scholarly journals Stratified versus usual care for the management of primary care patients with sciatica: the SCOPiC RCT

2020 ◽  
Vol 24 (49) ◽  
pp. 1-130
Author(s):  
Nadine E Foster ◽  
Kika Konstantinou ◽  
Martyn Lewis ◽  
Reuben Ogollah ◽  
Benjamin Saunders ◽  
...  
Keyword(s):  
Primary Care ◽  
Health Technology Assessment ◽  
Usual Care ◽  
Technology Assessment ◽  
Fast Track ◽  
Primary Outcome ◽  
Health Technology ◽  
Care Pathways ◽  
Care Model ◽  
Stratified Care

Background Sciatica has a substantial impact on patients and society. Current care is ‘stepped’, comprising an initial period of simple measures of advice and analgesia, for most patients, commonly followed by physiotherapy, and then by more intensive interventions if symptoms fail to resolve. No study has yet tested a model of stratified care in which patients are subgrouped and matched to different care pathways based on their prognosis and clinical characteristics. Objectives The objectives were to investigate the clinical effectiveness and cost-effectiveness of a stratified care model compared with usual, non-stratified care. Design This was a two-parallel group, multicentre, pragmatic, 1 : 1 randomised controlled trial. Setting Participants were recruited from primary care (42 general practices) in North Staffordshire, North Shropshire/Wales and Cheshire in the UK. Participants Eligible patients were aged ≥ 18 years, had suspected sciatica, had access to a mobile phone/landline, were not pregnant, were not receiving treatment for the same problem and had not had previous spinal surgery. Interventions In stratified care, a combination of prognostic and clinical criteria associated with referral to spinal specialist services was used to allocate patients to one of three groups for matched care pathways. Group 1 received advice and up to two sessions of physiotherapy, group 2 received up to six sessions of physiotherapy, and group 3 was fast-tracked to magnetic resonance imaging and spinal specialist opinion. Usual care was based on the stepped-care approach without the use of any stratification tools/algorithms. Patients were randomised using a remote web-based randomisation service. Main outcome measures The primary outcome was time to first resolution of sciatica symptoms (six point ordinal scale, collected via text messages). Secondary outcomes (at 4 and 12 months) included pain, function, psychological health, days lost from work, work productivity, satisfaction with care and health-care use. A cost–utility analysis was undertaken over 12 months. A qualitative study explored patients’ and clinicians’ views of the fast-track care pathway to a spinal specialist. Results A total of 476 patients were randomised (238 in each arm). For the primary outcome, the overall response rate was 89.3% (88.3% and 90.3% in the stratified and usual care arms, respectively). Relief from symptoms was slightly faster (2 weeks median difference) in the stratified care arm, but this difference was not statistically significant (hazard ratio 1.14, 95% confidence interval 0.89 to 1.46; p = 0.288). On average, participants in both arms reported good improvement from baseline, on most outcomes, over time. Following the assessment at the research clinic, most participants in the usual care arm were referred to physiotherapy. Conclusions The stratified care model tested in this trial was not more clinically effective than usual care, and was not likely to be a cost-effective option. The fast-track pathway was felt to be acceptable to both patients and clinicians; however, clinicians expressed reluctance to consider invasive procedures if symptoms were of short duration. Limitations Participants in the usual care arm, on average, reported good outcomes, making it challenging to demonstrate superiority of stratified care. The performance of the algorithm used to allocate patients to treatment pathways may have influenced results. Future work Other approaches to stratified care may provide superior outcomes for sciatica. Trial registration Current Controlled Trials ISRCTN75449581. Funding This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 24, No. 49. See the NIHR Journals Library website for further project information.

scholarly journals Combining mirtazapine with SSRIs or SNRIs for treatment-resistant depression: the MIR RCT

