Evidence need:

With IMLYGIC the first-in-class treatment for injection directly into melanoma tumours, uncertainty existed amongst clinicians around which patients would benefit most from treatment with IMLYGIC and how this should be incorporated into the overall therapeutic approach. Data was needed to demonstrate the use of IMLYGIC in the real-world and the associated clinical outcomes

Solution:

A site-based, multi-centre retrospective chart review across seven academic oncology centres across the United States, allowing the collection of medical chart information for 83 eligible patients.

Adelphi Real World supported protocol development, electronic case report form (CRF) design and site set up, including ethics submissions and budget/contract negotiations

To assess the use of IMLYGIC in combination with immunotherapy, three cohorts were analysed:

• IMLYGIC prescribed after immunotherapy (n=22)
• IMLYGIC prescribed concurrent with immunotherapy (n=32)
• IMLYGIC prescribed without immunotherapy (n=29)

Key findings:

• Over two thirds of patients treated with IMLYGIC received it with immunotherapy, either concurrently or following immunotherapy, with few patients discontinuing treatment
  due to adverse event, demonstrating the safety of using this combination regimen
• The use of IMLYGIC across seven high treatment centres demonstrated the value of this as a treatment strategy for unresectable metastatic melanoma

Sun et al. (2020). Observational study of talimogene laherparepvec use in the anti-PD-1 era for melanoma in the US (COSMUS-2). Future Medicine, vol. 7 (2).

Evidence need:

There were limited published observational data describing both clinical outcomes and treatment persistence rates for anti-TNFs used to treat ulcerative colitis (UC), particularly for golimumab. This study looked to evaluate treatment persistence, switching patterns, and colectomy outcomes between golimumab and adalimumab in the real-world setting.

Solution:

A site-based, multi-centre retrospective chart review across sixteen NHS centres in the United Kingdom generated data for 183 patients with at least 6 months of follow up data post treatment initiation

Feasibility assessments were initially used to confirm the variables and endpoints appropriate for this real-world study. Following contracting and ethics approval, sites completed electronic case reports forms to provide clinical history, treatment patterns and effectiveness outcomes for eligible patients

Efficacy outcomes used in clinical trials, such as colonoscopy, were not routinely captured in routine clinical practice, so treatment persistence and colectomy free survival were used as a proxy

Key findings:

• Demographic and baseline characteristics were similar between the two treatment arms and persistence rates at 12 months were around 64% for both groups with no statistically significant differences. Colectomy-free survival was relatively similar within 1 year (92% for both arms)
• The real-world data collected in this study demonstrate comparable clinical effectiveness outcomes for golimumab compared with adalimumab 12 months following treatment initiation

Hogue et al. Treatment persistence and colectomy-free outcomes in patients with ulcerative colitis receiving golimumab or adalimumab: a UK experience. BMJ Open Gastroenterol 2020; 7(1): e000476

Evidence need:

The CDK 4/6 inhibitor palbociclib has demonstrated efficacy in clinical studies in patients with hormone receptor-positive (HR+), human epidermal growth factor 2 (HER2)-negative advanced/metastatic breast cancer (ABC/MBC). However, data in the real-world were scarce in 2017, and the IRIS study was therefore needed to demonstrate the associated real-world outcomes

Solution:

A global retrospective chart review which examined patient demographics, clinical characteristics, treatment patterns and outcomes for patients receiving palbociclib combinations was developed

Anonymised clinical chart data were collected from physicians in 20 countries across Europe, the US, Canada, Latin America and Asia through an electronic case report form (eCRF)

Patients diagnosed with HR+/HER2- ABC/MBC who started palbociclib combination therapy within a pre-specified index window were considered eligible for inclusion

Progression-free and survival rate analyses were planned for patients treated with either fulvestrant or aromatase inhibitors in combination with palbociclib

Key findings:

• Several country-level manuscripts have been developed. Findings from the US dataset showed palbociclib in combination with either AI or fulvestrant resulted in favourable progression-free and survival rates
• Results indicate concurrence between palbociclib’s outcomes in the clinical setting and in the real-world setting

Taylor-Stokes G, Mitra D, Waller J, et al. Treatment patterns and clinical outcomes among patients receiving palbociclib in combination with an aromatase inhibitor or fulvestrant for HR+/HER2-negative advanced/metastatic breast cancer in real-world settings in the US: Results from the IRIS study. Breast. 2018 Oct 20;43:22–7.

