ReviewAnnals of neurology2012
Unintended effects of orphan product designation for rare neurological diseases.
Review in Annals of neurology, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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
20 citing papers in PubMed, 50 citations in OpenAlex.
- The challenges and opportunities for equitable global access to medications for paediatric rheumatology in low-to middle-income countries: a review on behalf of the Paediatric Global Task Force for Musculoskeletal Health.Arthritis research & therapy · 2026Review
- Article
- Unlocking hidden potential: advancements, approaches, and obstacles in repurposing drugs for cancer therapy.British journal of cancer · 2024Review
- Patient and family experiences of lysosomal storage diseases in Canada: A qualitative interview study.JIMD reports · 2024Article
- Cost-effectiveness of ambroxol in the treatment of Gaucher disease type 2.Open medicine (Warsaw, Poland) · 2024Article
- Neurologic orphan diseases: Emerging innovations and role for genetic treatments.World journal of experimental medicine · 2023Review
- Disentangling the Cost of Orphan Drugs Marketed in the United States.Healthcare (Basel, Switzerland) · 2023Article
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
- Cost-effectiveness of miglustat versus symptomatic therapy of Niemann-Pick disease type C.International journal of clinical pharmacy · 2022Article
- A dashboard for exploring clinical trials sponsorship and potential virtual monopolies.JAMIA open · 2021Article
- Toxicological properties of Δ9-tetrahydrocannabinol and cannabidiol.Arhiv za higijenu rada i toksikologiju · 2020Review
- The changing landscape of clinical trials for mitochondrial diseases: 2011 to present.Mitochondrion · 2020Review
- Molecular insight into the therapeutic potential of phytoconstituents targeting protein conformation and their expression.Phytomedicine : international journal of phytotherapy and phytopharmacology · 2019Review
- VMAT2 Inhibitors in Neuropsychiatric Disorders.CNS drugs · 2018Review
- Therapy development in Huntington disease: From current strategies to emerging opportunities.American journal of medical genetics. Part A · 2018Review
- Rare neurological channelopathies--networks to study patients, pathogenesis and treatment.Nature reviews. Neurology · 2016Review
- Piedmont and Aosta Valley inter-regional network in the context of the Italian National Network for rare diseases.Blood transfusion = Trasfusione del sangue · 2014Review
- The new frontier of genetically targeted therapies for muscle disease.Continuum (Minneapolis, Minn.) · 2013Article
- New treatments for mitochondrial disease-no time to drop our standards.Nature reviews. Neurology · 2013Review
- The economic burden of fragile x syndrome: healthcare resource utilization in the United States.American health & drug benefits · 2013Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 2 institutions in 3 countries.
Funding
Abstract
Since the introduction of the Orphan Drug Act in 1983, designed to promote development of treatments for rare diseases, at least 378 orphan drugs have been approved. Incentives include financial support, tax credits, and perhaps most importantly, extended market exclusivity. These incentives have encouraged industry interest and accelerated research on rare diseases, allowing patients with orphan diseases access to treatments. However, extended market exclusivity has been associated with unacceptably high drug costs, both for newly developed drugs and for drugs that were previously widely available. We suggest that a paradoxical effect of orphan product exclusivity can be reduced patient access to existing drugs. In addition, the costs of each new drug are arguably unsustainable for patients and for the American health care system. Of all the specialties, neurology has the third highest number of orphan product designations, and neurological diseases account for at least one-fifth of rare diseases. Citing the use of tetrabenazine for chorea in Huntington disease, adrenocorticotropic hormone for infantile spasms, and enzyme replacement therapy with alglucosidase alpha for Pompe disease, we highlight these paradoxical effects.
Indexed as
Identifiers
What OpenQuestion holds
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.