Rare Diseases

PUBLICATIONS

What will the crowd fund? Preferences of prospective donors for drug development fundraising campaigns
Nick Dragojlovic, Larry D. Lynd
Drug Discov Today. 2016 Dec;21(12):1863-1868. doi: 10.1016/j.drudis.2016.07.002. Epub 2016 Jul 12.

A decision tool to guide the ethics review of a challenging breed of emerging genomic projects
Joly Y, So D, Osien G, Crimi L, Bobrow M, Chalmers D, Wallace SE, Zeps N, Knoppers B
Eur J Hum Genet. 2016 Aug;24(8):1099-103. doi: 10.1038/ejhg.2015.279. Epub 2016 Jan 20.

Fair shares and sharing fairly: a Survey of public views on open science, informed consent and participatory research in biobanking
Yann Joly, Gratien Dalpé, Derek So, Stanislav Birko
PLoS One. 2015 Jul 8;10(7):e0129893. doi: 10.1371/journal.pone.0129893. eCollection 2015.

Challenges in Measuring the Societal Value of Orphan Drugs: Insights from a Canadian Stated Preference Survey
Nick Dragojlovic, Shirin Rizzardo, Nick Bansback, Craig Mitton, Carlo A. Marra, Larry D. Lynd
Patient. 2015 Feb;8(1):93-101. doi: 10.1007/s40271-014-0109-5.

Why orphan drug coverage reimbursement decision-making needs patient and public involvement
Conor M. Douglas, Elizabeth Wilcox, Michael Burgess, Larry D. Lynd
Health Policy. 2015 May;119(5):588-96. doi: 10.1016/j.healthpol.2015.01.009. Epub 2015 Jan 19.

Toward a functional definition of a “rare disease” for regulatory authorities and funding agencies
Clarke JT, Coyle D , Evans G, Martin J, Winquist E
Value Health. 2014 Dec;17(8):757-61. doi: 10.1016/j.jval.2014.08.2672. Epub 2014 Oct 18. PMID: 25498770

Crowdfunding drug development: the state of play in oncology and rare diseases
Nick Dragojlovic, Larry D. Lynd
Drug Discov Today. 2014 Nov;19(11):1775-1780. doi: 10.1016/j.drudis.2014.06.019. Epub 2014 Jun 25. PMID: 24973645.

Opportunity cost of funding drugs for rare diseases: the cost-effectiveness of eculizumab in paroxysmal nocturnal hemoglobinuria
Coyle D, Cheung MC, Evans GA
Med Decis Making. 2014 Nov;34(8):1016-29. doi: 10.1177/0272989X14539731. Epub 2014 Jul 2. PMID:24990825

Returning incidental findings from genetic research to children: views of parents of children affected by rare diseases
Kleiderman E, Knoppers BM, Fernandez CV, Boycott KM, Ouellette G, Wong-Rieger D, Adam S, Richer J, Avard D
J Med Ethics. 2014 Oct;40(10):691-6. doi: 10.1136/medethics-2013-101648. Epub 2013 Dec 19. PMID: 24356209

Application of a policy framework for the public funding of drugs for rare diseases
Winquist E, Coyle D, Clarke JT, Evans GA, Seager C, Chan W, Martin J
J Gen Intern Med. 2014 Aug;29 Suppl 3:S774-9. doi: 10.1007/s11606-014-2885-y. PMID: 25029973

Clinical trial transparency and orphan drug development: recent trends in data sharing by the pharmaceutical industry
Derek So, Yann Joly, Bartha Maria Knoppers
Public Health Genomics. 2013;16(6):322-35. doi: 10.1159/000355941. Epub 2014 Feb 3.

‘Innovation’ in health care coverage decisions: all talk and no substance?
Bryan S., Lee H., Mitton C.
Journal of Health Services Research & Policy
January 2013; 18(1):57-60. PubMed PMID: 22977184. DOI: 10.1258/jhsrp.2012.012031

CONFERENCE PROCEEDINGS

Engaging the Canadian public on reimbursement decision-making for drugs for rare diseases: A national online survey
Polisena J, Burgess M, Mitton C, Lynd LD
Poster Presentation – 38th Annual Meeting of the Society for Medical Decision Making, Vancouver, BC, October 23-26, 2016.

Evaluating the validity of parental reports of healthcare utilization for children with suspected genetic disorders of unknown cause
Nick Dragojlovic, Ellen Kim, Alsion M. Elliott, Jan M. Friedman, Larry D. Lynd
Poster Presentation – 38th Annual Meeting of the Society for Medical Decision Making, Vancouver, BC, October 23-26, 2016.

Predictors of anxiety and depression in caregivers of children with chronic conditions of unknown but suspected genetic origin
Dragojlovic, N., Elliott, A.M., Friedman, J.M., & Lynd, L.D.
Poster Presentation – 2016 American Society of Human Genetics Meeting, Vancouver, BC, October 20, 2016.

Screening for treatable inborn errors of metabolism in 498 intellectual developmental disorder patients
Ghani, A., Dragojlovic, N., Birch, P., Salvarinova, R., Horvath, G., Generaux, D., Sayson, B., Lafek, M., Blydt-Hansen, T., Connolly, M., Demos, M., Lehman, A., Patel, M., Armstrong, L., Van Allen, M., McKinnon, M., Nelson, T., Eydoux, P., Matthews, A., Tarailo-Graovac, M., Vallance, H., Sinclair, G., Lynd, L.D., Stockler, S., and van Karnebeek, C.
Poster Presentation – 2016 American Society of Human Genetics Meeting, Vancouver, BC, October 19, 2016. (Selected for Special Jury Award).

