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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).

Although around 4-5000 people are known to be diagnosed with Fabry Disease in the United States (Mol Genet Metab 2008, 93:112-128), the US National Fabry Foundation proposes that just under 30,000 US citizens could be living, unknowingly, with classic and non-classic Fabry disease (https://www.fabrydisease.org/index.php/about-fabry-disease/how-many-people-have-fabry-disease

Movement disorders are commonly neurological, involving both voluntary and involuntary abnormal, reduced or slowed movement. Ataxia affects the cerebellum, the brain’s coordination centre, causing uncoordinated, clumsy balance and limb movements as well as impaired speech. Dystonia involves sustained involuntary muscle contractions with twisting, repetitive movements. Myoclonus muscle jerks can be lightning-quick, effecting either a single muscle or a whole group of muscles. Chorea is characterized by repetitive, brief, irregular rapid, involuntary movements commonly of the face, mouth, trunk and limbs. HCD Economics has already conducted a large Burden of Illness Study of Huntington’s Chorea, one of the most well know genetic diseases.

In 2017, a study was undertaken to understand the burden of illness for paediatric haemophilia patients, treated with the standard of care available at the time. 785 patients were included in the study, which was conducted across Germany, France, Italy, Spain and the United Kingdom. The study, identified by the acronym CHESS PAEDS raised awareness of the prevalence of problem joints and associated quality of life impairment for these patients.