality of Life Impact in Rare Disease? Recent Learnings in Spinal Muscular Atrophy
1 March 2019
The measurement of quality of life in the context of spinal muscular atrophy (SMA) is challenging. This is because the disease is experienced by children and is rare, which makes data collection difficult. This Briefing reports on a symposium that outlined some lessons that can be learnt from the SMA context that might be more widely applicable.
MoCA Initiative and its Value Framework: An Opportunity to Improve Decision Making for Orphan Drugs in Europe?
18 September 2018
Orphan drugs inherently face a number of challenges in their development and value assessment. In this blog post, we describe the Mechanism of Coordinated Action (MoCA)…
Establishing a Reasonable Price for an Orphan Drug
1 July 2018
The high cost of drugs for rare diseases (‘orphan drugs’) has generated considerable debate. While there is debate in the economic literature over whether a premium…
Appraising Ultra-Orphan Drugs: Is Cost-Per-QALY Appropriate? A Review of the Evidence
1 March 2018
The report addresses the implications of NICE appraising treatments for very rare diseases using a cost-per-QALY gained decision rule of the type used by NICE in…
Comparing Access to Orphan Medicinal Products (OMPs) in the United Kingdom and other European countries
1 March 2017
The European Commission’s (EC) Orphan Medicinal Products Regulation intended to incentivise the research, development and marketing of new treatments for rare and chronically disabling or life-threatening…