2018 ◽  
Vol 22 (63) ◽  
pp. 1-136 ◽  
Author(s):  
David Kessler ◽  
Alison Burns ◽  
Debbie Tallon ◽  
Glyn Lewis ◽  
Stephanie MacNeill ◽  
...  
Keyword(s):  
Primary Care ◽  
Health Technology Assessment ◽  
Usual Care ◽  
Reuptake Inhibitor ◽  
Technology Assessment ◽  
Primary Outcome ◽  
Health Technology ◽  
Treatment Resistant Depression ◽  
Treatment Resistant ◽  
Resistant Depression

Background Depression is usually managed in primary care and antidepressants are often the first-line treatment, but only half of those treated respond to a single antidepressant. Objectives To investigate whether or not combining mirtazapine with serotonin–noradrenaline reuptake inhibitor (SNRI) or selective serotonin reuptake inhibitor (SSRI) antidepressants results in better patient outcomes and more efficient NHS care than SNRI or SSRI therapy alone in treatment-resistant depression (TRD). Design The MIR trial was a two-parallel-group, multicentre, pragmatic, placebo-controlled randomised trial with allocation at the level of the individual. Setting Participants were recruited from primary care in Bristol, Exeter, Hull/York and Manchester/Keele. Participants Eligible participants were aged ≥ 18 years; were taking a SSRI or a SNRI antidepressant for at least 6 weeks at an adequate dose; scored ≥ 14 points on the Beck Depression Inventory-II (BDI-II); were adherent to medication; and met the International Statistical Classification of Diseases and Related Health Problems, Tenth Revision, criteria for depression. Interventions Participants were randomised using a computer-generated code to either oral mirtazapine or a matched placebo, starting at a dose of 15 mg daily for 2 weeks and increasing to 30 mg daily for up to 12 months, in addition to their usual antidepressant. Participants, their general practitioners (GPs) and the research team were blind to the allocation. Main outcome measures The primary outcome was depression symptoms at 12 weeks post randomisation compared with baseline, measured as a continuous variable using the BDI-II. Secondary outcomes (at 12, 24 and 52 weeks) included response, remission of depression, change in anxiety symptoms, adverse events (AEs), quality of life, adherence to medication, health and social care use and cost-effectiveness. Outcomes were analysed on an intention-to-treat basis. A qualitative study explored patients’ views and experiences of managing depression and GPs’ views on prescribing a second antidepressant. Results There were 480 patients randomised to the trial (mirtazapine and usual care, n = 241; placebo and usual care, n = 239), of whom 431 patients (89.8%) were followed up at 12 weeks. BDI-II scores at 12 weeks were lower in the mirtazapine group than the placebo group after adjustment for baseline BDI-II score and minimisation and stratification variables [difference –1.83 points, 95% confidence interval (CI) –3.92 to 0.27 points; p = 0.087]. This was smaller than the minimum clinically important difference and the CI included the null. The difference became smaller at subsequent time points (24 weeks: –0.85 points, 95% CI –3.12 to 1.43 points; 12 months: 0.17 points, 95% CI –2.13 to 2.46 points). More participants in the mirtazapine group withdrew from the trial medication, citing mild AEs (46 vs. 9 participants). Conclusions This study did not find convincing evidence of a clinically important benefit for mirtazapine in addition to a SSRI or a SNRI antidepressant over placebo in primary care patients with TRD. There was no evidence that the addition of mirtazapine was a cost-effective use of NHS resources. GPs and patients were concerned about adding an additional antidepressant. Limitations Voluntary unblinding for participants after the primary outcome at 12 weeks made interpretation of longer-term outcomes more difficult. Future work Treatment-resistant depression remains an area of important, unmet need, with limited evidence of effective treatments. Promising interventions include augmentation with atypical antipsychotics and treatment using transcranial magnetic stimulation. Trial registration Current Controlled Trials ISRCTN06653773; EudraCT number 2012-000090-23. Funding This project was funded by the NIHR Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 22, No. 63. See the NIHR Journals Library website for further project information.

