{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,19]],"date-time":"2026-09-19T19:17:26Z","timestamp":1789845446600,"version":"4.0.1"},"reference-count":30,"publisher":"Public Library of Science (PLoS)","issue":"8","license":[{"start":{"date-parts":[[2022,8,15]],"date-time":"2022-08-15T00:00:00Z","timestamp":1660521600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Department of Science and Technology, Government of India","award":["PDF\/2016\/002340"],"award-info":[{"award-number":["PDF\/2016\/002340"]}]},{"name":"Department of Electronics, IT, BT and S&T of the Government of Karnataka"}],"content-domain":{"domain":["www.plosglobalpublichealth.org"],"crossmark-restriction":false},"short-container-title":["PLOS Glob Public Health"],"abstract":"<jats:p>We wished to determine whether rare diseases patients from India had been enrolled in international trials to develop novel orphan drugs. There are two reasons to be interested in this. (a) Different ethnic or racial groups may respond differently to a particular drug. India has huge ethnic diversity, and to exclude such participants is to severely limit the diversity of any trial; (b) Even if a suitable drug for a rare disease is available in India, it may be astronomically priced, in a country where most healthcare expenditure is out-of-pocket. We identified 63 orphan drugs, approved by the US Food and Drug Administration (FDA) after 2008, for which there were 202 trials in the US government\u2019s clinical trial registry, ClinicalTrials.gov. Only nine of these trials had run in India. These trials pertained to six drugs. The drugs were for the conditions B-cell Lymphoma, Chronic Myeloid Leukemia, Gaucher disease Type 1, Malaria, Myeloma and Pulmonary Arterial Hypertension. Further research is required as to why patients from India are not part of foreign drug development programmes for rare diseases. We then asked how many of the remaining 193 trials had recruited people of Indian origin, residing in other countries, and found that not more than 1% of these trials had done so. Also, only 11 of the 193 trials had recruited from other lower income countries. Participation from low-income countries in trials for orphan drugs is poor.<\/jats:p>","DOI":"10.1371\/journal.pgph.0000890","type":"journal-article","created":{"date-parts":[[2022,8,15]],"date-time":"2022-08-15T17:24:45Z","timestamp":1660584285000},"page":"e0000890","update-policy":"https:\/\/doi.org\/10.1371\/journal.pgph.corrections_policy","source":"Crossref","is-referenced-by-count":15,"title":["Rare disease patients in India are rarely involved in international orphan drug trials"],"prefix":"10.1371","volume":"2","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0041-3986","authenticated-orcid":true,"given":"Monoswi","family":"Chakraborty","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6727-9702","authenticated-orcid":true,"given":"Mohua Chakraborty","family":"Choudhury","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6958-8269","authenticated-orcid":true,"given":"Indraneel","family":"Chakraborty","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2410-1431","authenticated-orcid":true,"given":"Gayatri","family":"Saberwal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"340","published-online":{"date-parts":[[2022,8,15]]},"reference":[{"key":"pgph.0000890.ref001","doi-asserted-by":"crossref","first-page":"570","DOI":"10.1126\/science.abe2565","article-title":"For better drugs, diversify clinical trials","volume":"371","author":"NN Bumpus","year":"2021","journal-title":"Science"},{"key":"pgph.0000890.ref002","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1038\/d41586-018-05049-5","article-title":"When will clinical trials finally reflect diversity?","volume":"557","author":"TC Knepper","year":"2018","journal-title":"Nature"},{"key":"pgph.0000890.ref003","article-title":"S.1 - 103rd Congress (1993\u20131994): National Institutes of Health Revitalization Act of 1993.","author":"EM Kennedy"},{"key":"pgph.0000890.ref004","doi-asserted-by":"crossref","first-page":"148","DOI":"10.1016\/j.cpcardiol.2018.11.002","article-title":"Increasing Diversity in Clinical Trials: Overcoming Critical Barriers.","volume":"44","author":"LT Clark","year":"2019","journal-title":"Curr Probl Cardiol"},{"key":"pgph.0000890.ref005","article-title":"FDA deals blow to Incyte\u2019s PD-1 retifanlimab after battering advisory panel meeting","author":"B. Adams","year":"2021","journal-title":"FierceBiotech"},{"key":"pgph.0000890.ref006","doi-asserted-by":"crossref","DOI":"10.1017\/S0016672314000111","article-title":"Organization for rare diseases India (ORDI)\u2013addressing the challenges and opportunities for the Indian rare diseases\u2019 community.","volume":"96","author":"HK Rajasimha","year":"2014","journal-title":"Genet Res (Camb)."