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The potential of gene therapy to transform treatment for patients with haemophilia is entering clinical practice. The literature in adeno-associated viral (AAV) vector-mediated gene therapy demonstrates improvement in endogenous factor levels over sustained periods, significant reduction in annualised bleed rates, lower exogenous factor usage and thus far a positive safety profile. Gene therapy is also associated improvements in the burden of illness for haemophilia patients as measured by HRQoL and economic analysis.

Rare Disease Day 2020 - raising awareness amongst the general public and decision-makers about rare diseases and their impact on patients' lives. Rare Disease Day will be on February 29th 2020. As a research partner and advocate for the rare disease community, HCD Economics is sharing news of this event with our website community.

HCD’s Hayley Hubberstey, Real World Evidence Manager and Dr Harpal Dhillon, Global Medical Affairs Director attended ASH in December 2019 for the shared objective to learn the latest in terms of medical advances/understanding in HCD’s research areas of Sickle Cell Disease and Haemophilia and to identify 2020 research areas, that would benefit from the patient empowered methodology HCD has pioneered, alongside a need in the future research area to unearth evidence of burden of illness and provide expertise pro bono, in support of patient and political advocacy.

The International Gaucher Alliance (IGA) publishes a regular monthly newsletter. In the November 2019 edition reports are filed from all parts of the IGA organisation, including established and emerging National members of the Gaucher community such as Belgium, Albania, Pakistan and the United States.

Hayley Hubberstey, HCD Economics Real World Evidence (RWE) lead for research in Sickle Cell Disease, attended ASCAT last week (21st – 23rd October) and shares her selection of highlights from the conference in this report, and comments on how these inform and support the need for RWE research in SCD.

HCD Economics are looking to hire an experienced Project Coordinator for an immediate start to help grow the project management side of the business

In Sickle Cell Disease (SCD), RWE (Real world evidence) is important and useful for individual patients and families. John James, CEO of the Sickle Cell Society (SCS), speaks to HCD Economics about our forthcoming RWE research partnership.

Writing in the Sage Journal, Medicine Access@Point of Care, Jasmine Barman-Aksözen reports on a personal case study that highlights the importance of patient empowerment and reports on what can be achieved in terms of access to treatment. The full article, published in August 2019, is available (link below), this news item focuses on the ‘take home messages’, summarised within the paper. HCD Economics completely endorses the criteria for meaningful participation as outlined in the paper.