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Summary: Background: The Mini Asthma Quality of Life Questionnaire (MiniAQLQ) is a validated disease-specific quality of life (QOL) paper (p) questionnaire. Electronic (e) versions enable inclusion of asthma QOL in electronic medical records and research databases. Purpose: To validate an e-version of the MiniAQLQ, compare time required for completion of e- and p-versions, and determine which version participants prefer. Methods: Adults with stable asthma were randomized to complete either the e- or p-MiniAQLQ, followed by a 2-h rest period before completing the other version. Agreement between versions was measured using the intraclass correlation coefficient (ICC) and Bland–Altman analysis. Results: Two participants with incomplete p-MiniAQLQ responses were excluded. Forty participants (85% female; age 47.7 ± 14.9 years; asthma duration 22.6 ± 16.1 years; FEV1 87.1 ± 21.6% predicted) with both AQLQ scores <6.0 completed the study. Agreement between e- and p-versions for the overall score was acceptable (ICC=0.95) with no bias (difference (Δ) p–e=0.1; P=0.21). ICCs for the symptom, activity limitation, emotional function and environmental stimuli domains were 0.94, 0.89, 0.90, and 0.91 respectively. A small but significant bias (Δ=0.3; P=0.004) was noted in the activity limitation domain. Completion time was significantly longer for the e-version (3.8 ± 1.9min versus 2.7 ± 1.1min; P<0.0001). The majority of patients (57.5%) preferred the e-MiniAQLQ; 35% had no preference. Conclusion: This e-version of the MiniAQLQ is valid and was preferred by most participants despite taking slightly longer to complete. Generalizabilty may be limited in younger (12–17) and older (>65) adults. [Copyright &y& Elsevier]

The last decade has witnessed tremendous progress in cell therapy (CT), regenerative medicine (RM), and gene therapy (GT). However, these advances come with their own set of challenges and opportunities, which may vary among jurisdictions depending on infrastructures, laws, regulations, access to funding, and socioeconomic, political and cultural aspects. In September 2023, a virtual symposium was held on the opportunities and challenges of CT, RM, and GT in Québec, Canada. The symposium brought together scientists, manufacturers, and experts in law, pharmacoeconomics, and regulatory affairs. This work reports on the discussions held during this symposium, with the aim of guiding the development of new policies for CT, RM, and GT treatments in Québec and within the Canadian ecosystem. The comments from three panels were grouped into seven strategic orientations that reflected dominant views among panelists. The orientations were that: (1) an ethical framework must be developed around CT, RM, and GT to enable fair and timely access to treatments for all; (2) governments should spur private and public research investments in CT, RM, and GT; (3) regulatory awareness may be improved among developers through outreach approaches (initiated by the regulator) and early consultations (initiated by developers); (4) the regulations governing the development of CT, RM, and GT products could be streamlined and further adapted to the needs of these emerging products; (5) the skill sets of developers should be mobilized to foster the development and production of CT, RM, and GT products in the academic and industrial setting, and the training of Québec's workforce could be better aligned with industry and population needs; (6) Québec should ensure that the province's production and health care capacity are aligned with current and future needs in CT, RM, and GT products, considering the rapidly evolving landscape of CT and RM; (7) ongoing efforts to reform the current reimbursement framework could be continued in Québec's public, single-payer health care system. This symposium provided guidance for addressing current limitations and taking advantage of opportunities in CT, RM, and GT in Québec. We surmise that other jurisdictions face challenges similar to those discussed in this symposium. The global CT, RM, and GT community should therefore share experiences to promote the optimal development and access to these promising therapies. [ABSTRACT FROM AUTHOR]