{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,4,16]],"date-time":"2025-04-16T04:20:26Z","timestamp":1744777226167,"version":"3.40.4"},"reference-count":20,"publisher":"Springer Science and Business Media LLC","issue":"5","license":[{"start":{"date-parts":[[2022,8,12]],"date-time":"2022-08-12T00:00:00Z","timestamp":1660262400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"},{"start":{"date-parts":[[2022,8,12]],"date-time":"2022-08-12T00:00:00Z","timestamp":1660262400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Pharm Med"],"published-print":{"date-parts":[[2022,10]]},"DOI":"10.1007\/s40290-022-00442-y","type":"journal-article","created":{"date-parts":[[2022,8,12]],"date-time":"2022-08-12T03:25:06Z","timestamp":1660274706000},"page":"279-286","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Medical Research Charities and Biopharmaceutical Companies as Partners in Patient-Centred R&D"],"prefix":"10.1007","volume":"36","author":[{"given":"Tina","family":"Flatau","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Julie","family":"Greenfield","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Brian","family":"Dickie","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Oli","family":"Rayner","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Helen","family":"Matthews","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5311-6555","authenticated-orcid":false,"given":"John","family":"Wise","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2022,8,12]]},"reference":[{"key":"442_CR1","unstructured":"AMRC. 2021: Our sector\u2019s footprint. 2021. https:\/\/www.amrc.org.uk\/our-sectors-footprint-in-2020. Accessed 28 Feb 2022."},{"issue":"4","key":"442_CR2","doi-asserted-by":"publisher","first-page":"460","DOI":"10.1177\/2168479017696268","volume":"51","author":"D du Plessis","year":"2017","unstructured":"du Plessis D, Sake JK, Halling K, et al. Patient centricity and pharmaceutical companies: is it feasible? Ther Innov Regul Sci. 2017;51(4):460\u20137. https:\/\/doi.org\/10.1177\/2168479017696268.","journal-title":"Ther Innov Regul Sci."},{"key":"442_CR3","unstructured":"US Food and Drug Administration. FDA and European Medicines Agency Patient Engagement Cluster. 2018. https:\/\/www.fda.gov\/patients\/learn-about-fda-patient-engagement\/fda-and-european-medicines-agency-patient-engagement-cluster. Accessed 28 Feb 2022."},{"key":"442_CR4","unstructured":"Medicines and Healthcare products Regulatory Agency. Proposed Patient and Public Involvement Strategy 2020-25. 2021. https:\/\/www.gov.uk\/government\/consultations\/mhra-patient-involvement-strategy-consultation\/proposed-patient-and-public-involvement-strategy-2020-25 Accessed 26 Feb 2022."},{"issue":"12","key":"442_CR5","doi-asserted-by":"publisher","DOI":"10.1001\/jamanetworkopen.2020.31640","volume":"3","author":"LA Eberly","year":"2020","unstructured":"Eberly LA, Kallan MJ, Julien HM, et al. Patient characteristics associated with telemedicine access for primary and specialty ambulatory care during the COVID-19 pandemic. JAMA Netw Open. 2020;3(12): e2031640. https:\/\/doi.org\/10.1001\/jamanetworkopen.2020.31640.","journal-title":"JAMA Netw Open"},{"key":"442_CR6","unstructured":"US Food and Drug Administration. FDA\u2014Rare Diseases: Natural History Studies for Drug Development\u2014Draft Guidance for Industry. 2020 https:\/\/www.fda.gov\/regulatory-information\/search-fda-guidance-documents\/rare-diseases-natural-history-studies-drug-development. Accessed 26 Feb 2022."},{"key":"442_CR7","doi-asserted-by":"publisher","DOI":"10.1038\/sdata.2016.18","volume":"3","author":"M Wilkinson","year":"2016","unstructured":"Wilkinson M, Dumontier M, Aalbersberg I, et al. The FAIR Guiding Principles for scientific data management and stewardship. Sci Data. 2016;3: 160018. https:\/\/doi.org\/10.1038\/sdata.2016.18.","journal-title":"Sci Data"},{"key":"442_CR8","doi-asserted-by":"publisher","unstructured":"Wise, JCM, Grebe de Barron A, Splendiani A, et al. Implementation and relevance of FAIR data principles in biopharmaceutical R&D. Drug Discovery Today. 2019;24(4):933\u20138. https:\/\/doi.org\/10.1016\/j.drudis.2019.01.008.","DOI":"10.1016\/j.drudis.2019.01.008"},{"key":"442_CR9","unstructured":"National Institute for Health and Care Excellence. How NICE measures value for money in relation to public health interventions. 2013. https:\/\/www.nice.org.uk\/Media\/Default\/guidance\/LGB10-Briefing-20150126.pdf Accessed 26 Feb 2022."},{"key":"442_CR10","unstructured":"Department of Health Policy paper. Long Term Conditions Compendium of Information: Third Edition. 2012. https:\/\/www.gov.uk\/government\/publications\/long-term-conditions-compendium-of-information-third-edition. Accessed 26 Feb 2022."},{"key":"442_CR11","unstructured":"European Commission. Rare diseases. 2022. https:\/\/ec.europa.eu\/health\/non-communicable-diseases\/steering-group\/rare-diseases_en. Accessed 28 Feb 2022."},{"key":"442_CR12","unstructured":"International Federation of Pharmaceutical Manufacturers & Associations, Code of Practice. 2019. https:\/\/www.ifpma.org\/subtopics\/new-ifpma-code-of-practice-2019\/ Accessed 26 Feb 2022."},{"key":"442_CR13","doi-asserted-by":"publisher","first-page":"21","DOI":"10.1038\/s41531-018-0058-0","volume":"4","author":"C Marras","year":"2018","unstructured":"Marras C, Beck JC, Bower JH, et al. Prevalence of Parkinson\u2019s disease across North America. NPJ Parkinson\u2019s Disease. 