Determinants of managed entry agreements in the context of Health Technology Assessment: a comparative analysis of oncology therapies in four countries

BACKGROUND: Managed Entry Agreements (MEAs) are increasingly used to address uncertainties arising in the Health Technology Assessment (HTA) process due to immature evidence of new, high-cost medicines on their real-world performance and cost-effectiveness. The literature remains inconclusive on the HTA decision-making factors that influence the utilization of MEAs. We aimed to assess if the uptake of MEAs differs between countries and if so, to understand which HTA decision-making criteria play a role in determining such differences. METHODS: All oncology medicines approved since 2009 in Australia, England, Scotland, and Sweden were studied. Four categories of variables were collected from publicly available HTA reports of the above drugs: (i) Social Value Judgments (SVJs), (ii) Clinical/Economic evidence submitted, (iii) Interpretation of this evidence, and (iv) Funding decision. Conditional/restricted decisions were coded as Listed With Conditions (LWC) other than an MEA or LWC including an MEA (LWCMEA). Cohen's κ-scores measured the inter-rater agreement of countries on their LWCMEA outcomes and Pearson's chi-squared tests explored the association between HTA variables and LWCMEA outcomes. RESULTS: A total of 74 drug-indication pairs were found resulting in n = 296 observations; 8 percent (n = 23) were LWC and 55 percent (n = 163) were LWCMEA. A poor-to-moderate agreement existed between countries (-.29 < κ < .33) on LWCMEA decisions. Cross-country differences within the LWCMEA sample were partly driven by economic uncertainties and largely driven by SVJs considered across agencies. CONCLUSIONS: A set of HTA-related variables driving the uptake of MEAs across countries was identified. These findings can be useful in future research aimed at informing country-specific, "best-practice" guidelines for successful MEA implementation

Health related quality of life aspects not captured by EQ-5D-5L: results from an international survey of patients

In this paper we discuss and present evidence on whether a generic Health Related Quality of Life (HRQoL) measurement tool, the EQ-5D-5L, captures the dimensions of quality of life (QoL) which patients consider significant. METHODS: An online survey, of individuals with a chronic condition, mainly breast cancer (BC), blood cancers (BLC), rheumatoid arthritis (RA), asthma, and rare diseases (RD) was conducted to collect data on HRQoL and important QoL aspects that respondents thought were not captured by the EQ-5D-5L. Patient organisations across 47 countries were invited to voluntarily share the survey tool with their membership network. RESULTS: 767 responses from 38 countries showed that important QoL aspects were not captured by EQ-5D-5L for 51% of respondents, including fatigue (19%) and medication side effects (12%), among others. Fatigue (17%) was also the most commonly reported QoL aspect that changed over the course of patients’ illness, suggesting that the current version of the EQ-5D-5L might miss capturing significant clinical changes in important QoL domains. CONCLUSIONS: Utilisation of the EQ-5D-5L in HRQoL measurement raises inconsistencies in capturing QoL attributes and changes in disease-specific patient populations. Further research is needed to clarify the extent to which other generic HRQoL measurement tools capture the aspects of health that really matter for patients

Differentiation of health related quality of life outcomes between five disease areas; results from an international survey of patients

Objectives: Health-related quality of life (HRQoL) data generated by generic, preference-based instruments (i.e., EQ-5D) are highly demanded in health policy decision making, because they allow for direct comparisons of HRQoL outcomes between disease areas. We aimed to quantify HRQoL outcomes in breast cancer (BC), rheumatoid arthritis (RA), multiple sclerosis (MS), rare cancers (RC), and rare disease (RD) patients and understand the patterns that differentiate HRQoL outcomes between these disease areas, and more specifically between rare and more common disease population groups. Methods: An international, Web survey of patients measured HRQoL (EQ-5D-5L), self-perceived health (EQ-5D-5L Visual Analogue Scale), and additional QoL dimensions, such as patient disability level. Results: We received 675 completed responses. Average utility loss was 53.5 percent, 32.5 percent, and 33.3 percent for RD, RA, and MS patients, respectively, in contrast to 18.6 percent for BC and RC patients. Statistically significant differences (p < .05) were observed between disease groups in all EQ-5D-5L domain outcomes, apart from that of “Anxiety/Depression.” Severe and/or extreme problems were reported in performing usual activities for RD and RC (34 percent and 13 percent of overall problems reported respectively), mobility for MS (18 percent), pain/discomfort for RA (13 percent), and anxiety/depression for BC (7 percent) patients. Conclusions: We demonstrated significant differences in the dimensions that drive HRQoL outcomes between rare and more common diseases and showcased that the same EQ-5D utility may reflect very different severities depending on the patient population under investigation. Future research should examine whether outcomes in other, critical HRQoL domains not included in generic measures also highlight significant differences across disease areas

