Patient-Driven RWD Outside of the Clinic: Demystifying the Capture of Home Reported Outcomes in Rare Diseases

Author(s)

Zhang C1, Luo N2
1Folia Health, Kansas City, MO, USA, 2Folia Health, Boston, MA, USA

OBJECTIVES: The inaugural home-reported outcomes (HROs) study in a rare disease must be focused on achieving important objectives: (1) develop an ‘Outcome Inventory’ of outcomes-of-interest to that population, including less-understood symptoms & behaviors; (2) understand the value of home-tracking and ideal features for the population in order to better design and implement future studies; and (3) describe the relationship between home-reported versus traditional outcomes in this population (ie. relationship between home-tracked treatments and claims-reported prescriptions).

METHODS: In launching HRO studies in multiple diseases, we have identified a set of best-practices for consideration by research teams: (A) Study Design; (B) Enrollment; and (C) Onboarding.

RESULTS: (A) Study Design must begin with assessing study objectives, followed by the immediate inclusion of participant perspectives. Too often, study design is nearly complete prior to soliciting community feedback. Starting with a simpler draft of objectives and study outline before engaging with the community enables the greatest possible flexibility, resulting in a truly patient-driven approach, as opposed to one with patients included as an addendum.

(B) Enrollment in Decentralized Trials is written about in other contexts. We add the following observations: To dramatically reduce time-to-launch, it is important to utilize a central IRB and obtain waivers from clinical site IRBs. There is strong utility in hybrid recruitment methods, inclusive of community channels and traditional methods. It is possible for participants to complete digital enrollment without clinician walkthrough.

(C) Onboarding participants is a key part of study launch, requiring significant care and resourcing. In our experience, onboarding successfully will drive retention and long-term engagement. Successful onboarding includes a well-oiled platform that guides participants through the first phases of the study; live support staffed by research professionals; and frequent positive reinforcement points, during the first stages of the study to ensure success.

CONCLUSIONS: Observational studies can benefit from following patient-centric best-practices.

Conference/Value in Health Info

2023-05, ISPOR 2023, Boston, MA, USA

Value in Health, Volume 26, Issue 6, S2 (June 2023)

Code

SA24

Topic

Patient-Centered Research, Study Approaches

Topic Subcategory

Patient-reported Outcomes & Quality of Life Outcomes, Prospective Observational Studies

Disease

Neurological Disorders, Oncology, Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory), Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)

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