scholarly journals An open randomised study of autoinflation in 4- to 11-year-old school children with otitis media with effusion in primary care

2015 ◽  
Vol 19 (72) ◽  
pp. 1-150 ◽  
Author(s):  
Ian Williamson ◽  
Jane Vennik ◽  
Anthony Harnden ◽  
Merryn Voysey ◽  
Rafael Perera ◽  
...  
Keyword(s):  
Primary Care ◽  
Young Children ◽  
Adverse Events ◽  
Health Technology Assessment ◽  
Usual Care ◽  
Middle Ear ◽  
School Children ◽  
Technology Assessment ◽  
Otitis Media With Effusion ◽  
Health Technology

BackgroundOtitis media with effusion (OME) is a very common problem in primary care, but one that lacks an evidence-based non-surgical treatment.ObjectiveTo determine the clinical effectiveness of nasal balloon autoinflation for the treatment of OME in children.DesignA pragmatic, two-arm, open randomised controlled trial.SettingForty-three general practices from 17 UK primary care trusts recruited between January 2012 and February 2013.ParticipantsSchool children aged 4–11 years with a history of OME symptoms or related concerns in the previous 3 months, and a type B tympanogram, diagnostic of a middle ear effusion, in one or both ears.InterventionThree hundred and twenty children were randomised, 160 to each group, using independent web-based computer-generated randomisation (with minimisation based on age, sex and baseline severity of OME) to either nasal balloon autoinflation performed three times per day for 1–3 months plus usual care, or usual care alone.Main outcome measuresThe proportion of children demonstrating clearance of middle ear fluid in at least one ear (with normal tympanograms) at 1 and 3 months, assessed blind to treatment. An ear-related measure of quality of life (QoL) [a 14-point questionnaire on the impact of OME (OMQ-14)], weekly diary recorded symptoms, compliance and adverse events were all secondary outcomes.ResultsAt 1 month, the proportion of children with normal tympanograms was 47.3% (62/131) in those allocated to autoinflation and 35.6% (47/132) in those receiving usual care [adjusted relative risk (RR) 1.36, 95% confidence interval (CI) 0.99 to 1.88]. At 3 months, the proportions were 49.6% (62/125) and 38.3% (46/120), respectively (adjusted RR 1.37, 95% CI 1.03 to 1.83; number needed to treat = 9). The change in OMQ-14 also favoured the intervention arm (adjusted global score difference –0.42;p = 0.001). Reported compliance was good: 89% in the first month and 80% in months 2 and 3. Adverse events included otalgia in 4% of treated children compared with 1% in the control group. Minor nosebleeds (14% vs. 15%) and respiratory tract infections (18% vs. 13%) were noted.ConclusionWe found the use of autoinflation in young children with OME to be feasible in primary care and effective in both clearing effusions and improving child and parent ear-related QoL and symptoms. This method has scope to be used more widely. Further research is needed for very young children, and to inform prudent use in different health settings.Trial registrationCurrent Controlled Trials ISRCTN55208702.FundingThis project was funded by the National institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full inHealth Technology Assessment, Vol. 19, No. 72. See the NIHR Journals Library website for further project information.

scholarly journals From idealistic rookies to a regional leader: The history of health technology assessment in Poland

2009 ◽  
Vol 25 (S1) ◽  
pp. 156-162 ◽  
Author(s):  
Rafał Niżankowski ◽  
Norbert Wilk
Keyword(s):  
Primary Care ◽  
Health Care ◽  
Decision Making ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
The Public ◽  
History Of ◽  
Decision Making Processes ◽  
Public Payer

