188: Expensive Therapies: Legal and Ethical Analyses
Bibliographic record
Abstract
Increasingly expensive therapies are becoming available for “orphan” diseases. For example, ivacaftor given orally targets and corrects the dysfunctional cell membrane protein in cystic fibrosis (CF), but only in individuals who carry one of several rare genetic CF mutations. Ivacaftor is priced at $300,000 CDN per patients over 6 years of age, per year, for life. Ivacaftor does not cure CF, but is associated with increased lung function, quality of life and body weight. It may slow disease progression, but it will not be known for many years if it does this more effectively than the present, burdensome, approach. To analyse the legal processes and ethical landscape in the Canadian Health Care System relative to provision and funding of expensive therapies for Canadian children with orphan diseases. A scoping review was conducted to understand the extent of the orphan drug issue in Canada and globally. News articles, government websites and peer-reviewed journals were reviewed to understand the approval process. Moral and ethical theories regarding fairness in the allocation of resources for minorities were reviewed, and a legal analysis was conducted to determine if the current policy for orphan drug allocation could be at risk of a tortious claim. The exorbitant cost of orphan drugs impacts public and private insurers worldwide. Canada has no national pharmaceutical strategy nor any orphan drug legislation. A proposed (in 2012) federal framework to address orphan drugs, has not yet been implemented. In Alberta, decisions to cover orphan drugs for a particular patient are made on a case-by-case basis under two dedicated programs. The process seems to lack clarity and transparency, and lobbying and political pressure seem to play a role. The threat of growing orphan drug costs to overall health budgets is real. We found no legal or ethical justification for automatic funding of these effective, but expensive therapies. We do find strong ethical reasons for a national framework, with agreed criteria and processes, including appeal processes and budgets. However, legal and ethical analyses both support the necessity of the process being transparent.
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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 | 0.056 | 0.158 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.004 | 0.004 |
| Science and technology studies | 0.011 | 0.022 |
| Scholarly communication | 0.017 | 0.010 |
| Open science | 0.002 | 0.004 |
| Research integrity | 0.015 | 0.015 |
| Insufficient payload (model declined to judge) | 0.010 | 0.002 |
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".