How Stakeholders Can Optimize Future Strategies, Care Experiences for Patients With Orphan Diseases

According to research presented at AMCP 2021, although there is interest in expanding support offerings in the future, current care management and disease state education remain the mainstays of support among health care stakeholders for patients with orphan diseases.

“As the number of orphan drugs gaining FDA approval increases, health care payers, employers, and providers are considering opportunities to better support patients and caregivers impacted by orphan diseases,” Erin Lopata, PharmD, MPH, and colleagues explained.

To better understand current as well as future strategies aimed at optimizing both patient-reported outcomes (PROs) and the overall care experience for patients with orphan disease, Dr Lopata and her team conducted a survey with payer, provider, and employer decision makers. These decision makers were all asked about their experiences and activities in the orphan disease space.

According to the findings, care management programs and disease state education for patients with orphan diseases were found to be the top resources that are currently offered by payers (80% and 68%, respectively), providers (81% and 90%), and employers (91% and 90%).

“In the next 18 months, many more payers, providers, and employers expect to link patients with community resources and patient advocacy groups, provide financial resources, and offer caregiver support for patients with orphan diseases,” they wrote.

Additional findings show that although providers are looking to expand strategies to identify undiagnosed patients, there were fewer payers and employers that currently have or plan to implement initiatives. Further, according to the survey, the majority of payers (74%) do not currently track PROs for their patients with orphan diseases. However, providers on the other hand are tracking PROs for the following:

• symptom burden (71%);
• pain (62%);
• health-related quality-of-life (62%); and
• activities of daily living (52%).

“Care management and disease state education remain the mainstays of support for patients with orphan diseases across healthcare stakeholders,” they concluded. “While many providers are currently evaluating PROs for their patients with orphan diseases, use has been limited by payers.”

—Julie Gould  

Reference:

Lopata E, Terrone C, Gopalan A, Ladikos N, Richardson T. Understanding payer, provider, and employer resources and opportunities to support patients and caregivers impacted by orphan diseases. Poster presented at: AMCP 2021, April 12-16, 2021; Virtual.