This preprint is under consideration at Orphanet Journal of Rare Diseases. A preprint is a preliminary version of a manuscript that has not completed peer review at a journal. Research Square does not conduct peer review prior to posting preprints. The posting of a preprint on this server should not be interpreted as an endorsement of its validity or suitability for dissemination as established information or for guiding clinical practice.
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Research Article
Galliano Zanello
Institut national de la santé et de la recherche médicale: INSERM
Corresponding Author
ORCiD: https://orcid.org/0000-0001-8512-4384
License:
This work is licensed under a CC BY 4.0 License. Read Full License
Background
Treatments are often unavailable for rare disease patients, especially in low-and-middle-income countries. Reasons for this include lack of financial support for therapies and onerous regulatory requirements for approval of drugs. Other barriers include lack of reimbursement, administrative infrastructure, and knowledge about diagnosis and drug treatment options. The International Rare Diseases Research Consortium set up the Rare Disease Treatment Access Working Group with the first objective to develop an essential list of medicinal products for rare diseases.
Results
The Working Group extracted 215 drugs with Orphan designation in the FDA, EMA databases and/or China’s Rare Diseases Catalog. The drugs were organized in seven disease categories: metabolic, neurologic, hematologic, anti-inflammatory, endocrine, pulmonary, and immunologic, plus a miscellaneous category.
Conclusions
The proposed list of essential medicinal products for rare diseases is intended to initiate discussion and collaboration among patient advocacy groups, health care providers, industry and government agencies to enhance access to appropriate medicines for all rare disease patients throughout the world.
Keywords
rare disease medicine, medicine access
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Peer Review Timeline
CURRENT STATUS: Under Revision
Version 1
Posted 22 Mar, 2021
No community comments so far
Review #2 received
Received 13 Apr, 2021
Editorial decision: Major revision
On 13 Apr, 2021
Reviewer #2 agreed
On 05 Apr, 2021
Review #1 received
Received 20 Mar, 2021
Reviewer #1 agreed
On 19 Mar, 2021
Review #? received
Received 19 Mar, 2021
Reviewers invited
Invitations sent on 19 Mar, 2021
Editor assigned
On 16 Mar, 2021
Submission checks complete
On 16 Mar, 2021
Editor invited
On 16 Mar, 2021
First submitted
On 26 Feb, 2021
Metrics
Comments: 0
PDF Downloads: ...
HTML Views: ...
Subject Areas
Preventive Medicine
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This preprint is under consideration at Orphanet Journal of Rare Diseases. A preprint is a preliminary version of a manuscript that has not completed peer review at a journal. Research Square does not conduct peer review prior to posting preprints. The posting of a preprint on this server should not be interpreted as an endorsement of its validity or suitability for dissemination as established information or for guiding clinical practice.
Learn more about In Review
Research Article
Galliano Zanello
Institut national de la santé et de la recherche médicale: INSERM
Corresponding Author
ORCiD: https://orcid.org/0000-0001-8512-4384
Badges
Prescreen
This manuscript passed our Prescreen assessment
Complete author information
Appropriate declaration statements
Potential risks to human health
Prescreen
Peer Review Timeline
CURRENT STATUS: Under Revision
Version 1
Posted 22 Mar, 2021
No community comments so far
Review #2 received
Received 13 Apr, 2021
Editorial decision: Major revision
On 13 Apr, 2021
Reviewer #2 agreed
On 05 Apr, 2021
Review #1 received
Received 20 Mar, 2021
Reviewer #1 agreed
On 19 Mar, 2021
Review #? received
Received 19 Mar, 2021
Reviewers invited
Invitations sent on 19 Mar, 2021
Editor assigned
On 16 Mar, 2021
Submission checks complete
On 16 Mar, 2021
Editor invited
On 16 Mar, 2021
First submitted
On 26 Feb, 2021
Metrics
Comments: 0
PDF Downloads: ...
HTML Views: ...
Subject Areas
Preventive Medicine
License:
This work is licensed under a CC BY 4.0 License. Read Full License
Background
Treatments are often unavailable for rare disease patients, especially in low-and-middle-income countries. Reasons for this include lack of financial support for therapies and onerous regulatory requirements for approval of drugs. Other barriers include lack of reimbursement, administrative infrastructure, and knowledge about diagnosis and drug treatment options. The International Rare Diseases Research Consortium set up the Rare Disease Treatment Access Working Group with the first objective to develop an essential list of medicinal products for rare diseases.
Results
The Working Group extracted 215 drugs with Orphan designation in the FDA, EMA databases and/or China’s Rare Diseases Catalog. The drugs were organized in seven disease categories: metabolic, neurologic, hematologic, anti-inflammatory, endocrine, pulmonary, and immunologic, plus a miscellaneous category.
Conclusions
The proposed list of essential medicinal products for rare diseases is intended to initiate discussion and collaboration among patient advocacy groups, health care providers, industry and government agencies to enhance access to appropriate medicines for all rare disease patients throughout the world.
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