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Quality of Life and Rare Disease: Lessons from Spinal Muscular Atrophy
2 April 2019
…and Martina Garau. The Briefing outlines each speaker’s perspective on quality of life measurement in SMA and rare diseases more broadly. Based on the discussion, we highlight four possible strategies…
Assessing the Productivity Value of Vaccines in Health Technology Assessment: Worth a Shot?
17 September 2020
…The avoidance of long-term morbidity, disability and premature deaths from vaccine-preventable diseases unlock productivity gains in the working population. While this creates value inside the health sector, for example through…
The Broader Value of Existing Vaccines in the Fight Against COVID-19: Beware of Tunnel Vision
8 October 2020
…and outbreaks of emerging diseases, but because they can increase the lifetime productivity of vaccinated individuals (Jit et al., 2015; Bärnighausen et al., 2014; Brassel, et al., 2020b). These are…
A NICE Wind of Change? The What and So What of the NICE Methods Consultation
7 December 2020
…patients’ quality of life in addition to life extensions. This means that a larger set of interventions – including those for rare diseases – can potentially be appraised against a…
Childhood Vaccination: Current Controversies
1 July 1984
…and Wales. Effective vaccines are available for both of these diseases so that a substantial proportion of this morbidity may be regarded as unnecessary. Currently, however, only one child in…
Early Experience with Health Technology Assessment of Gene Therapies in the United States: Pricing and Paying for Cures
1 February 2019
Gene therapies, which can provide cures for diseases, are a new area of research for ICER. Some are within two to three years of becoming available publicly. This is exciting,…
A switch to vegan diets could save the NHS £6.7 billion per year, new research reveals
8 January 2024
…many diseases including cancer, cardiovascular disease, cataracts, type 2 diabetes, diverticular disease and obesity. Every 1 million people who switch to a vegan diet would generate an estimated £121 million…
What is the impact of Uniform Pricing, Indication-Based Pricing (IBP) and alternative commercial arrangements for new pharmaceuticals in the UK NHS?
19 November 2024
…Pricing: Efficient Prices for Drugs in a Global Context: VALUE-BASED DIFFERENTIAL PRICING. Health Economics, 24(3), pp.294–301. Danzon, P.M., 2018. Affordability Challenges to Value-Based Pricing: Mass Diseases, Orphan Diseases, and Cures. Value in…
Around the world in HTAs: Abu Dhabi – Advancing HTA and value-based healthcare in the UAE
1 July 2026
…diseases classified as rare by the EMA or FDA are eligible for up to a 3.0x multiplier, and ultra-rare diseases are subject to case-by-case multiplier determination. The CET is calculated…