Role of feasibility and pilot studies in randomised controlled trials: a cross-sectional study.

OBJECTIVES: To assess the value of pilot and feasibility studies to randomised controlled trials (RCTs) funded by the National Institute for Health Research (NIHR) Health Technology Assessment (HTA) programme. To explore the methodological components of pilot/feasibility studies and how they inform full RCTs. STUDY DESIGN: Cross-sectional study. SETTING: Both groups included NIHR HTA programme funded studies in the period 1 January 2010-31 December 2014 (decision date). Group 1: stand-alone pilot/feasibility studies published in the HTA Journal or accepted for publication. Group 2: all funded RCT applications funded by the HTA programme, including reference to an internal and/or external pilot/feasibility study. The methodological components were assessed using an adapted framework from a previous study. MAIN OUTCOME MEASURES: The proportion of stand-alone pilot and feasibility studies which recommended proceeding to full trial and what study elements were assessed. The proportion of 'HTA funded' trials which used internal and external pilot and feasibility studies to inform the design of the trial. RESULTS: Group 1 identified 15 stand-alone pilot/feasibility studies. Study elements most commonly assessed were testing recruitment (100% in both groups), feasibility (83%, 100%) and suggestions for further study/investigation (83%, 100%). Group 2 identified 161 'HTA funded' applications: 59 cited an external pilot/feasibility study where testing recruitment (50%, 73%) and feasibility (42%, 73%) were the most commonly reported study elements: 92 reported an internal pilot/feasibility study where testing recruitment (93%, 100%) and feasibility (44%, 92%) were the most common study elements reported. CONCLUSIONS: 'HTA funded' research which includes pilot and feasibility studies assesses a variety of study elements. Pilot and feasibility studies serve an important role when determining the most appropriate trial design. However, how they are reported and in what context requires caution when interpreting the findings and delivering a definitive trial.

Time to publication for NIHR HTA programme-funded research: a cohort study.

OBJECTIVE: To assess the time to publication of primary research and evidence syntheses funded by the National Institute for Health Research (NIHR) Health Technology Assessment (HTA) Programme published as a monograph in Health Technology Assessment and as a journal article in the wider biomedical literature. STUDY DESIGN: Retrospective cohort study. SETTING: Primary research and evidence synthesis projects funded by the HTA Programme were included in the cohort if they were registered in the NIHR research programmes database and was planned to submit the draft final report for publication in Health Technology Assessment on or before 9 December 2011. MAIN OUTCOME MEASURES: The median time to publication and publication at 30 months in Health Technology Assessment and in an external journal were determined by searching the NIHR research programmes database and HTA Programme website. RESULTS: Of 458 included projects, 184 (40.2%) were primary research projects and 274 (59.8%) were evidence syntheses. A total of 155 primary research projects had a completion date; the median time to publication was 23 months (26.5 and 35.5 months to publish a monograph and to publish in an external journal, respectively) and 69% were published within 30 months. The median time to publication of HTA-funded trials (n=126) was 24 months and 67.5% were published within 30 months. Among the evidence syntheses with a protocol online date (n=223), the median time to publication was 25.5 months (28 months to publication as a monograph), but only 44.4% of evidence synthesis projects were published in an external journal. 65% of evidence synthesis studies had been published within 30.0 months. CONCLUSIONS: Research funded by the HTA Programme publishes promptly. The importance of Health Technology Assessment was highlighted as the median time to publication was 9 months shorter for a monograph than an external journal article.

Setting the future policy agenda for health technology assessment: a specialty mapping approach.

BACKGROUND: There is a need for innovative methodologies to identify and prioritize topics for health technology assessment (HTA). A pilot project to evaluate the methodology for specialty mapping was undertaken in the area of child and adolescent health. Two case studies are presented, in the area of sexually transmitted infections and acute pain. METHODS: The methodology comprised sequential stages, based on principles of systematic review. A "stakeholder model" encouraged wider participation. Key stages included identifying the topic area and setting the scope; developing a care pathway; searching for clinical guidelines/guidance, and evaluation literature; synthesis and mapping of literature to the "nodes" of the care pathway to highlight gaps; prioritizing the topics with stakeholders; and referring priorities to the appropriate agencies. RESULTS: A total of thirty guidelines/guidance documents and sixteen evaluation studies were mapped across the two case studies. In some nodes of the care pathway, more literature was mapped than others, suggesting important gaps in research and policy guidance. Sixty-two policy questions were identified and were rated by stakeholders in prioritization workshops. The highest priorities have been considered by senior committees for likely commissioning as research or guidelines/guidance. CONCLUSIONS: This is one of the few published examples of innovative methodology to identify and prioritize topics for HTA. Specialty mapping can make a positive contribution to the policy agenda, with several research and policy gaps being fed into existing prioritization channels. Adequate time, resources, and capacity is required particularly in engaging stakeholders and developing a care pathway. Implementation of specialty mapping in other topic areas with on-going evaluation is recommended.

UK department of health guidance on prescribing for impotence following the introduction of sildenafil: potential to contain costs in the average health authority district.

OBJECTIVES: To evaluate the effectiveness at containing service costs of the UK's Department of Health (DoH) guidance on prescribing for impotence implemented after the introduction of sildenafil and taking effect from 1 July 1999. DESIGN: A pragmatic economic analysis of the impact of the DoH guidance on specialist-care activity and costs and primary-care prescribing costs from the perspective of the UK National Health Service. Primary-care prescribing costs and specialist-care activity and cost data were collected for 12-month periods before and after the introduction of the guidance. SETTING: Portsmouth and South East Hampshire Health Authority. RESULTS: Specialist-care activity and associated costs fell by 70% in the first year following the introduction of the DoH guidance while primary-care prescribing costs doubled. The overall cost for providing impotence services in Portsmouth and South East Hampshire in 1999-2000 was pound 232,619, and is similar to the cost incurred in 1998-1999 of pound 225,108 (uplifted to 1999-2000 values). CONCLUSIONS: The DoH guidance on prescribing for impotence has effectively reduced specialist-care activity and costs in Portsmouth and South East Hampshire. It offers the potential to allow the overall costs of impotence services in the district to be contained even with the use of higher cost drugs, such as sildenafil.