2018;4:21. https:\/\/doi.org\/10.1038\/s41531-018-0058-0.","journal-title":"NPJ Parkinson's Disease."},{"issue":"supp1","key":"442_CR14","doi-asserted-by":"publisher","first-page":"s2","DOI":"10.1016\/j.parkreldis.2016.11.018","volume":"33","author":"KR Chaudhuri","year":"2016","unstructured":"Chaudhuri KR, Bhidayasiri R, van Laar T. Unmet needs in Parkinson\u2019s disease: New horizons in a changing landscape. Parkinson Relat Disord. 2016;33(supp1):s2\u20138. https:\/\/doi.org\/10.1016\/j.parkreldis.2016.11.018.","journal-title":"Parkinson Relat Disord."},{"issue":"3","key":"442_CR15","doi-asserted-by":"publisher","first-page":"307","DOI":"10.1111\/ejn.14175","volume":"49","author":"P Brundin","year":"2019","unstructured":"Brundin P, Wyse RK. The Linked Clinical Trials initiative (LCT) for Parkinson\u2019s disease. Eur J Neurosci. 2019;49(3):307\u201315. https:\/\/doi.org\/10.1111\/ejn.14175.","journal-title":"Eur J Neurosci"},{"key":"442_CR16","doi-asserted-by":"publisher","first-page":"38","DOI":"10.1186\/1750-1172-8-38","volume":"8","author":"P Giunti","year":"2013","unstructured":"Giunti P, Greenfield J, Stevenson AJ, et al. Impact of Friedreich\u2019s Ataxia on health-care resource utilization in the United Kingdom and Germany. Orphanet J Rare Dis. 2013;8:38. https:\/\/doi.org\/10.1186\/1750-1172-8-38.","journal-title":"Orphanet J Rare Dis"},{"key":"442_CR17","unstructured":"US Food and Drug Administration. CDER patient-focused drug development. 2021. https:\/\/www.fda.gov\/drugs\/development-approval-process-drugs\/cder-patient-focused-drug-development. Accessed 28 Feb 2022."},{"issue":"2","key":"442_CR18","doi-asserted-by":"publisher","first-page":"82","DOI":"10.4103\/2229-516X.157148","volume":"5","author":"R Mahajan","year":"2015","unstructured":"Mahajan R. Real world data: additional source for making clinical decisions. Int J Appl Basic Med Res. 2015;5(2):82. https:\/\/doi.org\/10.4103\/2229-516X.157148.","journal-title":"Int J Appl Basic Med Res"},{"key":"442_CR19","unstructured":"Telehealth Cos Are Seeing A Wave Of Consolidation. Here\u2019s what that means for the future of virtual care. CB Insights Newsletter. 2021. https:\/\/www.cbinsights.com\/research\/telehealth-healthcare-mergers-acquisitions-trends-analysis\/?utm_source=CB+Insights+Newsletter&utm_campaign=d7cdd34691-newsletter_general_Tues_20210518&utm_medium=email&utm_term=0_9dc0513989-d7cdd34691-88164293. Accessed 26 Feb 2022."},{"key":"442_CR20","unstructured":"How health care is turning into a consumer product. The Economist. 2022. https:\/\/www.economist.com\/business\/how-health-care-is-turning-into-a-consumer-product\/21807114. Accessed 26 Feb 2022."}],"container-title":["Pharmaceutical Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40290-022-00442-y.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40290-022-00442-y\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40290-022-00442-y.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,4,15]],"date-time":"2025-04-15T11:17:16Z","timestamp":1744715836000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40290-022-00442-y"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,8,12]]},"references-count":20,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2022,10]]}},"alternative-id":["442"],"URL":"https:\/\/doi.org\/10.1007\/s40290-022-00442-y","relation":{},"ISSN":["1178-2595","1179-1993"],"issn-type":[{"type":"print","value":"1178-2595"},{"type":"electronic","value":"1179-1993"}],"subject":[],"published":{"date-parts":[[2022,8,12]]},"assertion":[{"value":"7 July 2022","order":1,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"12 August 2022","order":2,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"Action Duchenne, Ataxia UK, the Motor Neurone Disease Association and Cure Parkinson\u2019s are all members of the Pistoia Alliance.\u00a0The Pistoia Alliance provided funding for a consultant, John Wise, as a coordination resource for the questionnaire and joint authorship of this paper.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Funding"}},{"value":"The authors listed below are volunteers or employees of charities consulted in this manuscript, they declare no affiliations with, or direct or indirect financial interest in the subject matter of this manuscript: Tina Flatau, Julie Greenfield, Brian Dickie, Oli Rayner, Helen Matthews.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflicts of interest"}},{"value":"Not applicable.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval"}},{"value":"Not applicable.","order":5,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent to participate"}},{"value":"Not applicable.","order":6,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"Not applicable.","order":7,"name":"Ethics","group":{"name":"EthicsHeading","label":"Availability of data and material"}},{"value":"Not applicable.","order":8,"name":"Ethics","group":{"name":"EthicsHeading","label":"Code availability"}},{"value":"All authors contributed to the questionnaire conception and design. Material preparation, collection of inputs and analysis were performed by John Wise and Tina Flatau. The first draft of the manuscript was written by Tina Flatau and all authors commented on previous versions of the manuscript. All authors read and approved the final manuscript. The authors confirm that they have all agreed with the content and gave explicit consent to submit.","order":9,"name":"Ethics","group":{"name":"EthicsHeading","label":"Authors' contributions"}}]}}