Overcoming Refill Barriers for Rare Disease Patients

Our study, presented at the NORD Rare Summit, shows the positive impact interventions can have on medication adherence for people with rare disease.
Rare Disease V2

For people with a rare disease, their lives depend on their medication.

Given that so much is at stake, you might think that medication adherence is unlikely to be a problem for patients who are at high risk of complications and who have few treatment alternatives. However, while patients with orphan conditions may be more likely to be optimally adherent compared to patients with less acute conditions, many still struggle with adherence barriers from time to time.

In fact, a recent analysis by Accredo showed that more than 90% of rare disease patients were late to refill at least once despite proactive engagement via phone and/or text in an 18-month period.

The High Risk of Late Refills for People with Rare Disease

To better understand medication adherence among people who use medication for a rare disease, we examined trends in refill barriers in a sample of rare disease populations who fill their medications at Accredo. We looked at fill data for 17 different specialty orphan drugs, including:

  • Cystic fibrosis
  • Generalized lipodystrophy
  • Homozygous familial hypercholesterolemia
  • Urea cycle disorders
  • Phenylketonuria

Through proactive outreach, we collected data from more than 13,000 patients with rare disorders who received adherence interventions when they were at least one day late to fill their specialty, rare disease medication and who self-reported barriers to care. Examples of barriers to care include side effects from therapy, prior authorization process, missed doctor’s appointment, clinical care protocols, such as lab tests, and financial constraints. We gathered this data from the person’s medical record as well as prescriber and/or patient assessment conducted in Accredo’s therapy management system.

Through this study, we analyzed our efforts to prevent gaps in care when an Accredo rare disease specialist pharmacist engages with patients and prescribers to identify root cause for the refill gap, and then customize an action plan to address the identified barrier(s), whether they are financial, clinical, psychosocial, etc.

A pre/post analysis was conducted to assess the impact of the gap prevention on the length of refill gaps, frequency of refill gaps, fill timelines and days supply received before and after the gap prevention program was implemented.

To determine the impact of the gap in care on the patient, we assessed whether doses were missed during the refill gap. Rare patients may accumulate drug during hospital stays or through manufacturer assistance programs, in which case the patient stayed on therapy, but may have filled late due to supply accumulation. Our findings show that 30% of late refills did not involve missed doses. However, 70% evaluated did miss one or more doses at least once in the 18-month study period. This finding holds critical insight as we know even short gaps in adherence can add up over time.

For example, if a patient misses on average only 2 doses in a month -- nearly perfect adherence -- that patient has still missed nearly a month (24 days) of therapy over 12 months. In the setting of rare conditions, even these minor gaps in adherence can have a significant impact on disease management. This is why applying additional gap mitigation in the rare disease space is crucial.

Proactive Outreach Prevents Gaps in Care

Though understanding reasons for gap in care provides valuable insight into behavior, the most important outcome is using that insight to resolve refill gaps through continued treatment.  Once the cause for the gap is identified, Accredo collaborates with physicians and patients to implement an action plan to resolve the barrier and refill as quickly as possible.

Adding Accredo’s intervention strategies to reduce or prevent gaps in care to our already robust proactive refill outreach model decreased the length of refill gaps by 12.6 days.  Additionally, another 19% of patients who had one or more gaps before the program experienced zero gaps after the gap in care program started.  Overall, having gap in care prevention strategies in place increased the average days’ supply received per patient in the 6-month intervention period by 29 days. 

Through Accredo's Rare Disease TRC and our 14 other TRCs that focus on specialty and complex conditions, rare disease patients have the support of clinicians and patient care advocates who work to ensure rare disease patients receive therapy quickly, experience no lapse in therapy, and receive follow-up clinical care and education.


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