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Our news coverage in 2022, has focused on our commitment to generating evidence to inform decisions that deliver health equity. Our news reports share methodologies and results from our innovative research programme covering a wide range of disease areas and applied health economic analytical techniques.

Approximately 6,000–7,000 of the 30,000 known diseases could be defined as rare or orphan diseases with a prevalence threshold of around 40-50 cases per 100,000 people. Meaning the total global prevalence of rare disease is around 5%, or 400 million people worldwide. With over 30 million rare disease sufferers living in the EU and 25 million from the US.

Medical advances over the last century have afforded us longer and healthier lives. We possess the ability to heal injuries, fight infectious and chronic diseases. However the longer people live, the greater their chance of developing dementia.

Research published by HCD Economics and partners during 2022 is informing clinical and policy decisions for the optimal management of paediatric haemophilia. These decisions are particularly relevant as innovation in haemophilia research and development is accelerating.

A couple of weeks ago HCD staff attended an interesting online event: ‘Health Technology Assessment of Gene Therapies: Are Methods Fit for Purpose’. Facilitated by the Office of Health Economics (OHE). This was non-promotional meeting discussing a recent Pfizer funded, consulting report.

HCD Economics has championed the application of Time Trade Off (TTO) methodology, to the research objective of expanding the health economic evidence for treatments that improve the life of patients. A number of TTO studies are underway and in the March 22nd edition of the prestigious European Journal of Health Economics (impact factor 3.689 [2020] - 472,118 downloads (2021), the TTO study undertaken by HCD Economics was published and is available now as an open access publication. (https://link.springer.com/article/10.1007/s10198-022-01449-7).

In the 23 June 2022 announcement published by the UK National Institute for Health and Care Excellence (NICE), launching the NICE real-world evidence (RWE) framework (Corporate document ECD9), three key considerations for the conduct of RWE studies are included in the overview.

The European Medicines Agency (EMA), which serves a population of 500 million citizens, has the stated remit of promoting and protecting public health in European Union (EU) member states, as well as the countries in the European Economic Area, by ensuring all medicines available on the EU market are safe, effective and of high quality.

The treatment landscape for the Haemophilia community has broadened in recent years, largely because of the recent advances in genetic treatment and other innovations

The HCD and Prime Access team attended ISPOR (The International Society for Pharmaeconomics and Outcomes Research) ‘in-person’, the first time since the COVID-19 pandemic that the conference returned to an in-person event (with on-line participation still available for those delegates preferring to participate virtually).