Compassionate Considerations

The current public debate Biogen finds itself in regarding compassionate use/expanded access to Tofersen is an unfortunate situation for them and other companies who find themselves in a similar dilemma. Unfortunately, this will likely happen again unless handled differently. At RareiTi, we believe alternative steps can be taken to avert a public outcry, providing better…

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Expanded Access – the Right Applies

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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