In clinical medicine, shared decision making (SDM) is a well-recognized strategy to enhance engagement of both patients and clinicians in medical decisions. The success of liver-directed gene therapy (GT) to transform severe congenital haemophilia A (HA) from an incurable to a curable disease has launched a shift beyond current standards of treatment. However, GT acceptance remains low in the community of HA persons. We argue for both persons with haemophilia (PWH) and specialists in HA care including clinicians, as needing SDM-oriented educational programs devoted to GT. Here, we provide an ad hoc outline to implement education to SDM and tailor clinician information on GT to individual PWHs. Based on routine key components of SDM: patient priorities; recommendations based on individual risk reduction; adverse effects; drug-drug interactions; alternatives to GT; and ongoing re-assessment of the objectives as risk factors (and individual priorities) change, this approach is finalized to exploit efficacious communication.

In the context of a pandemic, the rapid development of clinical practice guidelines (CPGs) is critical. The guideline development process includes prioritization of the guideline topic, questions and health outcomes. This case study describes the application of a new methodology to prioritize questions and rate the importance of health outcomes for a COVID-19 dental guideline.
Panel members rated the topic and the questions\' overall importance, using a 9-point scale (1 = least important; 9 = most important). In addition, they rated six criteria if multiple questions received the same overall importance rating: common in practice, uncertainty in practice, variation in practice, new evidence available, cost consequences, not previously addressed. Panellists also rated the importance of each outcome, defined with health outcome descriptors, using a 9-point scale and the utility of health outcomes on a visual analogue scale. The correlation between each criterion and overall question importance was tested by Spearman correlation coefficient.
Of seven topics, four were rated as high priority and three were rated as important, but not of high priority. Thirty-six percent of the questions (18/50) were rated as high priority to address in the guideline and 64% (32/50) were rated as an important question but not of high priority. Of the 11 outcomes, 72.7% were rated as critical for decision making. The mean utility rating was 0.57 (SD 0.32), with a minimum mean rating of 0.16 and a maximum of 0.76 (SD 0.23).
This case study demonstrated that this approach provides a rigorous and transparent methodology to conduct the prioritizations of guideline topics, questions and health outcomes.