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Physicians' attitudes towards accelerated access to medicines

Published online by Cambridge University Press:  31 October 2019

Jessica Pace Open the ORCID record for Jessica Pace [Opens in a new window] ,
Ian Kerridge ,
Sallie Pearson  and
Wendy Lipworth
Jessica Pace*
Affiliation:
Sydney Health Ethics, University of Sydney, Medical Foundation Building (K25), Sydney, New South Wales, Australia
Ian Kerridge
Affiliation:
Sydney Health Ethics, University of Sydney, Medical Foundation Building (K25), Sydney, New South Wales, Australia
Sallie Pearson
Affiliation:
Centre for Big Data Research in Healthcare, University of NSW, Sydney, Australia
Wendy Lipworth
Affiliation:
Sydney Health Ethics, University of Sydney, Medical Foundation Building (K25), Sydney, New South Wales, Australia
*
*Corresponding author. Email: [email protected]
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Abstract

In recent years, a variety of ‘accelerated access’ schemes have been introduced by pharmaceutical regulators and funders globally. These schemes aim to overcome perceived regulatory and reimbursement barriers to accessing medicines – particularly for patients with limited time or therapeutic options. However, patient access to approved medicines is mediated by a number of third parties including regulators and payers, and physicians who act both as gatekeepers and guides to prescribed medications. It is therefore essential to know how physicians think about accelerated access as they are responsible for advising patients on and prescribing medicines made available via these pathways. We conducted semi-structured interviews with 18 Australian physicians focusing on their attitudes towards accelerated access. We identified three ‘archetypes’ of physicians: ‘confident accelerators’, ‘cautious accelerators’, and ‘decelerators’. Although all acknowledged the potential risks and benefits of accelerated access, they disagreed on their magnitude and extent and how they should be balanced in both policy formation and clinical practice. Overall, our results illustrate the diversity of clinical opinions in this area and the importance of monitoring both the prescribing and clinical outcomes that result from accelerated access programmes to ensure that these are both clinically and morally acceptable.

Keywords

Accelerated accessconditional registrationcoverage with evidence developmentpharmaceutical fundingpharmaceutical regulation
Type
Article
Information
Health Economics, Policy and Law , Volume 16 , Issue 2 , April 2021 , pp. 154 - 169
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Copyright
Copyright © Cambridge University Press 2019

