ReviewPloS one2015
Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries.
Review in PloS one, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 111 papers, 3 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
111 citing papers in PubMed, 3 syntheses or guidelines pooled it.
- A systematic review of moral reasons on orphan drug reimbursement.Orphanet journal of rare diseases · 2021Pooled it
- Health-Related Quality of Life in Patients with Adult-Onset Myotonic Dystrophy Type 1: A Systematic Review.The patient · 2019Pooled it
- A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries.Frontiers in public health · 2019Pooled it
- Extracting homogenous data from heterogenous diseases: RaraSwed, the Swedish national rare disease quality registry.BMC global and public health · 2026Review
- Rare disease patients protection: theoretical discussion and institutional construction of orphan drug designation in China.Orphanet journal of rare diseases · 2026Article
- Price determinants and pricing policies concerning potentially innovative health technologies: a scoping review.The European journal of health economics : HEPAC : health economics in prevention and care · 2026Article
- Orphan Medications: An Innovative and Promising Approach against Leishmaniasis.Current microbiology · 2026Review
- Research on the innovation of medical expenses protection mechanism for rare disease patients in China.Frontiers in public health · 2026Article
- Assessing rare disease understanding: a novel disease readiness level framework.Orphanet journal of rare diseases · 2025Article
- FDA Approves First Targeted Treatment for Cerebrotendinous Xanthomatosis: A Perspective on a Landmark in Rare Lipid Storage Disease Therapy.Health science reports · 2025Article
- Advances in Cell and Gene Therapy for Rare Disease Treatment.International journal of stem cells · 2025Review
- Multi-criteria decision analysis of value evaluation of rare disease medical insurance drugs in China.BMC public health · 2025Article
- Access to Orphan Medicinal Products in Bulgaria: An Analysis of the Positive Drug List and Individual Access Schemes.Healthcare (Basel, Switzerland) · 2025Article
- Is Brazil following global trends in high-cost treatments? The case of Pompe Disease.Journal of community genetics · 2025Review
- Determinants of the Financial Impact of Orphan Drugs in Italy: Differences Between Expected and Observed Pharmaceutical Expenditure.Drugs - real world outcomes · 2025Article
- Rare disease challenges and potential actions in the Middle East.International journal for equity in health · 2025Article
- The role of public health in rare diseases: hemophilia as an example.Frontiers in public health · 2025Review
- Determinants of orphan drug health technology assessment in South Korea: an empirical analysis.Frontiers in pharmacology · 2025Article
- Research on the quality supervision strategy of drugs sold on the internet under the MAH system.Frontiers in public health · 2025Article
- Mapping challenges in the accessibility of treatment products for urea cycle disorders: A survey of European healthcare professionals.Journal of inherited metabolic disease · 2025Article
51 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveTo review existing regulations and policies utilised by countries to enable patient access to orphan drugs.
methodsA review of the literature (1998 to 2014) was performed to identify relevant, peer-reviewed articles. Using content analysis, we synthesised regulations and policies for access to orphan drugs by type and by country.
resultsFifty seven articles and 35 countries were included in this review. Six broad categories of regulation and policy instruments were identified: national orphan drug policies, orphan drug designation, marketing authorization, incentives, marketing exclusivity, and pricing and reimbursement. The availability of orphan drugs depends on individual country's legislation and regulations including national orphan drug policies, orphan drug designation, marketing authorization, marketing exclusivity and incentives such as tax credits to ensure research, development and marketing. The majority of countries (27/35) had in place orphan drug legislation. Access to orphan drugs depends on individual country's pricing and reimbursement policies, which varied widely between countries. High prices and insufficient evidence often limit orphan drugs from meeting the traditional health technology assessment criteria, especially cost-effectiveness, which may influence access.
conclusionsOverall many countries have implemented a combination of legislations, regulations and policies for orphan drugs in the last two decades. While these may enable the availability and access to orphan drugs, there are critical differences between countries in terms of range and types of legislations, regulations and policies implemented. Importantly, China and India, two of the largest countries by population size, both lack national legislation for orphan medicines and rare diseases, which could have substantial negative impacts on their patient populations with rare diseases.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.