In 1989, Poland started to slowly release itself not only from the burden of a half-century of communist indoctrination and soviet exploitation, but also from the consequences of the Semashko model of healthcare organization: low doctors' salaries, primary care based on multispecialty groups, overdeveloped hospital infrastructure, and limited access to sophisticated interventions overcome by patients' unofficial payments.A few years after the 1998 workshop on health technology assessment (HTA) in Budapest, the first HTA reports were elaborated in the National Center for Quality Assessment in Health Care, which could mark the beginning of HTA in Poland. Several individuals and organizations have been involved in developing HTA, both from noncommercial and commercial standpoints.A goal to establish a national HTA agency appeared among the priorities of the Polish Ministry of Health in 2004 and was realized a year later. The Agency for HTA in Poland published guidelines on HTA and established a sound and transparent two-step (assessment-appraisal) process for preparing recommendations on public financing of both drugs and nondrug technologies. The recommendations of the Agency's Consultative Council were warmly welcomed by the public payer. However, the recent major restructuring of the Agency and new drug reimbursement decisions aroused doubts as to keeping transparency of the decision-making processes.

scholarly journals Three wound-dressing strategies to reduce surgical site infection after abdominal surgery: the Bluebelle feasibility study and pilot RCT

2019 ◽  
Vol 23 (39) ◽  
pp. 1-166 ◽  
Author(s):  
Barnaby C Reeves ◽  
Leila Rooshenas ◽  
Rhiannon C Macefield ◽  
Mark Woodward ◽  
Nicky J Welton ◽  
...  
Keyword(s):  
Abdominal Surgery ◽  
Surgical Site Infection ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Value Of Information ◽  
Primary Outcome ◽  
Health Technology ◽  
Controlled Trials ◽  
Site Infection ◽  
Pilot Rct

Background Surgical site infection (SSI) affects up to 20% of people with a primary closed wound after surgery. Wound dressings may reduce SSI. Objective To assess the feasibility of a multicentre randomised controlled trial (RCT) to evaluate the effectiveness and cost-effectiveness of dressing types or no dressing to reduce SSI in primary surgical wounds. Design Phase A – semistructured interviews, outcome measure development, practice survey, literature reviews and value-of-information analysis. Phase B – pilot RCT with qualitative research and questionnaire validation. Patients and the public were involved. Setting Usual NHS care. Participants Patients undergoing elective/non-elective abdominal surgery, including caesarean section. Interventions Phase A – none. Phase B – simple dressing, glue-as-a-dressing (tissue adhesive) or ‘no dressing’. Main outcome measures Phase A – pilot RCT design; SSI, patient experience and wound management questionnaires; dressing practices; and value-of-information of a RCT. Phase B – participants screened, proportions consented/randomised; acceptability of interventions; adherence; retention; validity and reliability of SSI measure; and cost drivers. Data sources Phase A – interviews with patients and health-care professionals (HCPs), narrative data from published RCTs and data about dressing practices. Phase B – participants and HCPs in five hospitals. Results Phase A – we interviewed 102 participants. HCPs interpreted ‘dressing’ variably and reported using available products. HCPs suggested practical/clinical reasons for dressing use, acknowledged the weak evidence base and felt that a RCT including a ‘no dressing’ group was acceptable. A survey showed that 68% of 1769 wounds (727 participants) had simple dressings and 27% had glue-as-a-dressing. Dressings were used similarly in elective and non-elective surgery. The SSI questionnaire was developed from a content analysis of existing SSI tools and interviews, yielding 19 domains and 16 items. A main RCT would be valuable to the NHS at a willingness to pay of £20,000 per quality-adjusted life-year. Phase B – from 4 March 2016 to 30 November 2016, we approached 862 patients for the pilot RCT; 81.1% were eligible, 59.4% consented and 394 were randomised (simple, n = 133; glue, n = 129; no dressing, n = 132); non-adherence was 3 out of 133, 8 out of 129 and 20 out of 132, respectively. SSI occurred in 51 out of 281 participants. We interviewed 55 participants. All dressing strategies were acceptable to stakeholders, with no indication that adherence was problematic. Adherence aids and patients’ understanding of their allocated dressing appeared to be key. The SSI questionnaire response rate overall was 67.2%. Items in the SSI questionnaire fitted a single scale, which had good reliability (test–retest and Cronbach’s alpha of > 0.7) and diagnostic accuracy (c-statistic = 0.906). The key cost drivers were hospital appointments, dressings and redressings, use of new medicines and primary care appointments. Limitations Multiple activities, often in parallel, were challenging to co-ordinate. An amendment took 4 months, restricting recruitment to the pilot RCT. Only 67% of participants completed the SSI questionnaire. We could not implement photography in theatres. Conclusions A main RCT of dressing strategies is feasible and would be valuable to the NHS. The SSI questionnaire is sufficiently accurate to be used as the primary outcome. A main trial with three groups (as in the pilot) would be valuable to the NHS, using a primary outcome of SSI at discharge and patient-reported SSI symptoms at 4–8 weeks. Trial registration Phase A – Current Controlled Trials ISRCTN06792113; Phase B – Current Controlled Trials ISRCTN49328913. Funding This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 23, No. 39. See the NIHR Journals Library website for further project information. Funding was also provided by the Medical Research Council ConDuCT-II Hub (reference number MR/K025643/1).

