Market access for medicines treating rare diseases
Association between specialised processes for orphan medicines and funding recommendations
Bibliographic Data
| ID | 5158093 |
|---|---|
| Authors | Anna-Maria Fontrier (London School of Economics and Political Science, corresponding author) |
| Year | 2022 |
| Volume | 306 |
| Pages | 115119 |
| Publication date | 2022-08-01 |
| Peer Reviewed | Yes |
| Open Access | Yes |
| Type | ARTICLE |
| Venue | Social Science & Medicine (JOURNAL) |
| Journal identifiers | ISSN: 0277-9536 • E-ISSN: 1873-5347 |
| Publisher | Elsevier BV (PUBLISHER) |
| DOI | 10.1016/j.socscimed.2022.115119 |
| PMID | 35700552 |
| OpenAlex | W4281986092 |
| Language | EN |
| Citations received | 2 |
| References cited | 45 |
Access to medicines treating rare diseases ('orphan medicines') has proven challenging due to high prices and clinical uncertainty. To optimise market access to these medicines, some healthcare systems are implementing specialised pathways and/or processes during marketing authorisation (MA) and/or health technology assessment (HTA). Comparing one setting where these medicines are classed as "orphan" (Scotland) to another where they considered "non-orphan" (Canada), this study aims to explore whether the presence of specialised pathways and processes at MA and HTA levels is associated with more favourable funding recommendations and faster time to market access. A matched sample of 116 medicine-indication pairs with MA approval from 2001 to 2019 in Europe and Canada was identified, and publicly available sources were used for data extraction. Descriptive statistics were used for data analysis. All medicines were commercially marketed in both countries, except one instance in Scotland. In Scotland, more orphan medicines (68.1%) had a favourable HTA recommendation than in Canada (60.4%), while Canada issued more negative HTA recommendations (20.7%) than Scotland (15.5%). Low levels of agreement on HTA recommendations and the main reasons driving recommendations were found between settings. In both countries, medicines with specialised MA approval were less likely to receive negative HTA recommendations than medicines with standard MA. Time to market access was faster in Canada than Scotland, though medicines with specialised MA approval had slower timelines than medicines with standard MA approval in both countries. However, it is unclear whether the presence of orphan designation and HTA specialised processes alone could result in favourable funding recommendations without accounting for other healthcare system-related factors and differences in the decision-making processes across settings. Holistic approaches and better alignment of evidentiary requirements across regulators are needed to optimise access to orphan medicines
Access to medicines · Alternative medicine · Authorization · Business · Descriptive statistics · Economic growth · Economics · Essential medicines · Family medicine · Health care · Health technology · Market access · Marketing authorization · Orphan drug · Public health · Health Systems, Economic Evaluations, Quality of Life · Medicine · Pharmaceutical Economics and Policy · Pharmaceutical studies and practices
| Unique citing works | 2 |
|---|---|
| Citations per year | 1 |
| Citation span | 2024 - 2025 (2) |
| Citation velocity | recent |
| Highly cited | No |
| Citation types | Neutral: 2 |