Orphan drugs approved in Canada: availability and additional therapeutic value
Bibliographic record
Abstract
Orphan drugs approved in Canada: availability and additional therapeutic valuein their recent article in expert opinion on rare drugs, which is no longer accepting submissions, on the accessibility of orphan drugs in Canada, rawson and Adams conclude that "Canadians with rare disorders continue to suffer from a lack of timely and equitable access to innovative treatments" [1].they based this conclusion on an analysis of the percent of orphan drugs approved by either the Food and drug Administration (FdA) or the european Medicines Agency (eMA) from 2015 to 2020 that were submitted to health Canada, the time taken for these drugs to be evaluated by both the Canadian Agency for drugs and technologies in health (CAdth) and the pan-Canadian Pharmaceutical Alliance, the recommendations from these two agencies and finally how many of the drugs were eventually listed on provincial formularies.this letter to the editor examines the biases in what data rawson and Adams left out and their interpretation of their data.
Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.
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 | 0.011 | 0.102 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.004 | 0.007 |
| Science and technology studies | 0.004 | 0.005 |
| Scholarly communication | 0.008 | 0.004 |
| Open science | 0.003 | 0.002 |
| Research integrity | 0.006 | 0.010 |
| Insufficient payload (model declined to judge) | 0.009 | 0.001 |
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".