Validity of parental reports of healthcare utilization for children with suspected disorders of unknown cause
Dragojlovic, N., Elliott, A.M., Friedman, J.M., & Lynd, L.D.
Poster Presentation – 2016 Vancouver Health Economics Methodology meeting, Vancouver, BC, June 10, 2016.

Preferences for Donating Money to Support Drug Development Research Projects in a Sample of Canadian and U.S. Adults
Nick Dragojlovic & Larry Lynd
Oral Presentation – 2016 Canadian Society for Pharmaceutical Sciences Annual Symposium, Richmond, BC, June 3, 2016.

Protecting Privacy through Controlled Access: The Successful Five-Year Experience of the ICGC Data Access Compliance Office
Emilie de Vries-Séguin, Derek So, Bartha M. Knoppers, Yann Joly
Poster Presentation – Canadian GE3LS and Health Services & Policy Research Conference: Integrated Approaches to Opportunities and Challenges in the Genomics Era, Vancouver, BC, Canada, April 18-21, 2015.

Crowdfunding drug development: the state of play in oncology and rare diseases
Nick Dragojlovic & Larry Lynd
Poster Presentation – 2014 Vancouver Health Economics Methodology Meeting, Vancouver, BC, Canada, June 27, 2014.

Consideration for rare diseases in drug reimbursement decision-making
Presenting author: Sheena Gosain. Co-authors: Doug Coyle; Tammy Clifford; Barry Jones
Oral Presentation – 2016 Health Technology Assessment International (HTAi) Conference, Washington, DC, June 16 -18, 2014.

Does misinformation matter? Exploring the roles of technical and conceptual inaccuracies in a deliberative public engagement on biobanks
Elizabeth Wilcox & Michael Burgess
Paper Presentation – 9th International Conference on Interdisciplinary Social Sciences. June 11-13, 2014. Vancouver, BC.

How Canadians Value Rare Diseases Given Their Opportunity Cost?
Shirin Rizzardo, Nick Bansback, Craig Mitton, Carlo Marra, Larry Lynd
Podium Presentation – ISPOR 19th Annual International Meeting, Montreal, Quebec, May 31-June 4, 2014
Research abstracts published in Value in Health; Volume 17, Number 3, May 2014, ISSN 1098-3015; p. 10, PP3.

Values of the Canadian Public Towards Pharmaceutical Reimbursement Decisions
Shirin Rizzardo, Nick Bansback, Craig Mitton, Larry Lynd
Poster Presentation – ISPOR 19th Annual International Meeting, Montreal, Quebec, May 31-June 4, 2014
Research abstracts published in Value in Health; Volume 17, Number 3, May 2014, ISSN 1098-3015; p. 240, PSY59.

Consideration for Rare Diseases in Drug Reimbursement Decision-Making
Sheena Gosain, Doug Coyle, Tammy Clifford, Barry Jones
Poster Presentation – ISPOR 19th Annual International Meeting, Montreal, Quebec, May 31-June 4, 2014
Research abstracts published in Value in Health; Volume 17, Number 3, May 2014, ISSN 1098-3015; p. 242, PSY73

Engaging Publics in Reimbursement Decision-Making for Drugs for Rare Diseases
Elizabeth Wilcox, Craig Mitton, Larry Lynd
Poster presentation – 25th Annual Canadian Bioethics Society Conference, Vancouver, BC, Canada, May 28-31, 2014

Consideration for Rare Diseases in Drug Reimbursement Decision-Making
Sheena Gosain, Doug Coyle, Tammy Clifford, Barry Jones
Poster Presentation – 2014 CADTH Symposium, Gatineau, QC, Canada, April 6-8, 2014

Incorporating Societal Values into Healthcare Decision-Making: Engaging Publics in Reimbursement Decision-Making for Drugs for Rare Diseases
Elizabeth Wilcox, Craig Mitton, Larry Lynd
Oral presentation – 2014 CADTH Symposium, Gatineau, QC, Canada, April 6-8, 2014

Rare Diseases Equity and Funding; A Canadian Perspective
Shirin Rizzardo, Nick Bansback, Craig Mitton, Carlo Marra, Larry Lynd
Poster presentation – 2014 CADTH Symposium, Gatineau, QC, Canada, April 6-8, 2014

Canadian Societal Views and Trade-Offs for Rare Disease Treatment
Shirin Rizzardo, Nick Bansback, Craig Mitton, Carlo Marra, Larry Lynd
Oral Presentation – 2014 CADTH Symposium, Gatineau, QC, Canada, April 6-8, 2014

Resource Allocation for the Treatment of Rare Diseases
Sheena Gosain
2013 Canadian Society for Epidemiology & Biostatistics (CSEB) Conference, abstract selected for publication
Journal of Epidemiology & Community Health
October 2013; 67:e2 doi:10.1136/jech-2013-203098.20

Canadian Values, Attitudes and Willingness to Pay for Expensive Medications for Rare Diseases
Shirin Rizzardo and Larry Lynd
Poster Presentation – 16th Canadian Society for Pharmaceutical Sciences (CSPS) Annual Symposium, June 11-14, 2013, Vancouver, BC
Conference proceedings published in Journal of Pharmacy & Pharmaceutical Sciences; Vol 16, No 3 (2013)

Public Engagement: Understanding and Accounting for Public Values and Perspectives in Reimbursement Decision-Making Related to Expensive Drugs for Rare Diseases
Elizabeth Wilcox, Craig Mitton, Larry Lynd
Poster presentation – 2013 Vancouver Health Economics Methodology Meeting, Vancouver, BC, Canada, June 7, 2013

Canadian Values, Attitudes and Willingness to Pay for Expensive Medications for Rare Diseases

Shirin Rizzardo and Larry Lynd
Poster Presentation – International Rare Diseases Research Consortium (IRDiRC), Dublin, April 16-17, 2013