Real-world evidence generation from patients, their caregivers and physicians supporting clinical, regulatory and guideline decisions: an update on Disease Specific Programmes

Figures & data

Figure 1. Overview of how DSPs are designed, implemented and analyzed.

CSC, caregiver self-completion form; DSP, Disease Specific Programme; PRF, patient record form; PSC, patient self-completion form; QC, quality control

Source: Adapted with permission from the DSP methodology previously published in 2008.⁹ To access the original source, please, visit https://www.tandfonline.com/

Table 1. Current key RWE themes covered by DSPs with example findings supporting clinical, regulatory and guideline decisions.

Topics	Theme 1: Using RWE to inform clinical practice
	Real-world use of/informing CPGs	DSPs fed into CPGs and demonstrated how well CPG recommendations were reflected in real-world clinical practice.^18^,^29^,^32^,^52 DSPs provided evidence informing the appropriateness of CPGs in real-world clinical practice.^53
	Treatment/diagnostic pathways	DSPs highlighted treatment pathways chosen by physicians in everyday practice in diverse patients from those with rare conditions to common cancers and the reasons for physicians’ choice including if/why CPGs were (not) being followed.^16^,^17 A delay to consultation and/or diagnosis was shown to lead to disease progression in patients with dementia.^54
	Theme 2: Patient and caregiver engagement
	Alignment of patient-physician perspectives	Gathering patient perspectives on disease severity, symptom recognition, treatment satisfaction and disease control identified if/where patient and physician attitudes were aligned and the impact of misalignment.^23^,^48^,^55–57 DSPs showed that the degree of patient and physician alignment in attitudes on treatment satisfaction could positively influence patient overall healthcare outcomes.^26^,^48^,^50^,^56
	Patient-physician SDM	Collecting patient insights about aspects of their treatment helped HCPs to recognize where improvements to treatment could be made and/or more patient education was needed.^31^,^41^,^42^,^58–62
	Caregiver burden	By understanding caregiver perspectives, physicians could identify treatment priorities and potential areas for support and education to improve the QoL of both patients and their caregivers.^27^,^38–40
	Patient adherence	Features that patients and physicians prioritized in healthcare technologies informed their design, ensuring they were fit for purpose, convenient to use and were, therefore, likely to improve patient adherence to treatment.^30 Patient education and choice of device tailored to their needs showed that these could improve patient confidence, adherence and health status.^58
	Theme 3: Role of RWE in supporting HTAs and regulatory submissions
	Mapping real-world PROMs to measures used in CT/HTA contexts	DSP data were used to cross-walk between two different disease-specific tools, each of which was included in independent RCTs, thus enabling indirect treatment comparisons to be made.^63 Cross-walk analysis was undertaken from a disease-specific tool to a measure of general health for use in cost-effectiveness comparisons for HTA submission.^64^,^65
	Supporting regulatory submissions	DSPs provided evidence supporting regulatory submissions with regard to real-world clinical practice, for example, in prostate cancer and nocturia.^66^,^67
	Theme 4: Informing value-driven healthcare decisions
	Economic burden of illness	Association between patients’ rating of disease severity and the economic burden of disease was found, indicating that patients’ own perspective on their disease severity could also signal its economic impact.^22 Exploring the relative risk of developing more severe disease or related conditions indicated which patients should be targeted by screening programmes.^24 Studies on HCRU, work productivity and associated costs allowed for calculation of the economic cost of a disease in the real world.^25^,^37 Investigating treatment pathways allowed for payors to verify that treatments were being prescribed only to those patients with clinical need.^17
	Humanistic burden of illness	Identifying symptoms which were the most ubiquitous or had large impact on daily activities informed physicians which symptoms to target to best improve patient HRQoL.^20 High patient-reported disease burden was found to reduce patient satisfaction with treatment.^68 A greater understanding and use of PROMs by physicians could enable insights into how the person with the condition is feeling and provide clues regarding what might matter most to that person.^57
	Real-world drug effectiveness	Understanding of the individual patient disease phenotype or how disease manifested meant that treatment could be tailored better to the individual patient.^68
	Theme 5: Real-world patient subgroup differences
	Health outcomes in different patient subpopulations	Patient subgroups were identified, stratified by disease severity or phenotype, patient sex, etc., facilitating assessment of any differences in management strategies and outcomes across these subgroups.^18^,^19^,^69 By comparing treatment pathways of patients in different subgroups, it was possible to determine whether different subgroups received different care.^19
	Theme 6: Therapeutic inertia/unmet needs
	Patient unmet needs and gaps for future research	Identifying diseases with high unmet patient needs highlighted areas where novel therapies were needed and/or indicated where patients could be facing obstacles in the healthcare system.^11^,^21^,^28^,^70

CPG, clinical practice guideline; CT, clinical trial; DSP, Disease Specific Programme; HCP, healthcare professional; HCRU, healthcare resource utilization; HRQoL, health-related quality of life; HTA, health technology assessment; PROM, patient-reported outcome measure; QoL, quality of life; RCT, randomized controlled trial; RWE, real-world evidence; SDM, shared decision-making.

Figure 2. A map of the RWD source landscape and the placement of DSPs within it.

ClinROM, clinical-reported outcome measure; CROM, caregiver-reported outcome measure; DSP, Disease Specific Programme; EMR, electronic medical record; ObsROM, observer-reported outcome measure; PROM, patient-reported outcome measure; RWD, real-world data

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