scholarly journals Multicentre cluster randomised trial comparing a community group exercise programme and home-based exercise with usual care for people aged 65 years and over in primary care

2014 ◽  
Vol 18 (49) ◽  
pp. 1-106 ◽  
Author(s):  
Steve Iliffe ◽  
Denise Kendrick ◽  
Richard Morris ◽  
Tahir Masud ◽  
Heather Gage ◽  
...  
Keyword(s):  
Physical Activity ◽  
Primary Care ◽  
Usual Care ◽  
Primary Outcome ◽  
Health Technology ◽  
Exercise Programme ◽  
Cluster Randomised ◽  
Home Based ◽  
General Practices ◽  
Secondary Outcomes

BackgroundRegular physical activity (PA) reduces the risk of falls and hip fractures, and mortality from all causes. However, PA levels are low in the older population and previous intervention studies have demonstrated only modest, short-term improvements.ObjectiveTo evaluate the impact of two exercise promotion programmes on PA in people aged ≥ 65 years.DesignThe ProAct65+ study was a pragmatic, three-arm parallel design, cluster randomised controlled trial of class-based exercise [Falls Management Exercise (FaME) programme], home-based exercise [Otago Exercise Programme (OEP)] and usual care among older people (aged ≥ 65 years) in primary care.SettingForty-three UK-based general practices in London and Nottingham/Derby.ParticipantsA total of 1256 people ≥ 65 years were recruited through their general practices to take part in the trial.InterventionsThe FaME programme and OEP. FaME included weekly classes plus home exercises for 24 weeks and encouraged walking. OEP included home exercises supported by peer mentors (PMs) for 24 weeks, and encouraged walking.Main outcome measuresThe primary outcome was the proportion that reported reaching the recommended PA target of 150 minutes of moderate to vigorous physical activity (MVPA) per week, 12 months after cessation of the intervention. Secondary outcomes included functional assessments of balance and falls risk, the incidence of falls, fear of falling, quality of life, social networks and self-efficacy. An economic evaluation including participant and NHS costs was embedded in the clinical trial.ResultsIn total, 20,507 patients from 43 general practices were invited to participate. Expressions of interest were received from 2752 (13%) and 1256 (6%) consented to join the trial; 387 were allocated to the FaME arm, 411 to the OEP arm and 458 to usual care. Primary outcome data were available at 12 months after the end of the intervention period for 830 (66%) of the study participants.The proportions reporting at least 150 minutes of MVPA per week rose between baseline and 12 months after the intervention from 40% to 49% in the FaME arm, from 41% to 43% in the OEP arm and from 37.5% to 38.0% in the usual-care arm. A significantly higher proportion in the FaME arm than in the usual-care arm reported at least 150 minutes of MVPA per week at 12 months after the intervention [adjusted odds ratio (AOR) 1.78, 95% confidence interval (CI) 1.11 to 2.87;p = 0.02]. There was no significant difference in MVPA between OEP and usual care (AOR 1.17, 95% CI 0.72 to 1.92;p = 0.52). Participants in the FaME arm added around 15 minutes of MVPA per day to their baseline physical activity level. In the 12 months after the close of the intervention phase, there was a statistically significant reduction in falls rate in the FaME arm compared with the usual-care arm (incidence rate ratio 0.74, 95% CI 0.55 to 0.99;p = 0.042). Scores on the Physical Activity Scale for the Elderly showed a small but statistically significant benefit for FaME compared with usual care, as did perceptions of benefits from exercise. Balance confidence was significantly improved at 12 months post intervention in both arms compared with the usual-care arm. There were no statistically significant differences between intervention arms and the usual-care arm in other secondary outcomes, including quality-adjusted life-years. FaME is more expensive than OEP delivered with PMs (£269 vs. £88 per participant in London; £218 vs. £117 in Nottingham). The cost per extra person exercising at, or above, target was £1919.64 in London and £1560.21 in Nottingham (mean £1739.93).ConclusionThe FaME intervention increased self-reported PA levels among community-dwelling older adults 12 months after the intervention, and significantly reduced falls. Both the FaME and OEP interventions appeared to be safe, with no significant differences in adverse reactions between study arms.Trial registrationThis trial is registered as ISRCTN43453770.FundingThis project was funded by the NIHR Health Technology Assessment programme and will be published in full inHealth Technology Assessment; Vol. 18, No. 49. See the NIHR Journals Library website for further project information.

