The number of patient preference studies in health has increased dramatically. There is growing use of patient preferences in a wide variety of contexts, including health technology assessment. Patient preference studies can help inform decision makers on the needs and priorities of patients and the tradeoffs they are willing to make about health technologies.

This International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Task Force included international experts, health preference researchers and others from diverse backgrounds, including regulatory, health technology assessment, medicine, patient advocacy, and the pharmaceutical industry. The report underwent two rounds of written reviews by ISPOR Preferences Special Interest Group members until a final consensus was reached. The Task Force focused on developing a roadmap that would: (i) apply to the wide variety of preference methods, (ii) identify key domains to guide researchers and other stakeholders in making patient preference studies more useful to decision makers, and (iii) detail important questions to guide researchers conducting preference studies and those critically appraising them.

This Task Force report provides a novel roadmap that invites patient-preference researchers to work with decision makers, patients and other stakeholders to do even more to ensure that studies are useful and impactful. The ISPOR Roadmap consists of five key elements: (i) Context; (ii) Purpose; (iii) Population; (iv) Method; and (v) Impact. In this report, we define these five elements and provide good practices on how patient-preference researchers can actively contribute to increasing the usefulness and impact of patient preference studies in decision-making. We also present a set of key questions that can support researchers and other stakeholders in assessing efforts that promote preference studies’ intended and unintended impact.

This roadmap can help increase the usefulness and impact of patient preference studies in decision-making by challenging researchers to engage and partner with decision makers, patients and others, and together consider the intended and unintended impacts of patient preference studies on decision-making while actively fostering positive impact.

Reimbursement decision making is based on a relative effectiveness assessment (REA), which may be combined with a cost-effectiveness assessment, by national Health Technology Assessment (HTA) agencies. These assessments are based on clinical data where new interventions are compared to the current standard of care, which may differ between countries. Since most pivotal trials only include a limited number of interventions, indirect treatment comparisons (ITCs) can be used to compare multiple interventions. The aim of this study was to evaluate the use of ITCs in HTA decision making in the Netherlands and England.

All pharmaceutical assessments published between 2015 and 2019 by the National Health Care Institute (ZIN) and the National Institute for Health and Care Excellence (NICE) were reviewed to determine whether an ITC had been used. For detailed analysis we included all assessments of ZIN using an ITC, and a random sample of assessments of NICE using an ITC (10 assessments per publication year).

Between 2015 and 2019 a total of 106 and 265 assessments were conducted by ZIN and NICE, respectively. Of these assessments 48 from ZIN and 150 from NICE included an ITC. The detailed analysis showed that pharmaceutical assessments including indirect comparative evidence led to the REA conclusion of similar therapeutic evidence in 57 percent of 48 assessments by ZIN and in 52 percent of 50 assessments by NICE. Reimbursement recommendations including indirect comparative evidence most often resulted in positive recommendations by ZIN (57% assessments), and in restricted recommendations by NICE (50% assessments). Different methods were employed to incorporate indirect comparative evidence, such as naïve ITCs and network meta-analysis.

Our results showed a significant variability in the use of ITCs between NICE and ZIN, which may contribute to differences in their recommendations. Further analysis will provide deeper insight in these differences and may provide suggestions for a clearer international guidance on the use of ITCs for HTA.

This study aimed to provide insight into the congruity of acute cystitis (AC) diagnosis in women, measured both by the Acute Cystitis Symptom Score (ACSS) questionnaire and urine test(s).

The ACSS questionnaire was developed as a self-administering tool for assessing urinary symptoms, quality of life (QoL) and treatment outcomes in healthy, nonpregnant female patients.

This prospective observational cohort study compared AC diagnosis based on the questionnaire with a GP diagnosis based on dipstick/dipslide test(s). ACSS questionnaire form A (typical and differential symptoms, QoL and relevant conditions) was filled in by the patient group, women suspected for AC visiting a GP practice with a urine sample, and the reference group, women visiting a community pharmacy for any medication. Analyses were performed assuming that the GP diagnosis based on urine test(s) was correct. Divergent result(s) of urine test(s) and ACSS questionnaire were analysed for scores of all individual questionnaire domains. Statistical analyses included descriptive statistics and the positive predictive value (PPV) and the negative predictive value (NPV) of the ACSS questionnaire and the urine test(s).

In the patient group, 59 women were included, 38 of whom a GP positively diagnosed for AC. The reference group included 70 women. The PPV of the ACSS questionnaire was 77.3%, and the NPV was 73.3%. Analysis of patient data for divergent results showed that differential symptoms, QoL and relevant conditions explained false-positive and false-negative results. Revised results (most probable diagnosis) based on this analysis showed a PPV and NPV of 88.6% and 73.3% for the ACSS questionnaire and 100% and 76.2% for the urine test(s). For use in primary care, a reduction in false-positive and false-negative results can be achieved by including scores for differential symptoms, QoL and relevant conditions, alongside a total typical symptoms score of 6 or higher.

