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 outcomes for all stakeholders. 

Demand for early access to therapies still in clinical trials for the purpose of treating patients with no therapy options is especially familiar in rare diseases and also typical in other devastating diagnoses like terminal cancer, Parkinson’s Disease, Alzheimer’s, heart disease and infectious diseases. Legislation like Right to Try, whilst well-intentioned, can also serve to broaden the chasm between desperate patients and drug developers. 

Where no treatment exists at all or where no treatment is available in the country where a patient resides is it any wonder that such emotionally charged and difficult conversations spill into public consciousness? 

If you would like to learn more about our approach at RareiTi, get in touch, we would be happy to discuss and propose some recommendations to manage your specific needs. 

Natalie Douglas

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