In today's ACT Brief, we examine FDA input sought on early-phase ibogaine protocols, why mixed outsourcing models spread faster than oversight infrastructure, and a major partnership for a rare kidney disease treatment.
News|Podcasts|September 8, 2026
ACT Brief: Hidden RBQM Value, Rare Disease Patient Partnership, and Safety Perception in IBD
Author(s)Andy Studna, Senior Editor
In today's ACT Brief, we examine value hidden in the RBQM financial model, why rare disease research leads on genuine patient partnership, and how safety concerns shape IBD treatment decisions despite clinical efficacy.
This is the Applied Clinical Trials Brief—your fast track to the latest insights shaping clinical operations and drug development.
- In
part three of the interview , Sylviane de Viron of CluePoints and Abigail Dirks of Tufts CSDD discussed what the RBQM financial model left out: reduced rework, improved data integrity, and inspection readiness. Those excluded factors could represent even greater value than what was modeled, since the eNPV analysis captured only time savings and direct cost reductions visible in operational data. - In a
new contributed article , Sylvain Berthelot examined why patient engagement in most trials remains consultative, with patients invited only after key decisions are made. Rare disease communities demonstrate that genuine partnership from protocol inception improves feasibility, reduces unnecessary burden, and identifies outcomes that matter to patients themselves. - In an
interview from Pharmaceutical Executive , Christian Thienel, director at Back Bay Life Sciences, discussed the persistent safety perception problem that constrains oral IBD treatments despite strong clinical efficacy. While the leading oral JAK inhibitor shows strong data and a relatively favorable safety profile, the class carries baggage from earlier adverse events, cardiovascular risks, and malignancy concerns that physicians find difficult to separate from any individual product.
That's all for today's ACT Brief. Join us tomorrow for more updates shaping clinical operations and drug development. Thanks for listening.
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