Objectives: Various minimal clinically important difference (MCID) threshold estimation techniques have been applied to seasonal allergic rhinitis (SAR). The objectives of this study are to (i) assess the difference in magnitude of alternative SAR MCID threshold estimates and (ii) evaluate the impact of alternative MCID estimates on health technology assessment (HTA).

Methods: Data describing change from baseline of the reflective Total Nasal Symptom Score (rTNSS) for four intranasal SAR treatments were obtained from United States Food and Drug Administration-approved prescribing information. Treatment effects were then compared with anchor-based MCID thresholds derived by Barnes et al. and thresholds obtained from an Agency for Healthcare Research and Quality (AHRQ) panel.

Results: The change in rTNSS score from baseline, represented as the average of the twice-daily recorded scores of the rTNSS, was -2.1 (p < .001) for azelastine hydrochloride 0.10%, 1.35 (p = .014) for ciclesonide, and -1.47 (p < .001) for fluticasone furoate. The change in the rTNSS score from baseline, represented by sum of the AM and PM score, was -2.7 for MP-AzeFlu (p < .001). The rTNSS change from baseline for each product was compared with anchor-based MCID threshold and the AHRQ panel estimates. Comparison of the observed treatment effect to the anchor-based and AHRQ panel MCID thresholds results in different conclusions, with clinically important differences being inferred when anchor-based estimates serve as the reference point.

Conclusion: The AHRQ panel MCID threshold for the rTNSS was twelve times larger than the anchor-based estimates resulting in conflicting recommendations on whether different SAR treatments provide clinically meaningful benefit.

Objectives: The aim of this study was to obtain information on methods used to measure health technology assessment (HTA) influence, decisions that were influenced, and outcomes linked to HTA.

Methods: Electronic databases were used to locate studies in which HTA influence had been demonstrated. Inclusion criteria were studies that reliably reported consideration by decision makers of HTA findings; comparative studies of technology use before and after HTA; and details of changes in policy, health outcomes, or research that could be credibly linked to an HTA.

Results: Fifty-one studies were selected for review. Settings were national (24), regional (12), both national and regional (3) hospitals (9), and multinational (3). The most common approach to appraisal of influence was review of policy or administrative decisions following HTA recommendations (51 percent). Eighteen studies (35 percent) reported interview or survey findings, thirteen (26 percent) reviewed administrative data, and six considered the influence of primary studies. Of 142 decisions informed by HTA, the most common types were on routine clinical practice (67 percent of studies), coverage (63 percent), and program operation (37 percent). The most frequent indications of HTA influence were on decisions related to resource allocation (59 percent), change in practice pattern (31 percent), and incorporation of HTA details in reference material (18 percent). Few publications assessed the contribution of HTA to changing patient outcomes.

Conclusions: The literature on HTA influence remains limited, with little on longer term effects on practice and outcomes. The reviewed publications indicated how HTA is being used in different settings and approaches to measuring its influence that might be more widely applied, such as surveys and monitoring administrative data.

Background: Despite the cost of pharmaceuticals, studies assessing prices of osteoporosis drugs are lacking. This study examined trends in prices of osteoporosis drugs in the United States in the period 1988–2014, assessed pricing structure of osteoporosis drugs, and evaluated price trends before and after generic drugs market entry.

Methods: Data were derived from the U.S. Food and Drug Administration, the RedBook, the Centers for Medicare & Medicaid Services, and the Federal Supply Schedule (FSS). Descriptive statistics and segmented linear regression analyses were performed.

Results: In the period 1988–2014, osteoporosis drug prices increased faster than the inflation. The average wholesale price (AWP) of generic products at market entry represented 90 percent of the AWP for the corresponding brand. Prices of brand products continued to increase after generic entry. Drug prices showed a significant variation when compared with the brand AWP. The brand wholesale acquisition cost (WAC) was typically set at 83.3 percent of the AWP. Community pharmacies acquired osteoporosis brand drugs at a median of 80.5 percent of the brand AWP. Significant reductions in brand AWP were observed for Medicare Part B (78.5 percent of the brand AWP), generic National Average Drug Acquisition Cost (33.7 percent), and FSS (22.5 percent).

Conclusions: There are significant differences in the manufacturer prices, pharmacy acquisition costs and reimbursement rates of osteoporosis drugs. Pharmaceutical companies listed prices are higher than the pharmacy actual estimated acquisitions costs, and the prices used for reimbursement to providers. Generic drugs entry significantly drives down prices; still, prices of branded drugs facing generic competition continued to increase after generic market entry.

Objectives: To investigate the use, challenges and opportunities associated with using patient-reported outcomes (PROs) in studies with patients with rare lysosomal storage diseases (LSDs), we conducted interviews with researchers and health technology assessment (HTA) experts, and developed the methods for a systematic review of the literature. The purpose of the review is to identify the psychometrically sound generic and disease-specific PROs used in studies with patients with five LSDs of interest: Fabry, Gaucher (Type I), Niemann-Pick (Type B) and Pompe diseases, and mucopolysaccharidosis (Types I and II).

Methods: Researchers and HTA experts who responded to an email invitation participated in a telephone interview. We used qualitative content analysis to analyze the anonymized transcripts. We conducted a comprehensive literature search for studies that used PROs to investigate burden of disease or to assess the impact of interventions across the five LSDs of interest.

Results: Interviews with seven researchers and six HTA experts representing eight countries revealed five themes. These were: (i) the importance of using psychometrically sound PROs in studies with rare diseases, (ii) the paucity of disease-specific PROs, (iii) the importance of having PRO data for economic analyses, (iv) practical and psychometric limitations of existing PROs, and (v) suggestions for new PROs. The systematic review has been completed.

Conclusions: The interviews highlight current challenges and opportunities experienced by researchers and HTA experts involved in work with rare LSDs. The ongoing systematic review will highlight the experience, opportunities, and limitations of PROs in LSDs and provide suggestions for future research.

Objectives: Traditional economic evaluations for most health technology assessments (HTAs) have previously not included environmental outcomes. With the growing interest in reducing the environmental impact of human activities, the need to consider how to include environmental outcomes into HTAs has increased. We present a simple method of doing so.

Methods: We adapted an existing clinical-economic model to include environmental outcomes (carbon dioxide [CO2] emissions) to predict the consequences of adding insulin to an oral antidiabetic (OAD) regimen for patients with type 2 diabetes mellitus (T2DM) over 30 years, from the United Kingdom payer perspective. Epidemiological, efficacy, healthcare costs, utility, and carbon emissions data were derived from published literature. A scenario analysis was performed to explore the impact of parameter uncertainty.

Results: The addition of insulin to an OAD regimen increases costs by 2,668 British pounds per patient and is associated with 0.36 additional quality-adjusted life-years per patient. The insulin-OAD combination regimen generates more treatment and disease management-related CO2 emissions per patient (1,686 kg) than the OAD-only regimen (310 kg), but generates fewer emissions associated with treating complications (3,019 kg versus 3,337 kg). Overall, adding insulin to OAD therapy generates an extra 1,057 kg of CO2 emissions per patient over 30 years.

Conclusions: The model offers a simple approach for incorporating environmental outcomes into health economic analyses, to support a decision-maker's objective of reducing the environmental impact of health care. Further work is required to improve the accuracy of the approach; in particular, the generation of resource-specific environmental impacts.

In Husereau et al., the affiliation for Laura Sampietro-Colom was listed as “Hospital Clinic”. It should have instead been listed as, “Hospital Clinic, Barcelona, Spain”.