Beyond compassion

Patient advocates, with support from FDA, are honing new arguments to persuade drug companies to routinely provide pre-approval access to investigational drugs. Rather than appealing to companies’ compassion, or threatening to blacken their reputations through social media campaigns, advocacy groups are making the case that medium- and large-scale expanded access programs can produce real-world data to support regulatory and reimbursement decisions.

The case is especially compelling for rare conditions that have no effective treatments, as well as in circumstances when a randomized, controlled clinical trial is ethically impossible. It also can apply to more common indications, especially to generate safety data and demonstrate efficacy about supplementary indications. ...