The Impact of External Reference Pricing within and across Countries

An assessment of the impact of external reference pricing (ERP) systems on important health system goals such as availability, affordability and diffusion/utilisation of pharmaceuticals; and an analysis of the impact that ERP systems have at the domestic and international levels, particularly considering their likely spillover effects

Using IMPrESS to guide policy change in multiple sclerosis

The International MultiPlE Sclerosis Study (IMPrESS) studied the significant impact of multiple sclerosis (MS) on the health and well-being of both people with the disease and their caregivers, along with its broader socioeconomic impact. Results confirmed that there is an urgent need to achieve better outcomes for people with MS. This paper uses results from the IMPrESS to present new international evidence on the socioeconomic burden of MS and discuss the merits of a likely paradigm shift in the management of MS towards the use of better (and more accurate) diagnostic follow-up to monitor disease progression and the earlier use of disease-modifying treatments (DMTs) to achieve better clinical, quality-of-life and socioeconomic results for individuals

Moving towards better outcomes in Multiple Sclerosis by addressing policy change

This study aims to estimate the societal economic burden and health-related quality of life (HRQoL) of patients with multiple sclerosis (MS) and evaluate the role of receiving early disease modifying treatment (DMT) (i.e., within 12 months of a single neurological attack) towards better disease outcomes

The IMPrESS (International Multiple Sclerosis Study);socio-economic burden, health related quality of life andexperiences of multiple sclerosis patients in France

OBJECTIVES: This study aimed to capture the socio-economic costs, Health Related Quality of Life (HRQoL) and experience of Multiple Sclerosis (MS) patients in France. METHODS: An online survey of non-institutionalised MS patients collected data on demographic, disease and clinical related variables, Disease Modifying Drug (DMD) consumption, healthcare resource and informal care utilisation, productivity losses and HRQoL (EQ-5D-5L, EQ-5D-5L VAS) and patients’ experience, gauged by questions on treatment satisfaction, future treatment expectations, caregiving arrangements, preferred sources of information for MS and preferred strategy for deciding about their therapy. Three national MS organisations were invited to voluntarily share the survey with their members. RESULTS: Using 2014 as a reference price, a sample of 97 individuals, demonstrated an average annual MS patient cost of €24,403 (±18,538), 57% of which (€13,838, ±15,796) accounted for indirect costs due to productivity losses and 36% and 7% for mean annual direct, health and non-health care costs respectively. Indirect costs were primarily driven by permanent work disability and temporary sick leave (42% and 12% of mean annual cost respectively), whereas direct costs were shaped by DMD utilisation, formal/informal caregiving and inpatient care (31%, 7% and 5% of mean annual cost respectively). Mean EQ-5D-5L index and VAS scores were 0.49 (±0.3) and 60 (±22.6) respectively, whereas patient reported outcomes underscored a moderate satisfaction (7.4 out of 10) with the care received by the national health system, the need for a new therapy that targets symptoms of Mobility and Fatigue/Weakness (43% and 40% of respondents respectively) and the need to receive formal/informal care (7%). CONCLUSIONS: MS poses a significant cost burden for the French society, with medication and indirect costs representing 88% of total average costs and substantial unmet needs being reported in the clinical management and social care currently received by MS patients in France