Evidence need:

With the approval of the first-in-class treatment for sickle cell disease (SCD) due at the end of 2019, it was important to generate data showcasing the burden and unmet need within this disease area. Objectives centred around the physical, emotional and social impact of disease, as well as highlighting the current poor management of vaso-occlusive crises (VOCs) in SCD patients

Solution:

A global health survey in SCD patients and healthcare providers was developed. A multi-channel recruitment strategy across sixteen different countries was executed, utilising existing sponsor relationships with a number of country-level patient advocacy groups (PAGs). The study was endorsed by a steering committee of worldwide experts

In order to ensure representation of paediatric respondents in the study, it was necessary to develop a ‘proxy survey’ which a caregiver could complete on behalf of the patient to ensure their burden was represented in the findings

Over 2,000 patients and 350 healthcare professionals were enrolled, representing the unmet need of SCD across Europe, North America, South America, Asia and Africa

Key findings:

• SWAY was the first survey to assess the impact of SCD on patients’ lives, with high impact reported on patients’ emotional wellbeing and school achievement
• The findings emphasised the high VOC burden experience by patients, often underreported, and therefore highlighting the urgency for novel treatment solutions

Osunukwo I, Andermariam B, Minniti, CP et al. Impact of sickle cell disease on patients’ daily lives, symptoms reported, and disease management strategies: Results from the international Sickle Cell World Assessment Survey (SWAY). Am J Hematol. 2021; 96(4):404-417

Evidence need:

Lenalidomide is often used off-label as maintenance treatment for patients with MM post-ASCT. Although survival data had demonstrated a benefit associated with maintenance vs. no maintenance treatment, the impact on quality of life (QoL) was unknown

Solution:

A combined retrospective chart review and cross-sectional patient-reported outcome (PRO) study was designed. A total of thirteen academic centres in the US and Canada participated, leading to the recruitment of 303 patients overall

PROs captured included the EQ-5D-5L, EORTC QLQ-C30, and the EORTC QLQ-MY20, allowing for an assessment of burden of disease across several different validated measures

QoL measures were assessed across:

• Baseline patients (30–100 days post stem cell transplant and had not yet initiated maintenance treatment)
• Maintenance patients (>100 days post stem cell transplant and had initiated on maintenance treatment)
• No maintenance patients (>100 days post stem cell transplant but had not initiated on maintenance treatment)

Key findings:

• Differences between groups were explored with ordinary least squares regressions, with few differences observed between those patients who did and did not receive maintenance therapy
• Findings overall showed maintenance therapy to not be associated with notable QoL detriment, therefore supporting the benefits associated with this treatment regimen

Tay J, Norkin M, Buadi F et al. Health related quality of life for multiple myeloma patients according to treatment strategy after autologous stem cell transplant: a cross-sectional study using EORTC, EQ-5D and MY-20 scales. Leuk Lymphoma. 2019; 60(5): 1275–1282

Evidence need:

Previous studies had demonstrated the impact of fracture in terms of direct costs in the acute setting but there was limited data on the longer-term impacts to the individual, and this study aimed to address this issue

Objectives included understanding the impact on activities of daily living, productivity and caregiver use

Solution:

A cross-sectional patient survey of patients capturing the burden of disease in relation to activities of daily living, productivity loss, and caregiver support

A matched electronic case record form (eCRF) was completed by sites to provide clinical history

Cohorts of women with and without osteoporotic fractures

41 research sites were contracted, covering 5 countries and enrolling more than 1200 patients (Germany, Spain, Australia, South Korea and USA)