},{"key":"pgph.0000890.ref007","unstructured":"Ministry of Health and Family Welfare, Government of India. National policy for treatment of rare diseases. 2017. Available: https:\/\/www.google.com\/url?sa=t&rct=j&q=&esrc=s&source=web&cd=&cad=rja&uact=8&ved=2ahUKEwj32tLSxNPzAhUAyzgGHZQQDAMQFnoECAYQAQ&url=https%3A%2F%2Fmain.mohfw.gov.in%2Fsites%2Fdefault%2Ffiles%2FRare%2520Diseases%2520Policy%2520FINAL.pdf&usg=AOvVaw1-REDpLtPhYTf3kHn6VzJn. [Accessed 29 Jan 2022]."},{"key":"pgph.0000890.ref008","doi-asserted-by":"crossref","first-page":"20","DOI":"10.1016\/j.ymgme.2008.10.003","article-title":"Clinical research for rare disease: Opportunities, challenges, and solutions","volume":"96","author":"RC Griggs","year":"2009","journal-title":"Mol Genet Metab"},{"key":"pgph.0000890.ref009","doi-asserted-by":"crossref","first-page":"117","DOI":"10.1186\/s13023-019-1093-6","article-title":"The role of patient organizations in the rare disease ecosystem in India: an interview based study.","volume":"14","author":"MC Choudhury","year":"2019","journal-title":"Orphanet J Rare Dis"},{"key":"pgph.0000890.ref010","unstructured":"Orphan Drug Designations and Approvals. Available: https:\/\/www.accessdata.fda.gov\/scripts\/opdlisting\/oopd\/. [Accessed 29 Jan 2022]."},{"key":"pgph.0000890.ref011","unstructured":"FDA\u2019s Orange Book. Available: https:\/\/www.fda.gov\/drugs\/drug-approvals-and-databases\/approved-drug-products-therapeutic-equivalence-evaluations-orange-book. [Accessed 29 Jan 2022]."},{"key":"pgph.0000890.ref012","unstructured":"Orange Book: Approved Drug Products with Therapeutic Equivalence Evaluations. Available: https:\/\/www.accessdata.fda.gov\/scripts\/cder\/ob\/index.cfm. [Accessed 29 Jan 2022]."},{"key":"pgph.0000890.ref013","unstructured":"Drugs@FDA: FDA-Approved Drugs. Available: https:\/\/www.accessdata.fda.gov\/scripts\/cder\/daf\/ [Accessed 29 Jan 2022]."},{"key":"pgph.0000890.ref014","doi-asserted-by":"crossref","first-page":"e068791","DOI":"10.1136\/bmj-2021-068791","article-title":"Searching clinical trials registers: guide for systematic reviewers","volume":"377","author":"KE Hunter","year":"2022","journal-title":"BMJ"},{"key":"pgph.0000890.ref015","doi-asserted-by":"crossref","first-page":"e0251191","DOI":"10.1371\/journal.pone.0251191","article-title":"A comparative analysis of important public clinical trial registries, and a proposal for an interim ideal one.","volume":"16","author":"N Venugopal","year":"2021","journal-title":"PLOS ONE"},{"key":"pgph.0000890.ref016","doi-asserted-by":"crossref","first-page":"911","DOI":"10.1007\/s12038-012-9256-9","article-title":"Complex genetic origin of Indian populations and its implications","volume":"37","author":"R Tamang","year":"2012","journal-title":"J Biosci"},{"key":"pgph.0000890.ref017","unstructured":"Sengar MS. Appeal By Girl Mahi,7, For Treatment Of Rare Genetic Disorder Morquio Syndrome That Costs 2.5 Crore. 21 Aug 2021. Available: https:\/\/www.ndtv.com\/india-news\/appeal-by-girl-mahi-7-for-treatment-of-rare-genetic-disorder-morquio-syndrome-that-costs-2-5-crore-2515433#pfrom=home-ndtv_topstories. [Accessed 29 Jan 2022]."},{"key":"pgph.0000890.ref018","unstructured":"Menon VK. Mumbai: Six-month-old baby Teera finally gets her lifesaving shot. 27 Feb 2021. Available: https:\/\/www.mid-day.com\/mumbai\/mumbai-news\/article\/mumbai-six-month-old-baby-teera-finally-gets-her-lifesaving-shot-23161526. [Accessed 29 Jan 2022]."},{"key":"pgph.0000890.ref019","unstructured":"Anonymous. Regulating India\u2019s regressive health insurance\u2014The Hindu. 