Prompted by the current high-profile case of Lisa Stockman Mauriello’s devastating rare disease ALS diagnosis and her family’s fight to access an investigational therapy, tofersen, potentially the only option to alter the course of her disease, we comment on the issues surrounding this case and the possible ethical dilemma this presents whilst applying the long-established…
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Plan to Succeed – consider all your access options when meeting challenges in rare disease.
Global drug launches always pose unique challenges, but are often even greater when in rare disease. The combination of higher prices, complex therapies, smaller patient populations, unique health systems and public health priorities likely mean you’ll need more data and additional supportive evidence to meet the needs of international markets. An early access program…
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Keeping Drug Development Moving – through COVID-19 and Beyond
Along with its direct impact on human health and survival, COVID-19 is indirectly impacting health by causing many companies to hit the pause button on clinical trials. Hundreds of studies have been suspended, and many more are pushing out their start dates. The reasons are many: Site closures Risk of infection from visiting a medical…
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Care Continuity in a Crisis – How home healthcare in the UK plays an invaluable role.
With so much pressure on health systems across the world in dealing with the urgency of the COVID-19 pandemic it is reassuring to know that a major health system like the NHS has the ability and agility to tap into alternative care pathways with home healthcare models to protect more lives, particularly those already coping…
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