Key findings:

• The study showed differential impact in productivity and activities of daily living between cohorts and differences in caregiver use and results were presented at ASBMR 2022
• A manuscript has recently been accepted and will be published in 2023

Yeh EJ, Rajkovic-Hooley O, Silvey M et al. Impact of fragility fractures on activities of daily living and productivity in community-dwelling women: a multi-national study. Osteoporos Int 2023. https://doi.org/10.1007/s00198-023-06822-7

Evidence need:

Conduct a patient preference study to understand the relative importance that COPD patients place on alleviating symptoms vs. conventional clinical endpoints (i.e. FEV1/exacerbation frequency), to inform clinical trial endpoints meaningful to patients.

Solution:

A gold-standard DCE in five countries (US, UK, FR, AU, JP) comprising literature review, qualitative research and quantitative assessment

NICE provided scientific advice for the first time on a patient preference study design, informing subsequent publication on the use of patient preference studies in HTA decision making

Recruitment of 1050 patients through five PAGs and patient panels

Clinical input on design from two key opinion leaders in France and the US

Key findings:

• ‘Novel’ clinical endpoints such as cough, mucus, sleep quality are more important to patients than clinical endpoints typically assessed (i.e. exacerbations)
• This study is one of ten case studies included in the PREFER recommendations, for patient preference, which subsequently received EMA qualification opinion

Nigel S. Cook, Gerard J. Criner, Pierre-Régis Burgel, Katie Mycock et al. People living with moderate-to-severe COPD prefer improvement of daily symptoms over the improvement of exacerbations: a multicountry patient preference study. ERJ Open Research Apr 2022, 8 (2) 00686-2021

Evidence need:

An in-depth exploration of perceptions and satisfaction with an ultra-rapid insulin amongst adults with diabetes and endocrinologists/diabetologists was needed to understand the initial experience associated with switching to Lyumjev® to demonstrate this treatment strategy as acceptable and beneficial to patients

Solution:

A multi-market qualitative study utilising in-depth semi-structured interviews with HCPs and patients in Germany and the US

Participants were recruited via panels and physicians and external moderators conducted interviews with respondents using a discussion guide co-developed with a key opinion leader. Overall, 22 patients and 12 HCPs were recruited

A thematic analysis was undertaken to identify and report patterns in the data, with specialist software used to develop a robust and consistent code-frame

Key findings:

• Primary themes and subthemes were identified and reported, with key messaging centring around the valued attributed associated with Lyumjev®

Stuckey HL, Schapiro D, de Oliveira CP et al. Perception of real world Lyumjev® (insultin lispro-aabc) by patients and healthcare providers: A qualitative study. Poster presented at ADCES 2022

Evidence need:

Multiple medication options with different forms of administration & dosing regimens are now available as well as the option of active surveillance only (‘watch & wait’). The study aimed to generate insights into patient preferences for active medication versus active surveillance ‘watch & wait’ treatment options and preferences for different forms of administration

Solution:

Cross-sectional survey with n=153 patients with newly-diagnosed EOC conducted in the US

Treatment preference was assessed via a time trade-off (TTO) simulation

Adaptations to the traditional TTO methodology were implemented:

• Conducted with patient rather than general population
• Full health trade off adapted to current health trade off
• Trade off based on time to disease progression rather than death

Survey also included a range of PRO tools to examine the burden of disease amongst patients and content aimed at ascertaining the existence of sociodemographic health disparity.

Key findings:

• Results demonstrated that patients have clear preferences for specific treatment approaches and specific formulation and dosing options. Health utility values for different treatment options were generated for economic modelling purposes
• Multiple poster presentations and manuscript in development

Wethington SL, Shukla S, de Courcy J et al. VOCAL (Views of Ovarian Cancer Patients – How Maintenance Therapy Affects their Lives) Study: Patient Preference for Treatment Formulation and Administration. Poster presented at European Society of Gynaecological Oncology 2022