scholarly journals Adding emollient bath additives to standard eczema management for children with eczema: the BATHE RCT

2018 ◽  
Vol 22 (57) ◽  
pp. 1-116 ◽  
Author(s):  
Miriam Santer ◽  
Kate Rumsby ◽  
Matthew J Ridd ◽  
Nick A Francis ◽  
Beth Stuart ◽  
...  
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Primary Outcome ◽  
Intervention Group ◽  
Additive Group ◽  
Health Technology ◽  
Control Group ◽  
Open Label ◽  
Secondary Outcomes ◽  
Bath Additives

BackgroundChildhood eczema is very common. Treatment often includes emollient bath additives, despite there being little evidence of their effectiveness.ObjectivesTo determine the clinical effectiveness and cost-effectiveness of emollient bath additives in the management of childhood eczema.DesignPragmatic, randomised, open-label, multicentre superiority trial with two parallel groups.SettingNinety-six general practices in Wales, the west of England and southern England. Invitation by personal letter or opportunistically.ParticipantsChildren aged between 12 months and 12 years fulfilling the UK Diagnostic Criteria for Atopic Eczema. Children with inactive or very mild eczema (a score of ≤ 5 on the Nottingham Eczema Severity Scale) were excluded, as were children who bathed less than once per week or whose parents/carers were not prepared to accept randomisation.InterventionsThe intervention group were prescribed bath additives by their usual clinical team and were asked to use them regularly for 12 months. The control group were asked to use no bath additives for 12 months. Both groups continued standard eczema management, including regular leave-on emollients and topical corticosteroids (TCSs) when required.Main outcome measuresThe primary outcome was eczema control measured by Patient Oriented Eczema Measure [POEM, 0 (clear) to 28 (severe)] weekly for 16 weeks. The secondary outcomes were eczema severity over 1 year (4-weekly POEM), number of eczema exacerbations, disease-specific quality of life (QoL) (Dermatitis Family Impact Questionnaire), generic QoL (Child Health Utility-9 Dimensions) and type and quantity of topical steroid/calcineurin inhibitors prescribed. Children were randomised (1 : 1) using online software to either bath additives plus standard eczema care or standard eczema care alone, stratified by recruiting centre, and there was open-label blinding.ResultsFrom December 2014 to May 2016, 482 children were randomised: 51% were female, 84% were white and the mean age was 5 years (n = 264 in the intervention group,n = 218 in the control group). Reported adherence to randomised treatment allocation was > 92% in both groups, with 76.7% of participants completing at least 12 (80%) of the first 16 weekly questionnaires for the primary outcome. Baseline POEM score was 9.5 [standard deviation (SD) 5.7] in the bath additives group and 10.1 (SD 5.8) in the no bath additives group. Average POEM score over the first 16 weeks was 7.5 (SD 6.0) in the bath additives group and 8.4 (SD 6.0) in the no bath additives group, with no statistically significant difference between the groups. After controlling for baseline severity and confounders (ethnicity, TCS use, soap substitute use) and allowing for clustering of participants within centres and responses within participants over time, POEM scores in the no bath additive group were 0.41 points higher than in the bath additive group (95% confidence interval –0.27 to 1.10), which is well below the published minimal clinically important difference of 3 points. There was no difference between groups in secondary outcomes or in adverse effects such as redness, stinging or slipping.LimitationsSimple randomisation resulted