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For several years HCD Economics has keenly watched gene therapy developments in Haemophilia and in 2022 the first two gene therapies, for Haemophilia A & B, were granted marketing authorisation by both the EMA & FDA.

Lysosomal storage diseases (LSDs) are metabolic disorders caused by mutations in genes encoding for the proteins involved in lysosomal homeostasis. Collectively affecting 1:5000 live births and often present as pediatric neurodegenerative diseases.

Idiopathic pulmonary fibrosis (IPF) is a rare, progressive illness of the respiratory system. Tissue in the lungs becomes thick and stiff, forming scars which affects the air sacs, leading to an irreversible decline in lung function. Early symptoms include a gradual onset of shortness of breath, a dry cough and feeling tired. Progression results in numerous associated complications such as pulmonary hypertension, heart failure, pneumonia or pulmonary embolism. 13.5% of IPF patients end up with Lung Cancers.

Informal care is generally thought to mean unpaid care, typically provided to elderly and dependent persons by those they have a social relationship with. Such as a spouse, parent, child or other relative. As well as maybe a neighbour or close friend.

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).