International Society for Cell & Gene Therapy Expanded Access Working Group position paper: key considerations to support equitable and ethical expanded access to investigational cell- and gene-based interventions
Bibliographic record
Abstract
This position paper reviews the Expanded Access pathway for cell and gene therapies, examining its critical role at the nexus of patient need, regulatory frameworks, and scientific advancement. Spearheaded by the International Society for Cell & Gene Therapy's Expanded Access Working Group, it explores how investigational therapies are accessed outside of clinical trials for patients with serious or life-threatening conditions when no approved alternatives exist. Access to cell and gene therapy products are of specific interest to patients because many times the products are bespoke, being used to treat serious and/or incurable conditions, and are potentially curative. As the field of cell and gene therapy rapidly progresses, healthcare professionals face mounting challenges in navigating the balance between access and oversight. Key considerations include transparent communication with patients, robust data reporting, and a discussion of cost recovery models and their implications for long-term commercialization strategies. Equity and inclusivity are central themes, highlighting the need to design pathways that are accessible to diverse patient populations while upholding high scientific and ethical standards. This position paper is presented as a resource for clinicians, researchers, and policymakers navigating the evolving landscape of investigational cell and gene therapies. It emphasizes the importance of ethical frameworks and equitable practices in delivering transformative treatments to patients in need.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.041 | 0.065 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.001 | 0.001 |
| Science and technology studies | 0.005 | 0.007 |
| Scholarly communication | 0.018 | 0.012 |
| Open science | 0.004 | 0.011 |
| Research integrity | 0.033 | 0.027 |
| Insufficient payload (model declined to judge) | 0.033 | 0.014 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".