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References

Australian Government Department of Health (2019) TGA and PBAC Parallel Process and Requirements. Last modified 14 September 2018. http://www.pbs.gov.au/info/publication/factsheets/shared/tga-pbac-parallel-process.Google Scholar
Banzi, R, Gerardi, C, Bertele, V and Garattini, S (2015) Approvals of drugs with uncertain benefit-risk profiles in Europe. European Journal of Internal Medicine 26, 572–584. https://doi.org/10.1016/j.ejim.2015.08.008.CrossRefGoogle ScholarPubMed
Bateman-House, A and Robertson, CT (2018) The Federal Right to Try Act of 2017 – a wrong turn for access to investigational drugs and the path forward. JAMA Internal Medicine 178, 321–322. https://doi.org/10.1001/jamainternmed.2017.8167.CrossRefGoogle ScholarPubMed
Bateman-House, A, Kimberly, L, Redman, B, Dubler, N and Caplan, A (2015) Right-to-try laws: hope, hype, and unintended consequences. Annals of Internal Medicine 163, 796. https://doi.org/10.7326/M15-0148.CrossRefGoogle ScholarPubMed
Bishop, D and Lexchin, J (2013) Politics and its intersection with coverage with evidence development: a qualitative analysis from expert interviews. BMC Health Services Research 13, 88–88. https://doi.org/10.1186/1472-6963-13-88.CrossRefGoogle ScholarPubMed
Boon, WPC, Moors, EHM, Meijer, A and Schellekens, H (2010) Conditional approval and approval under exceptional circumstances as regulatory instruments for stimulating responsible drug innovation in Europe. Clinical Pharmacology & Therapeutics 88, 848–853. https://doi.org/10.1038/clpt.2010.207.CrossRefGoogle ScholarPubMed
Brugger, U, Horisberger, B, Ruckstuhl, A, Plessow, R, Eichler, K and Gratwohl, A (2015) Health technology assessment in Switzerland: a descriptive analysis of ‘coverage with evidence development’ decisions from 1996–2013. BMJ Open 5(3), e007021. https://doi.org/10.1136/bmjopen-2014-007021.CrossRefGoogle Scholar
Charmaz, K (2006) Constructing Grounded Theory: a Practical Guide Through Qualitative Analysis. London: Sage.Google Scholar
Chim, L, Salkeld, G, Kelly, PJ, Lipworth, W, Hughes, DA and Stockler, MR (2017) Societal perspective on access to publicly subsidised medicines: a cross sectional survey of 3080 adults in Australia. PLOS One 12, e0172971. https://doi.org/10.1371/journal.pone.0172971.CrossRefGoogle ScholarPubMed
Chim, L, Salkeld, G, Kelly, PJ, Lipworth, W, Hughes, DA and Stockler, MR (2019) Community views on factors affecting medicines resource allocation: cross-sectional survey of 3080 adults in Australia. Australian Health Review 43, 254–260. https://doi.org/10.1071/AH16209.CrossRefGoogle ScholarPubMed
Downing, NS, Aminawung, JA, Shah, ND, Krumholz, HM and Ross, JS (2014) Clinical trial evidence supporting FDA approval of novel therapeutic agents, 2005–2012. JAMA 311, 368–377. https://doi.org/10.1001/jama.2013.282034.CrossRefGoogle ScholarPubMed
Downing, NS, Shah, ND, Aminawung, JA, Pease, AM, Zeitoun, JD, Krumholz, HM and Ross, JS (2017) Postmarket safety events among novel therapeutics approved by the US Food and Drug Administration between 2001 and 2010. JAMA 317, 1854–1863. https://doi.org/10.1001/jama.2017.5150.CrossRefGoogle ScholarPubMed
Food and Drug Administration (2016) Accelerated approval program. Last modified 10 March 2016. https://www.fda.gov/drugs/information-healthcare-professionals-drugs/accelerated-approval-program.Google Scholar
Frank, C, Himmelstein, DU, Woolhandler, S, Bor, DH, Wolfe, SM, Heymann, O, Zallman, L and Lasser, KE (2014) Era of faster FDA drug approval has also seen increased black box warnings and market withdrawals. Health Affairs 33, 1453–1459. https://doi.org/10.1377/hlthaff.2014.0122.CrossRefGoogle ScholarPubMed
Gallego, G, Taylor, SJ and Brien, JE (2007) Provision of pharmaceuticals in Australian hospitals: equity of access? Pharmacy World & Science 29, 47–50. https://doi.org/10.1007/s11096-006-9066-y.CrossRefGoogle ScholarPubMed