in an imbalance in baseline group size, although baseline characteristics were well balanced between groups.ConclusionThis trial found no evidence of clinical benefit of including emollient bath additives in the standard management of childhood eczema.Future workFurther research is required on optimal regimens of leave-on emollients and the use of emollients as soap substitutes.Trial registrationCurrent Controlled Trials ISRCTN84102309.FundingThis project was funded by the NIHR Health Technology Assessment Programme and will be published in full inHealth Technology Assessment; Vol. 22, No. 57. See the NIHR Journals Library website for further project information.

scholarly journals The importance of health technology assessment to ensure an efficient and equitable access to high-cost therapies

2020 ◽  
Author(s):  
◽  
Ion Agirrezabal
Keyword(s):  
Primary Care ◽  
Resource Allocation ◽  
Insulin Glargine ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Real World ◽  
Health Technology ◽  
Decision Makers ◽  
The Real ◽  
Innovative Drugs

According to the World Health Organization, the key goal of health systems is to improve the average level of the population health and to reduce health inequalities in the population. In order to realise this goal, health system decision-makers need to decide which health technologies to invest in and which not to. Health technology assessment (HTA) provides a framework for decision-makers to make resource allocation and priority setting decisions based on the existing evidence. Considering the increasingly tight healthcare budgets and the rich pipeline of high-cost, innovative drugs very likely coming to market in the next few years, it is crucial that a robust and transparent HTA process be undertaken to assess these drugs, evaluating all aspects of the disease and treatment and involving all stakeholders affected. We conducted three standalone projects analysing different aspects of recently launched innovative drugs. In our first study, we combined high-quality sources of evidence, both from the real-world and randomised controlled trials, to evaluate the cost-effectiveness of carfilzomib for treating multiple myeloma patients. By harnessing the power of these data sources, we demonstrated that the reimbursement of carfilzomib is likely to represent an efficient allocation of existing resources. Despite the availability of good sources of evidence, the real-world distribution and use of innovative drugs may not be efficient nor fair, and this is what we demonstrated with our two other studies. Firstly, we showed that significant inequalities exist in the distribution of anti-osteoporosis drugs in primary care in England. The most striking case was that of denosumab, a high-cost innovative treatment, with prescriptions disproportionately concentrated among the least deprived. Substantial inequalities also exist in the use of insulin glargine biosimilars in primary care in England, even though guidelines and initiatives to promote the use of biosimilars have been put in place. In this study we observed that the real-world savings realised from the use of insulin glargine biosimilars represents a small proportion compared with what could have been achieved should their uptake had been higher. The results of these two studies, therefore, show that resource allocation may not be efficient nor fair in the real world, and similar situations are likely to exist in other disease areas. In summary, even though in many cases ample evidence exists to assist healthcare authorities making resource allocation decisions, we have demonstrated that resource allocation in the real world may not be optimal.

