Drugmakers Are Putting Family-Funded Rare Disease Therapeutic Research on Hold

May 16, 2023

Families of patients have often led funding for rare disease therapeutic development through donation campaigns. However, many drugmakers have put these programs on hold, with 26 rare disease projects shelved in the past 16 months. This has caused an outcry from patient advocates. Pharma analysts argue financial reasons are to blame.

According to Jared Whitlock, “The shelved therapies target everything from seizures to intellectual disability to brain diseases that rob children of their abilities and then kill them at a young age. The list, while extensive, is limited by what companies disclose. Non-public companies can say little, and other drug developers often don’t talk about pre-clinical work that’s been cut. It was not always clear why programs were shelved. But the cut programs were largely discontinued as part of company restructurings, amid an ongoing industry downturn that took hold in early 2022. The pullback was predominantly in treatments for ultra-rare diseases that face regulatory challenges and marginal monetary incentives.”

To read more, click here.