Although health technology assessment (HTA) and healthcare quality improvement are distinct processes, a greater level of alignment in outcome measures used may increase the quality and efficiency of data collection. This study evaluates the agreement in outcome measures used in oncology for healthcare quality improvement and HTAs, and how these align to the International Consortium for Health Outcomes Measurement (ICHOM) standard sets.

We conducted a cross-sectional comparative analysis of ICHOM sets focusing on oncological indications and publicly available measures for healthcare quality and HTA reports published by the National Health Care Institute from the Netherlands and the National Institute for Health and Care Excellence from the United Kingdom.

All ICHOM sets and HTAs used overall survival, whereas quality improvement used different survival estimates. Different progression estimates for cancer were used in HTAs, ICHOM sets, and quality improvement. Data on health-related quality of life (HRQoL) was recommended in all ICHOM sets and all HTAs, but selectively for quality improvement. In HTAs, generic HRQoL questionnaires were preferred, whereas, in quality improvement and ICHOM sets, disease-specific questionnaires were recommended. Unfavorable outcomes were included in all HTAs and all ICHOM sets, but not always for quality improvement.

Although HTA and quality improvement use outcome measures from the same domains, a greater level of alignment seems possible. ICHOM may provide input on standardized outcome measures to support this alignment. However, residual discrepancies will remain due to the different objectives of HTA and quality improvement.

To study whether changes in drug preferences in the Dutch guideline for the treatment of Urinary Tract Infection (UTI) for General Practitioners (GPs) in 2013, resulted in corresponding changes in antibiotic dispensing.

For the treatment of uncomplicated UTI, nitrofurantoin remained the first choice, while fosfomycin became the second choice and changed ranks with trimethoprim. For a subsequent febrile UTI, ciprofloxacin became the first choice and changed ranks with amoxicillin/clavulanic acid, co-trimoxazole remained the third choice.

In this observational cross-sectional study, routinely collected dispensing data from the Dutch Foundation of Pharmaceutical Statistics from 2012 to 2017 were used. The number of women 18 years and older, treated with one of the guideline antibiotics for uncomplicated UTI and subsequent febrile UTI were analysed annually. Proportions were calculated. Data were stratified for age categories. Failure of uncomplicated UTI treatment was defined as the dispensing of an antibiotic for febrile UTI within 14 days after the dispensing of an antibiotic for uncomplicated UTI.

Data were available from 81% of all pharmacies in 2012 to 89% in 2017. Percentages of women dispensed nitrofurantoin were relatively stable with 87.4% in 2012 and 84.4% in 2017. Percentages of women dispensed fosfomycin increased from 5.4% in 2012 to 21.8% in 2017, whereas percentages of women dispensed trimethoprim decreased from 17.8% to 8.0%. Within age categories, the percentage of women dispensed fosfomycin increased from 12.4% in women 18–30 years old to 36.7% in women above 80 years old. Percentages of women dispensed antibiotics for febrile UTI remained stable at 5% annually. Percentages of women receiving ciprofloxacin increased from 1.9% in 2012 to 3.3% in 2017, while those receiving amoxicillin/clavulanic acid decreased from 2.9% to 1.8%. New guideline recommendations resulted in corresponding changes in dispensed antibiotics for uncomplicated UTI and subsequent febrile UTI. Drug choices differed for age categories.

Differences between healthcare datasets in structure, content, and coding systems are widely recognized as significant barriers to generating robust evidence for regulatory and medical decision making. As a result, there is a growing interest in using common data models embedded within large data networks. By standardizing the structure, contents, and semantics of disparate healthcare databases, common data models like the Observational and Medical Outcomes Partnerships common data model (OMOP-CDM) enable multidatabase studies to be undertaken at speed and in a transparent way. To date, little attention has been given to their potential role in health technology assessment (HTA).

We identify the uses of observational data in generating evidence in HTA, some common analytical challenges faced in their estimation, and the infrastructural, technical, and data reusability constraints that limit its wider use. We discuss where and how the OMOP-CDM could overcome these barriers in relation to different types of evidence requirements.

The OMOP-CDM increases the interoperability of otherwise disparate datasets, allowing reliable evidence to be generated from multidatabase studies at speed and transparently. The current analytical tools are best suited for clinical characterization and population-level effect estimation. Further developments to these tools are required to support analyses common in HTA like parametric survival modeling. Differences in costing methods as well as the structure of healthcare delivery between countries may limit the feasibility and value of standardization.

The OMOP-CDM has the potential to support reliable and timely evidence generation in HTA. The analytical tools should be further developed to support common HTA use cases.

Women with a history of bipolar disorder or postpartum psychosis are at extremely high risk of relapse postpartum. Although lithium prophylaxis has demonstrated efficacy in reducing postpartum relapse, the timing of prophylaxis remains controversial. Here we evaluate the use of lithium during pregnancy compared to initiation postpartum in women at high risk for postpartum psychosis.