11 Aug 2016. Available: https:\/\/www.thehindu.com\/sci-tech\/health\/policy-and-issues\/Malady-Nation-Regulating-India%E2%80%99s-regressive-health-insurance\/article14564554.ece. [Accessed 29 Jan 2022]."},{"key":"pgph.0000890.ref020","first-page":"105","article-title":"Increasing Out-Of-Pocket Health Care Expenditure in India-Due to Supply or Demand?","volume":"1","author":"T Jayakrishnan","year":"2016","journal-title":"Pharmacoeconomics"},{"key":"pgph.0000890.ref021","unstructured":"Anonymous. Diversity in Clinical Trials: Going Beyond Why to How. In: ACRP [Internet]. 20 Apr 2021. Available: https:\/\/acrpnet.org\/2021\/04\/20\/diversity-in-clinical-trials-going-beyond-why-to-how\/. [Accessed 29 Jan 2022]."},{"key":"pgph.0000890.ref022","doi-asserted-by":"crossref","first-page":"241","DOI":"10.1186\/s13023-019-1224-0","article-title":"The work, goals, challenges, achievements, and recommendations of orphan medicinal product organizations in India: an interview-based study.","volume":"14","author":"MC Choudhury","year":"2019","journal-title":"Orphanet J Rare Dis"},{"key":"pgph.0000890.ref023","doi-asserted-by":"crossref","first-page":"e2017018","DOI":"10.4084\/mjhid.2017.018","article-title":"\u03b2-Thalassemia Distribution in the Old World: an Ancient Disease Seen from a Historical Standpoint","volume":"9","author":"V De Sanctis","year":"2017","journal-title":"Mediterr J Hematol Infect Dis"},{"key":"pgph.0000890.ref024","unstructured":"Ministry of Health and Family Welfare. Govt of India. New Drugs and Clinical Trials Rules, 2019. 2019. Available: https:\/\/cdsco.gov.in\/opencms\/export\/sites\/CDSCO_WEB\/Pdf-documents\/NewDrugs_CTRules_2019.pdf"},{"key":"pgph.0000890.ref025","unstructured":"The Food and Drug Administration Amendments Act of 2007 (Public Law 110\u201385), amending the Federal Food, Drug, and Cosmetic Act, 21 U.S.C. \u00a7301."},{"key":"pgph.0000890.ref026","doi-asserted-by":"crossref","unstructured":"Zarin DA, Tse T, Williams RJ, Califf RM, Ide NC. The ClinicalTrials.gov Results Database\u2014Update and Key Issues. In: http:\/\/dx.doi.org\/10.1056\/NEJMsa1012065 [Internet]. 2 Mar 2011 [cited 27 Jan 2019]. doi: 10.1056\/NEJMsa1012065","DOI":"10.1056\/NEJMsa1012065"},{"key":"pgph.0000890.ref027","doi-asserted-by":"crossref","first-page":"e14701","DOI":"10.1371\/journal.pone.0014701","article-title":"The Quality of Registration of Clinical Trials.","volume":"6","author":"RF Viergever","year":"2011","journal-title":"PLoS ONE."},{"key":"pgph.0000890.ref028","doi-asserted-by":"crossref","first-page":"e017719","DOI":"10.1136\/bmjopen-2017-017719","article-title":"Discrepancies between ClinicalTrials.gov recruitment status and actual trial status: a cross-sectional analysis","volume":"7","author":"CW Jones","year":"2017","journal-title":"BMJ Open"},{"key":"pgph.0000890.ref029","doi-asserted-by":"crossref","first-page":"e0193088","DOI":"10.1371\/journal.pone.0193088","article-title":"Prevalence of clinical trial status discrepancies: A cross-sectional study of 10,492 trials registered on both ClinicalTrials.gov and the European Union Clinical Trials Register.","volume":"13","author":"J Fleminger","year":"2018","journal-title":"PLOS ONE."},{"key":"pgph.0000890.ref030","doi-asserted-by":"crossref","DOI":"10.1186\/s13063-019-3592-0","article-title":"An analysis of deficiencies in the data of interventional drug trials registered with Clinical Trials Registry\u2014India.","volume":"20","author":"M Pillamarapu","year":"2019","journal-title":"Trials"}],"container-title":["PLOS Global Public Health"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/dx.plos.org\/10.1371\/journal.pgph.0000890","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,8,15]],"date-time":"2022-08-15T17:25:27Z","timestamp":1660584327000},"score":1,"resource":{"primary":{"URL":"https:\/\/dx.plos.org\/10.1371\/journal.pgph.0000890"}},"subtitle":[],"editor":[{"given":"Nikita","family":"Mehra","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"editor"}]}],"short-title":[],"issued":{"date-parts":[[2022,8,15]]},"references-count":30,"journal-issue":{"issue":"8","published-online":{"date-parts":[[2022,8,15]]}},"URL":"https:\/\/doi.org\/10.1371\/journal.pgph.0000890","relation":{},"ISSN":["2767-3375"],"issn-type":[{"value":"2767-3375","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,8,15]]}}}