A National Approach to Reimbursement Decision-Making on Drugs for Rare Diseases in Canada? Insights from Across the Ponds
Bibliographic record
Abstract
Introduction: Regardless of the type of health system or payer, coverage decisions on drugs for rare diseases (DRDs) are challenging.While these drugs typically represent the only active treatment option for a progressive and/or life-threatening condition, evidence of clinical benefit is often limited because of small patient populations and the costs are high.Thus, decisions come with considerable uncertainty and risk.In Canada, interest in developing a pan-Canadian decision-making approach informed by international experiences exists.Objective: To develop an inventory of existing policies and processes for making coverage decisions on DRDs around the world.Methods: A systematic review of published and unpublished documents describing current policies and processes in the top 20 gross domestic product countries was conducted.Bibliographic databases, the Internet and government/health technology assessment organization websites in each country were searched.Two researchers independently extracted information and tabulated it to facilitate qualitative comparative analyses.Policy experts from each country were contacted and asked to review the information collected for accuracy and completeness.Results: Almost all countries have multiple mechanisms through which coverage for a DRD may be sought.However, they typically begin with a review that follows the same process as drugs for more common conditions (i.e., the centralized review process), although specific submission requirements could differ (e.g., no need to submit a cost-effectiveness analysis).When drugs fail to receive a positive recommendation/decision, they are reconsidered by "safety net"type programs.Eligibility criteria vary across countries, as do the decision options, which may be applied to individual patients or patient groups.Conclusions: With few exceptions, countries have not created separate centralized review processes for DRDs.Instead, they have modified components of existing mechanisms and added safety nets.[26] HEALTHCARE POLICY Vol.10 No.4, 2015 Hilary Short et al.qui décrivent les politiques et procédures dans les 20 pays se classant en tête selon le produit intérieur brut.Les bases de données bibliographiques, l'Internet et les sites Web des gouvernements et des organismes d' évaluation des technologies de la santé de chaque pays ont été consultés.Deux chercheurs ont indépendamment recueilli les données et les ont tabulées pour permettre d' effectuer des analyses comparatives qualitatives.Nous avons demandé à des experts des politiques dans chacun des pays de réviser la précision et l' exhaustivité de l'information recueillie.Résultats : Presque tous les pays sont dotés de multiples mécanismes par lesquels on peut obtenir une couverture pour les MMR.Cependant, cela commence habituellement par un examen qui suit les mêmes processus que dans le cas d'un médicament pour une maladie plus commune (c' est-à-dire un processus de révision centralisé), bien que les exigences pour soumettre un dossier peuvent être différentes (par exemple, il n'y a pas besoin de présenter une analyse du coût-efficacité).Si un médicament ne reçoit pas de recommandation ou décision positive, on l' aborde alors en fonction de programmes de type « filet de sécurité ».Les critères d' admissibilité varient d'un pays à l' autre, de même que les choix de décisions, lesquelles peuvent s' appliquer parfois à des patients individuels, parfois à des groupes de patients.Conclusions : Sauf quelques exceptions, les pays n' ont pas créé de processus de révision distincts centralisés pour les MMR.Ils ont plutôt modifié les composantes des mécanismes déjà en place et ont ajouté des filets de sécurité.T
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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 | 0.090 | 0.187 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.002 | 0.002 |
| Bibliometrics | 0.011 | 0.022 |
| Science and technology studies | 0.016 | 0.010 |
| Scholarly communication | 0.020 | 0.007 |
| Open science | 0.004 | 0.007 |
| Research integrity | 0.004 | 0.004 |
| Insufficient payload (model declined to judge) | 0.004 | 0.000 |
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".