Sticking to the medicine in rare disease

We all know how important it is for rare disease patients to take their medications as prescribed. Many rare diseases have no cures and the treatment options available are usually for managing symptoms, rather than treating the cause. Not taking the right medications at the right time can lead to symptoms returning.

Known reasons for treatment non-adherence

Why patients don’t take their medicines is complicated. A study found that reasons for non-adherence can be based on a multitude of factors, including: how well patients understand their health condition and the effects of treatment; how strictly physicians prescribe drug regimens and how well they explain the benefits of sticking to medication; and how well-equipped healthcare systems are.

In rare disease, we know from speaking to our patient communities that the way a particular medication is taken or delivered can also affect how closely patients stick to it. For example, among members of our hemophilia community, some members want to switch treatments because they are tired of infusing intravenously.

Another key factor to consider is medicine side effects. Most of the therapy options available to myasthenia gravis patients, for example, are associated with significant side effects, including nausea, gastrointestinal problems, increased infection risk, weight gain, or liver damage. Having to deal with such side effects may be too much for some patients and it may affect how closely they follow their treatment plans. A recent study we conducted with our cystic fibrosis community showed a similar result. Not all patients asked were taking their medication as prescribed, with some missing doses anywhere from once a month to as often as once a day. The reasons they gave included lack of appetite in the morning (they need to be taken with food) and not being able to keep the medicine down.

Raremark's research into rare disease treatment experiences

To dig deeper, we’re running two major research studies with our patient communities. Our digital study into treatment satisfaction is focused on five rare disease areas: cystic fibrosis, hemophilia, idiopathic pulmonary fibrosis, myasthenia gravis and sickle cell.  We want to see what patients think about the treatments they are on now and any medications they have taken in the past. We also want to compare the opinions of people taking newly available treatments with people on older, more established medications.

Our longitudinal study will focus on non-adherence to medications in rare disease patients using a validated, patient-reported outcome (PRO) tool. The Medication Adherence Reasons Scale (MAR-Scale) can reliably tell us why patients may not be taking their medications. This is the first time the MAR-Scale will be used for rare disease research and we’re collaborating with the creator of the tool, Dr Elizabeth Unni.

Understanding the reasons why rare disease patients may be taking their medication incorrectly or missing doses can help researchers make informed choices around the types of treatments they develop in the future. And by conducting a longitudinal study to assess patient behavior over an extended period, we can start to build a clearer picture of real-world adherence to meds.

Sign up below for updates on both of these studies.

Get in touch

Interested in hearing more? Feel free to reach out to us, and we’ll make sure to get back to you as soon as possible.