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Patient-based evidence: its role in decision making on end-of-life, orphan, and ultra-orphan medicines
Part of:
Special Collection - Methods
Published online by Cambridge University Press: 11 April 2023
Abstract
Objectives
The Scottish Medicines Consortium (SMC) conducts early health technology assessment (HTA) of new medicines on behalf of NHSScotland. Assessment of end-of-life (EoL), orphan, and ultra-orphan medicines includes a process to gather evidence from patients and carers during Patient and Clinician Engagement (PACE) meetings. The output of PACE meetings is a consensus statement describing the medicine’s added value from the perspective of patients/carers and clinicians. The PACE statement is used by SMC committee members in decision making. This study compared how PACE participants and SMC committee members rate the importance of information in PACE statements for these medicines.
Methods
A survey was undertaken of patient group (PG) representatives and clinicians who participated in PACE meetings, and SMC committee members.
Results
PACE participants who responded (26 PG representatives and 14 clinicians) rated health benefits and ability to take part in normal life as important/very important. Convenience of administration and treatment choice received the lowest rating. Hope for the future received the most diverse response. PACE participants generally rated the importance of quality of life themes higher than committee members (n = 20) but the rank order was similar. Differences between the proportion of PACE participants and committee members who rated themes as important/very important were greatest for treatment choice and hope for the future.
Conclusions
In general, PACE themes and subthemes that were rated highly by PACE participants were also considered important by SMC committee members, indicating that information captured during PACE meetings is relevant when making decisions on EoL, orphan, and ultra-orphan medicines.
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- Method
- Information
- Copyright
- © Crown Copyright - Healthcare Improvement Scotland, 2023. Published by Cambridge University Press
References
Morrell, L, Wordsworth, S, Fu, H, Rees, S, Barker, R. Cancer drug funding decisions in Scotland: Impact of new end-of-life, orphan and ultra-orphan processes. BMC Health Serv Res. 2017;17(1):613.Google ScholarPubMed
Scottish Medicines Consortium [Internet]. Patient and clinician engagement (PACE) meetings overview; 2022. Available from: https://www.scottishmedicines.org.uk/how-we-decide/pace/ (accessed 2022 November 15).Google Scholar
Scottish Medicines Consortium [Internet]. Scottish Medicines Consortium Update Report; 2016. Available from: https://www.scottishmedicines.org.uk/media/2728/smc-update-report.pdf (accessed 2022 November 15)Google Scholar
Angelis, A, Kanavos, P. Multiple criteria decision analysis (MCDA) for evaluating new medicines in health technology assessment and beyond: The advance value framework. Soc Sci Med. 2017;188:137–156.CrossRefGoogle ScholarPubMed
Gauvin, FP, Abelson, J, Giacomini, M, Eyles, J, Lavis, JN. “It all depends”: Conceptualizing public involvement in the context of health technology assessment agencies. Soc Sci Med. 2010;70:1518–1526.CrossRefGoogle ScholarPubMed
Weeks, L, Polisena, J, Scott, AM, et al. Evaluation of patient and public involvement initiatives in health technology assessment: A survey of international agencies. Int J Technol Assess Health Care. 2017;33(6):715–723.CrossRefGoogle ScholarPubMed
Berglas, S, Jutai, L, Mackean, G, Weeks, L. Patients’ perspectives can be integrated in health technology assessments: An exploratory analysis of CADTH Common Drug Review. Res Involv Engagem. 2016;2(1):21.CrossRefGoogle ScholarPubMed
Dipankui, MT, Gagnon, MP, Desmartis, M, et al. Evaluation of patient involvement in a health technology assessment. Int J Technol Assess Health Care. 2015;31(3):166–170.CrossRefGoogle Scholar
Hashem, F, Calnan, MW, Brown, PR. Decision making in NICE single technological appraisals: How does NICE incorporate patient perspectives? Health Expect. 2017;21(1):128–137.CrossRefGoogle ScholarPubMed
Jones, J, Harkess-Murphy, E, Lockhart, L, et al. Added value of end of life and orphan medicines: A thematic analysis from the patient/carer perspective. Poster presented at: Health Technology Assessment International (HTAi) annual meeting; 2016 May 10–14; Tokyo, Japan.Google Scholar
Nierop-van Baalen, C, Grypdonck, M, Van Hecke, A, Verhaeghe, S. Health professionals’ dealing with hope in palliative patients with cancer, an explorative qualitative research. Eur J Cancer Care (Engl). 2019;28:e12889.Google ScholarPubMed
Nierop-van Baalen, C, Grypdonck, M, Van Hecke, A, Verhaeghe, S. Hope dies last … A qualitative study into the meaning of hope for people with cancer in the palliative phase. Eur J Cancer Care (Engl). 2016;25:570–579.CrossRefGoogle Scholar
Booker, QS, Austin, JD, Balasubramanian, BA. Survey strategies to increase participant response rates in primary care research studies. Fam Pract. 2021;38:699–702.Google ScholarPubMed
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