Patient-based evidence: its role in decision making on end-of-life, orphan, and ultra-orphan medicines.

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.

A qualitative study of the relationship between the Scottish Medicines Consortium and their clinical experts.

RATIONALE, AIMS AND OBJECTIVES: Internationally, health technology assessments (HTAs) are ubiquitous drivers to health policy. Within Scotland, the Scottish Medicines Consortium undertakes the medicine review process. Input from clinical experts, involved in frontline care, is an integral component of the assessment process. This paper explores the relationship between the clinical experts and the HTA agency within Scotland to better understand what motivates expert clinicians to devote their time to the medicine review process with no remuneration. METHODS: Twenty-seven clinical experts from 16 different clinical specialties took part in one-to-one interviews at their place of work between October 2011 and March 2012. Data analysis was inductive and comprised the organization of data into a framework and a subsequent thematic analysis. RESULTS: Three distinct themes were identified: (1) recruitment, which identified two types of explanations for the experts' appointment: external justification (nominated by another) and internal justification (being recognized as an expert); (2) flexibility of the procedures, with experts able to determine their own response style and negotiate timelines; (3) health care systems, demonstrating that their affiliation to the health system underpinned the relationship and their motivation to be clinical experts. CONCLUSIONS: The findings of this study provide insight into the elements important to clinicians who voluntarily contribute to HTA processes. Examination of these elements in the context of the organizational citizenship behavior literature provides a foundation on which to improve understanding of this relationship and sustain and improve clinical expert participation in an increasingly intensified clinical environment and within cash-limited HTA systems.

An investigation into the effect of advice from the Scottish Medicines Consortium on the use of medicines in Scotland's Health Service.

AIMS: The aims of the study were to determine the effect of advice from the Scottish Medicines Consortium (SMC) on the use of medicines within Scotland's National Health Service (NHS) and generate hypotheses that may explain differences in the impact of advice on the use of individual medicines. METHODS: A retrospective analysis of medicine advice issued between January 2002 and December 2005 was performed. The inclusion criterion was medicines with a 'not recommended for use' decision (NRD) from the SMC (57 out of 207 medicines submitted). The exclusion criteria were medicines used predominately in secondary care and medicines with multiple indications. In total, 20 medicines fulfilled these criteria. The volume of prescribing was measured by each medicine's gross ingredient cost to the prescribing budget. RESULTS: Before the SMC published advice there was use, though limited, of all 20 medicines. After an NRD, the pattern of use was variable, with the use of some medicines stabilizing or declining but others increasing. We identified factors to help explain unexpected use in some cases. These included delays between medicine launch and initial SMC advice, the publication of conflicting advice from different national bodies and failure to engage with relevant clinical experts early in the medicine review process. CONCLUSIONS: This study demonstrates the complex relationship between advice following health technology assessment and change in clinical practice. When this study was done there were significant limitations in the collection of prescribing data within the NHS, which recent changes promise to improve.