Below a few examples of our previous work 

- Strategic pipeline input 

- HEOR/HTA strategy and payer evidence generation

- Value communication planning and execution

- HTA strategy and dossier development

- RWE data generation using registries or databases

Strategic pipeline input including outcome measure assessment and testing

Provided strategic input into clinical trial protocols regarding payer evidence needs and requirements. This ranged from critical appraisal of trial design, outcome assessment using modelling and PRO/COA assessment and testing. Potential challenges were highlighted, and mitigation strategies suggested, if needed.

One supporting activity to help inform the choice of outcome measures in payer evidence projects, upcoming health economic analyses and clinical trials was the assessment and testing of different preference-based measures (PBMs). In collaboration with external experts e.g. previous use, validity, relevance and sensitivity for assessing the impact on the HRQoL on patients and caregivers were evaluated (e.g. Büsch et al 2022; Li et al 2022; Smith et al 2022).

HEOR/HTA strategy and payer evidence generation for a gene therapy launch

Developed the global HEOR/HTA strategy and payer evidence generation plan to support a gene therapy launch in rare diseases. Those were based on HTA needs and the critical assessment of potential key objections during the assessments. The execution of projects were done in close collaboration with multiple external partners and experts across all key areas e.g.

• systematic/targeted literature reviews e.g. to describe the burden of signs and symptoms (Bakidou et al 2020) or to identify proxy diseases for the economic model in the absence of disease specific information (Sudhapalli et al 2020)
• analysis of patient level trial data to (1) estimate the long-term impact of the intervention to be used in the economic model (Simons et al 2021a) and (2) to explore the evidence of a global symptom improvement for patients after treatment to support expert discussions (Simons et al 2020)
• clinician interviews to obtain information on the burden of disease and health care resource utilization in different Southern European countries as input data for the economic model (Saberian et al 2022)
• utility elicitation using clinician validated vignettes and DCE attributes in the general population in the UK and France to generate utilities for the economic model in the absence of trial-based utilities (e.g. Hanbury et al 2021; Smith et al 2021; Smith et al 2022)
• economic modelling for HTA submissions to demonstrate survival and quality of life benefits (Simons et al 2021b), which was validated with clinical experts during a regional and global ad-board as well as with HEOR experts from 8 countries during an expert meeting (e.g. Sopena et al 2021)

To support the key initiatives, a qualitative interview study with caregivers provided insights into the HRQoL of individuals across different health states in an economic model (Williams et al 2022). In addition, information on the time for caring suggested that >100 hours per week were spend on caring and 75% of caregivers had to reduce or stop working (Büsch et al 2022) and described the overall HRQoL impact on caregivers (Skrobanski et al 2021).

The evidence was used in several on-going HTA submissions with positive outcome e.g. recommendation by NICE.

Value communication planning and execution

Developed the evidence dissemination plan and orchestrated the scientific communication at international congresses and in peer reviewed journals. The early external communication on congresses allowed the usage of the evidence for disease awareness activities, informed other evidence projects and supported the value messaging and local market access efforts.

Over the course of 2 years, over 20 poster/oral presentations were given and build the value story on the topics of the burden of disease, HRQoL, utilities, health care resource use, the economic burden of caregiving as well as the long-term effects of treatment and impact on global symptom improvement. In addition, over 10 peer-reviewed manuscripts were published building a robust evidence base on literature reviews, trial data analysis, qualitative and quantitative data collection from different stakeholder perspectives (including clinicians and caregivers), and economic modelling for HTA submissions.

HTA strategy and dossier development

Developed the local HTA strategies and dossiers for >10  submissions to obtain or maintain pricing and reimbursement for interventions within immunology, oncology and hepatitis C in Sweden (all successful). Those were based on HTA landscape mapping including different access approaches and HTA competitor analysis and included pricing scenarios and payer proposals for three party negotiations, where needed.

Supported HTA submission development globally e.g. to NICE, SMC, HAS, INFARMED, PBAC and CADTH across a wide range of products and indications including immunology, virology and rare diseases.

RWE data generation using registers or databases

Conducted major register linkages involving several government agencies e.g. within immunology with almost 1 million study participants and within chronic hepatitis C with approx. 50.000 patients and 250.000 matched comparators (e.g. Büsch et al 2020). Analysed the data in SAS or designed different analysis to be conducted by external collaborators. Worked with databases from Sweden, Germany, France and the US within immunology, respiratory, oncology and rare diseases.