In total, 76 pregnant women at high risk for postpartum psychosis were referred to our psychiatric outpatient clinic between 2003 and 2010. Women who were medication-free at the time of initial evaluation were advised to start lithium prophylaxis immediately postpartum. In contrast, women already on maintenance lithium during pregnancy were advised to continue this treatment.

All women with a history of psychosis limited to the postpartum period (PP, n = 30) remained stable throughout pregnancy despite being medication-free. In contrast, 23.9% of women with a history of bipolar disorder (BD, n = 46) relapsed during pregnancy, despite the majority using prophylaxis throughout pregnancy. Postpartum relapse was highest in women with BD who experienced mood episodes during pregnancy (63.6%). Remarkably however, none of the 21 women with PP using postpartum prophylaxis relapsed, compared to 44.4% of PP patients who declined lithium prophylaxis.

We recommend initiating prophylactic treatment immediately postpartum in women with a history of psychosis limited to the postpartum period, offering an important clinical advantage by avoiding in utero fetal exposure to prophylactic medication. Additionally, patients with BD require continuous prophylaxis throughout pregnancy and the postpartum period to effectively reduce their peripartum relapse risk.

There has been a move towards the development of disease-modifying agents in Alzheimer's disease (AD) and it is likely that early disease-modifying treatments will initially be offered to people who have positive AD markers and mild cognitive impairment (MCI). Consequently, disease-modifying drugs will have distinctive features as compared to currently licensed symptomatic treatments, which makes the implications of these new agents for regulatory and health technology assessment (HTA) processes unclear.

The ROADMAP (Real-world Outcomes across the AD spectrum for better care: Multi-modal data Access Platform) project provides the foundation for a European data platform for real-world evidence in AD, and established an expert advisory group (EXAG) consisting of regulatory and HTA experts. This presentation will summarize the key lessons from the first year of ROADMAP's EXAG and identifies the next steps that are required to prepare Europe's healthcare systems for a disease-modifying drug.

The EXAG identified a need for: (i) establishing the rationale for the selection of priority outcomes in pre-clinical AD and MCI; (ii) establishing accepted outcomes for defining prevention of AD or delayed AD onset; (iii) exploring modern technology that could assist in testing cognition that could easily be used in clinical practice; and (iv) establishing caregiver-relevant outcomes (e.g. quality of life, loss of income, carer time) that are important to capture; and found that not all evidence to support modelling assumptions can be generated through RCTs, making the case for using real-world evidence.

Many of the challenges that the EXAG identified can be solved by generating better real-world data in AD. There is a clear need to agree on the outcomes that will facilitate and inform regulatory and HTA decision-making. Once the gaps in the availability of outcomes in AD will be closed, we will be one step closer towards being ready for a disease-modifying drug.

Antidepressants are frequently prescribed but results regarding their efficacy have been equivocal for different spectra of the severity continuum and their side-effects are often burdensome. Non-adherence is a likely consequence. The objective was therefore to examine patients’ trade-offs between the efficacy, side-effects and other drawbacks of antidepressants and whether these trade-offs predicted non-adherence.

Trade-offs from 225 antidepressant users, recruited through community pharmacies, were assessed with an Adaptive Conjoint Analysis (ACA) choice task that was customized to each individual patient. From the estimated utilities, relative importance scores of treatment properties were calculated. Non-adherence was measured through self-report and pharmacy refill data.

Relapse prevention and symptom relief were on average equally important. Side-effects were as important and the side-effect stomach and intestine complaints was on average even slightly more important than relapse prevention and symptom relief. Additional treatment with psychotherapy was preferred by 61% of the patients. A benefit/drawback ratio revealed that 18% of the patients did not consider the efficacy to outweigh the drawbacks. A higher benefit/drawback ratio was associated with a decreased odds of intentional non-adherence [odds ratio (OR) 0.2, 95% confidence interval (CI) 0.07–0.7, Wald = 6.7, p = 0.01).

For nearly one in five patients, the efficacy of antidepressants does not outweigh their drawbacks. Knowing patients’ trade-offs is likely to aid both physicians and patients to identify important treatment preferences, to improve adherence and to make more deliberate decisions on whether or not to continue treatment.

In previous research, lapses in dosing of paroxetine or sertraline were associated with significantly more discontinuation symptoms and deterioration of depressive symptomatology compared with fluoxetine.

To evaluate dosing lapses in patients chronically treated with selective serotonin reuptake inhibitors (SSRIs) in uncontrolled circumstances.

In a prospective observational study we evaluated compliance data in chronic users of SSRIs using electronic drug exposure monitors.

During a 3-month follow-up we found that 50/69 (72.5%) patients missed at least one dosing day and 20/69 (29.0%) missed two or more consecutive days.

About 30% of patients treated with short-acting SSRIs had dosing lapses of 2 or more days, which, as described in prior studies, is long enough to result in clinically relevant deterioration of mental status.