Uncertainty management in regulatory and health technology assessment decision-making on drugs: guidance of the HTAi-DIA Working Group.

Objectives: Uncertainty is a fundamental component of decision making regarding access to and pricing and reimbursement of drugs. The context-specific interpretation and mitigation of uncertainty remain major challenges for decision makers. Following the 2021 HTAi Global Policy Forum, a cross-sectoral, interdisciplinary HTAi-DIA Working Group (WG) was initiated to develop guidance to support stakeholder deliberation on the systematic identification and mitigation of uncertainties in the regulatory-HTA interface.

Barriers and solutions to improve access for chimeric antigen receptor therapies.

Chimeric antigen receptor T cells are a potentially curative new therapeutic option, but access challenges remain. The limited number of certified treatment centers and the need to travel to these centers, the expenses of travel and lodging and the out-of-pocket costs associated with treatment pose a challenge for patients. Further, the logistics of follow-up coupled with an reimbursement environment make chimeric antigen receptor T-cell treatment an unattractive proposition for many providers.

A genomewide association study on individuals with occludable angles identifies potential risk loci for intraocular pressure.

Glaucoma is a heterogeneous group of optic neuropathies and is one of the leading causes of irreversible blindness worldwide. Primary angle closure glaucoma (PACG) is a major subtype, prevalent mostly in east and south Asia, where occludable anterior chamber angle is considered as a primary risk factor, which in turn could be responsible for high intraocular pressure (IOP) and subsequent neurodegeneration of retinal ganglion cells. Clinically, IOP is considered as a major risk factor for glaucoma and viewed as an important endophenotype to promote the disease severity.