Garrison, LP Jr, Towse, A, Briggs, A, de Pouvourville, G, Grueger, J, Mohr, PE, Severens, JL (Hans), Siviero, P and Sleeper, M (2013) Performance-based risk-sharing arrangements – good practices for design, implementation, and evaluation: report of the ISPOR good practices for performance-based risk-sharing arrangements task force. Value in Health 16, 703–719. https://doi/org/10.1016/j.jval.2013.04.011.CrossRefGoogle ScholarPubMed
Gyawali, B, Hey, SP and Kesselheim, AS (2019) Assessment of the clinical benefit of cancer drugs receiving accelerated approval. JAMA Internal Medicine 179(7), 906–913. https://doi.org/10.1001/jamainternmed.2019.0462.CrossRefGoogle ScholarPubMed
Hutton, J, Trueman, P and Henshall, C (2007) Coverage with evidence development: an examination of conceptual and policy issues. International Journal of Technology Assessment in Health Care 23, 425–432. https://doi.org/10.1017/S0266462307070651.CrossRefGoogle ScholarPubMed
Jaroslawski, S and Toumi, M (2011) Market access agreements for pharmaceuticals in Europe: diversity of approaches and underlying concepts. BMC Health Services Research 11, 259. https://doi.org/10.1186/1472-6963-11-259.CrossRefGoogle ScholarPubMed
Levin, L, Goeree, R, Levine, M, Krahn, M, Easty, T, Brown, A and Henry, D (2011) Coverage with evidence development: the Ontario experience. International Journal of Technology Assessment in Health Care 27, 159–168. https://doi.org/10.1017/S0266462311000018.CrossRefGoogle ScholarPubMed
Lexchin, J (2015) Post-market safety warnings for drugs approved in Canada under the notice of compliance with conditions policy. British Journal of Clinical Pharmacology 79, 847–859. https://doi.org/10.1111/bcp.12552.CrossRefGoogle ScholarPubMed
Linley, WG and Hughes, DA (2013a) Decision-makers’ preferences for approving new medicines in Wales: a discrete-choice experiment with assessment of external validity. PharmacoEconomics 31, 345–355. https://doi.org/10.1007/s40273-013-0030-0.CrossRefGoogle Scholar
Linley, WG and Hughes, DA (2013b) Societal views on nice, cancer drugs fund and value-based pricing criteria for prioritising medicines: a cross-sectional survey of 4118 adults in Great Britain. Health Economics 22, 948–964. https://doi.org/10.1002/hec.2872.CrossRefGoogle Scholar
Lu, CY, Lupton, C, Rakowsky, S, Babar, ZUD, Ross-Degnan, D and Wagner, AK (2015) Patient access schemes in Asia-pacific markets: current experience and future potential. Journal of Pharmaceutical Policy and Practice 8, 6–6. https://doi.org/10.1186/s40545-014-0019-x.CrossRefGoogle ScholarPubMed
Malik, NN (2016) Pay-for-performance pricing for a breakthrough heart drug: learnings for cell and gene therapies. Regenerative Medicine 11, 225–227. https://doi.org/10.2217/rme-2016-0014.CrossRefGoogle ScholarPubMed
McHugh, N, Baker, R, Mason, H, Williamson, L, Exel, J, Deogaonkar, R, Collins, M and Donaldson, C (2015) Extending life for people with a terminal illness: a moral right and an expensive death? Exploring societal perspectives. BMC Medical Ethics 16, 14. https://doi.org/10.1186/s12910-015-0008-x.CrossRefGoogle Scholar
McHugh, N, van Exel, J, Mason, H, Godwin, J, Collins, M, Donaldson, C and Baker, R (2018) Are life-extending treatments for terminal illnesses a special case? Exploring choices and societal viewpoints. Social Science & Medicine 198, 61–69. https://doi.org/10.1016/j.socscimed.2017.12.019.CrossRefGoogle ScholarPubMed
Mohr, PE and Tunis, SR (2010) Access with evidence development: the US experience. PharmacoEconomics 28, 153–162. https://doi.org/10.2165/11531050-000000000-00000.CrossRefGoogle ScholarPubMed
Morse, JM (1994) ‘Emerging from the data’: The cognitive processes of analysis in qualitative inquiry. In Morse, JM (ed.), Critical Issues in Qualitative Research Methods. Thousand Oaks, CA: Sage, 23–46.Google Scholar