scholarly journals Carer administration of as-needed subcutaneous medication for breakthrough symptoms in people dying at home: the CARiAD feasibility RCT

2020 ◽  
Vol 24 (25) ◽  
pp. 1-150 ◽  
Author(s):  
Marlise Poolman ◽  
Jessica Roberts ◽  
Stella Wright ◽  
Annie Hendry ◽  
Nia Goulden ◽  
...  
Keyword(s):  
Health Care ◽  
Health Technology Assessment ◽  
Usual Care ◽  
Outcome Measures ◽  
Technology Assessment ◽  
Health Care Professionals ◽  
Retention Rate ◽  
Health Technology ◽  
Dying At Home ◽  
At Home

Background Most people who are dying want to be cared for at home, but only half of them achieve this. The likelihood of a home death often depends on the availability of able and willing lay carers. When people who are dying are unable to take oral medication, injectable medication is used. When top-up medication is required, a health-care professional travels to the dying person’s home, which may delay symptom relief. The administration of subcutaneous medication by lay carers, although not widespread UK practice, has proven to be key in achieving better symptom control for those dying at home in other countries. Objectives To determine if carer administration of as-needed subcutaneous medication for common breakthrough symptoms in people dying at home is feasible and acceptable in the UK, and if it would be feasible to test this intervention in a future definitive randomised controlled trial. Design We conducted a two-arm, parallel-group, individually randomised, open pilot trial of the intervention versus usual care, with a 1 : 1 allocation ratio, using convergent mixed methods. Setting Home-based care without 24/7 paid care provision, in three UK sites. Participants Participants were dyads of adult patients and carers: patients in the last weeks of their life who wished to die at home and lay carers who were willing to be trained to give subcutaneous medication. Strict risk assessment criteria needed to be met before approach, including known history of substance abuse or carer ability to be trained to competency. Intervention Intervention-group carers received training by local nurses using a manualised training package. Main outcome measures Quantitative data were collected at baseline and 6–8 weeks post bereavement and via carer diaries. Interviews with carers and health-care professionals explored attitudes to, experiences of and preferences for giving subcutaneous medication and experience of trial processes. The main outcomes of interest were feasibility, acceptability, recruitment rates, attrition and selection of the most appropriate outcome measures. Results In total, 40 out of 101 eligible dyads were recruited (39.6%), which met the feasibility criterion of recruiting > 30% of eligible dyads. The expected recruitment target (≈50 dyads) was not reached, as fewer than expected participants were identified. Although the overall retention rate was 55% (22/40), this was substantially unbalanced [30% (6/20) usual care and 80% (16/20) intervention]. The feasibility criterion of > 40% retention was, therefore, considered not met. A total of 12 carers (intervention, n = 10; usual care, n = 2) and 20 health-care professionals were interviewed. The intervention was considered acceptable, feasible and safe in the small study population. The context of the feasibility study was not ideal, as district nurses were seriously overstretched and unfamiliar with research methods. A disparity in readiness to consider the intervention was demonstrated between carers and health-care professionals. Findings showed that there were methodological and ethics issues pertaining to researching last days of life care. Conclusion The success of a future definitive trial is uncertain because of equivocal results in the progression criteria, particularly poor recruitment overall and a low retention rate in the usual-care group. Future work regarding the intervention should include understanding the context of UK areas where this has been adopted, ascertaining wider public views and exploring health-care professional views on burden and risk in the NHS context. There should be consideration of the need for national policy and of the most appropriate quantitative outcome measures to use. This will help to ascertain if there are unanswered questions to be studied in a trial. Trial registration Current Controlled Trials ISRCTN11211024. Funding This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 24, No. 25. See the NIHR Journals Library website for further project information.

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