Video
Rheumatic Disease Challenges and Goals of Therapy
Author(s):
Transcript:
Amar Majjhoo, MD: There are many challenges, as most rheumatologists know, dealing with rheumatic diseases, and this is true even prior to the pandemic. Our diseases are often challenging to diagnose. They’re multifaceted, and we lack all-encompassing diagnostic tests that are both absolute sensitive and specific; we have to rely on clinical diagnosis, and that’s not always easy. That’s one of the enjoyments of our field as well, but certainly this is challenging.
Once we’ve made a diagnosis, we have chronic diseases without cures, and although our treatment options have expanded in many different diseases, we still don’t know what treatment a particular patient is going to respond to best. When we’re thinking about first-line therapy or sequential therapy, second, third line, we don’t have a diagnostic test yet that will help us predict which drug is the best for that particular patient. Similarly, as I mentioned, our diseases are multifaceted and complex, so assessing a patient is equally challenging.
Determining how our patient is doing on therapy can be very time consuming and tedious, and we would benefit if we had biomarker that gave us a snapshot, a global assessment of how our patient is doing. That would help aid and facilitate our treatment of these patients in the COVID-19 [coronavirus disease 2019] setting. Although our treatment options have expanded in many different diseases, we’re still lacking treatment options in many of the diseases we treat.
The good news is there are a lot of clinical trials ongoing in some of these diseases, and it looks like in the future we’ll have more treatment options. Once we’ve picked a treatment option for our patient, and even when they’re responding, patient adherence and compliance is often an issue. Also, one of the biggest challenges we are continually facing is accessibility and costs associated with these medications.
The goals of therapy and the factors that guide therapy in patients with psoriatic arthritis are many. Each patient we’re confronted with in that room in that moment is different, and our treatments are individualized. We really have to have a good appreciation that this is a heterogeneous disease; it’s multifaceted. Each patient is unique.
The things that we look at are the domains that are involved specifically to psoriatic arthritis. We’re looking at skin involvement, peripheral joint involvement, spinal involvement, enthesitis and dactylitis. We’re looking at the severity of several symptoms within each domain. Specifically, we’re often looking at the severity of skin and joint involvement to help guide which treatments we may choose. We’re keeping this in the back of our minds, and we’re rapidly trying to formulate which is the best therapy.
We’re doing that in the context of other factors, such as comorbidities. Does the patient have inflammatory bowel disease [IBD]? Have they been diagnosed formally with inflammatory bowel disease, or are you suspicious that they have inflammatory bowel disease? That often happens as well. We see patients who have not been formally diagnosed with IBD, but they give this long-standing history of their abdominal pain, bloating, diarrhea, and maybe even mucous in their stool and sometimes even blood, but they’ve not been formally diagnosed.
Here we’re thinking that the patient may also have inflammatory bowel disease. They may have more subtle symptoms as well. They may provide a family history as well. This is one of the comorbidities that we think about. We also think about other factors, such as previous history of infections, diabetes, fatty liver disease. Then obviously we must factor into the equation patient preferences. Does the patient want an oral agent? Do they want something that they can self-administer at home?
Are they OK with subcutaneous injections? Do they prefer something that is administered by a health care professional, and the frequency of administration of the treatments. We also must look at what have they tried and failed in the past? Are there data that can help us guide our treatment decisions in terms of clinical trials, looking at failed therapies in the past, and what agent we should use next?
For those of you practicing rheumatology, you know that our field, our specialty, is challenging. This was true even prior to the pandemic that we’re in. Barring the issues that have come about with the pandemic, there are many challenges in the practice of rheumatology and treating our patients with rheumatic diseases. To begin with, we are often confronted with the fact that we don’t have all-encompassing tests that are very sensitive and at the same time specific to be able to accurately diagnose our patients with particular conditions.
A lot of our diagnoses are based on clinical judgment, classification criteria and diagnostic criteria, so there’s some room for practicing the art of medicine. This is one of the enjoyable parts of our specialty, but at the same time challenging in terms of making a definitive diagnosis many times. Once we’ve made a diagnosis, we’re very fortunate that in many of our diseases the treatment options have expanded in the last several years.
We are left in many diseases without treatment options, but the good news is there are a lot of clinical trials in progress even within these diseases, and so there may be hope in the future with some of these diseases that we don’t have approved medications for. In terms of the diseases for which we do have treatment options, we still don’t have cures. We are confronted with the fact that our patients have chronic diseases without cures, and we don’t often know what is the best therapeutic option for that patient.
We don’t have a way of predicting what drug a patient is going to respond to best as first line or as second or third line as we sequentially treat these patients with various agents; this is also another one of our challenges. Another of our challenges is that our diseases are oftentimes multifaceted, and our assessments of these patients can be very cumbersome, very time consuming, and require a clinician to be a good clinician.
It would be nice if we had biomarkers that would give us a global assessment of how our patient with a particular disease is doing, and we could base changing therapy or continuing therapy based on perhaps a number. That would be helpful for us. Some of the more common challenges that we deal with also include that our patients are oftentimes not adherent to medications for a variety of reasons.
We’re very fortunate that our treatment options have expanded. We have many FDA approved drugs within different diseases, and over the years these treatments have become very targeted, very select. They oftentimes can produce good results, and they’re cleaner, perhaps, than some of the drugs that we were using in the past that were not specifically developed for those particular diseases. In general, our efficacy has improved, our toxicity has reduced, Despite that, patients are oftentimes not adherent with therapy over the long term for a variety of reasons.
Studies support that, when you look at claims data, that patients often do not refill their medications or stay on therapy for a variety of reasons. This may include loss of response over time. It may include [adverse] effects. It may include coverage issues. It may include that the patient is feeling good and decides to stop therapy because they’re feeling good. I’ve seen that happen many times as well.
There are a variety of reasons that patients might not be adherent to medications. A lot of times patients are concerned about [adverse] effects. They haven’t experienced [adverse] effects, but they’re concerned about potential [adverse] events that they’ve heard about on TV, they’ve read on a package insert, they’ve heard from their pharmacist or other people. So they start to perhaps deviate away from the intended use of that drug, and they start to vary in terms of frequency of administration and so forth.
Last but certainly not least, one of the biggest challenges we face is that the drugs we have are often very costly. Although there are a lot of different avenues available to help reduce the out-of-pocket costs, there is significant cost associated with these medications. There are issues in terms of cost, coverage, and accessibility. There are always issues with insurance formularies, prior authorizations. There are always these continuous challenges despite the fact that we do have effective therapies for a lot of our patients.
Transcript Edited for Clarity
