Roche cancels rare disease trial, infuriates parents, scientists

Roche, the Swiss pharmaceutical giant, earlier this year abruptly cancelled an international clinical trial of a drug being studied to treat children with a rare genetic disorder, citing “trade-offs made … to increase the overall portfolio value.”

The news stunned parents of children enrolled in the Phase 2 study, as well as the academic researchers running it. Shannon Sadecki, whose son was in the trial, said she felt like she’d been “hit by a bus.” Parents say Roche’s decision left them heartbroken and angry because they had hoped the medication might be life-changing for their children, who have severe developmental delays caused by dup15q syndrome.

Drugmakers frequently shift research priorities in the face of pressure to produce steady profits, and drugs being developed for rare diseases are especially vulnerable to such cuts. But the ripple effects of those business decisions are often devastating and unseen.