Naci, H, Smalley, KR and Kesselheim, AS (2017) Characteristics of preapproval and postapproval studies for drugs granted accelerated approval by the US Food and Drug Administration. JAMA 318, 626–636. https://doi.org/10.1001/jama.2017.9415.CrossRefGoogle ScholarPubMed
Pace, J, Ghinea, N, Kerridge, I and Lipworth, W (2017a) Accelerated access to medicines: an ethical analysis. Therapeutic Innovation & Regulatory Science 51, 157–163. https://doi.org/10.1177/2168479016674043.CrossRefGoogle Scholar
Pace, J, Ghinea, N, Kerridge, I and Lipworth, W (2017b) Demands for access to new therapies: are there alternatives to accelerated access? BMJ 359, j4494. https://doi.org/10.1136/bmj.j4494.CrossRefGoogle Scholar
Pace, J, Ghinea, N, Kerridge, I and Lipworth, W (2018) An ethical framework for the creation, governance and evaluation of accelerated access programs. Health Policy 122, 984–990. https://doi.org/10.1016/j.healthpol.2018.07.014.CrossRefGoogle ScholarPubMed
Pauwels, K, Huys, I, Vogler, S, Casteels, M and Simoens, S (2017) Managed entry agreements for oncology drugs: lessons from the European experience to inform the future. Frontiers in Pharmacology 8, 171. https://doi.org/10.3389/fphar.2017.00171.CrossRefGoogle ScholarPubMed
Persson, U and Norlin, JM (2018) Multi-indication and combination pricing and reimbursement of pharmaceuticals: opportunities for improved health care through faster uptake of new innovations. Applied Health Economics and Health Policy 16, 157–165. https://doi.org/10.1007/s40258-018-0377-7.CrossRefGoogle ScholarPubMed
Pickin, M, Cooper, CL, Chater, T, O'Hagan, A, Abrams, KR, Cooper, NJ, Boggild, M, Palace, J, Ebers, G, Chilcott, JB, Tappenden, P and Nicholl, J (2009) The multiple sclerosis risk sharing scheme monitoring study – early results and lessons for the future. BMC Neurology 9, 1. https://doi.org/10.1186/1471-2377-9-1.CrossRefGoogle ScholarPubMed
Prescrire (2015) ‘Adaptive Licensing’ or ‘Adaptive Pathways’: Deregulation Under the Guise of Earlier Access. Brussels: Health Action International (HAI), The International Society of Drug Bulletins (ISDB), The Medicines in Europe Forum (MiEF), The Mario Negri Institute for Pharmacological Research, The Nordic Cochrane Centre, WEMOS.Google Scholar
Therapeutic Goods Administration (2017) Submissions received and TGA response: Changes to accessing unapproved therapeutic goods through the Authorised Prescriber (AP) and Special Access Schemes (SAS). Last modified 9 October 20187. https://www.tga.gov.au/submissions-received-and-tga-response-changes-accessing-unapproved-therapeutic-goods-through-authorised-prescriber-ap-and-special-access-schemes-sas.Google Scholar
Therapeutic Goods Administration (2018) Provisional approval pathway: Prescription medicines. Last modified 20 March 2018. https://www.tga.gov.au/provisional-approval-pathway-prescription-medicines.Google Scholar
Therapeutic Goods Administration (2019) First provisional approval. Last modified 17 July 2019. https://www.tga.gov.au/first-provisional-approval.Google Scholar
van de Vooren, K, Curto, A, Freemantle, N and Garattini, L (2015) Market-access agreements for anti-cancer drugs. Journal of the Royal Society of Medicine 108, 166–170. https://doi.org/10.1177/0141076814559626.CrossRefGoogle ScholarPubMed
Vitry, A and Roughead, E (2014) Managed entry agreements for pharmaceuticals in Australia. Health Policy 117, 345–352. https://doi.org/10.1016/j.healthpol.2014.05.005.CrossRefGoogle ScholarPubMed
Vitry, A, Nguyen, T, Entwistle, V and Roughead, E (2015) Regulatory withdrawal of medicines marketed with uncertain benefits: the bevacizumab case study. Journal of Pharmaceutical Policy and Practice 8, 25. https://doi.org/10.1186/s40545-015-0046-2.CrossRefGoogle ScholarPubMed
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