Biopharma experts article

Rhonda Hollingsworth 
Vice President, Client Solutions, Optime Care 

When choosing a specialty pharmacy (SP) for your rare or orphan drug, what is one of the first things you look for? A specialty pharmacy who is an expert in the disease state, of course. It’s a familiar approach that may work well for common conditions with large patient populations, such as hypertension or high cholesterol. 

But here’s the problem if your drug treats a rare or orphan disease: there are few experts anywhere for a given condition—perhaps only one or two physicians focused on academic research. In the SP world, no one is truly an expert precisely because the disease is rare, orphan and largely unknown. Yet, most biopharmaceutical manufacturers are looking to SPs for this expertise, and the consequences can adversely impact both their business model and patient care. 

The problem with seeking a clinical expert 

Conducting their research deep inside academic institutions, clinical experts are typically far removed from actual patient care. While they may be world renowned in the condition itself, they’re not expert at ensuring the patient population has high-touch support. That level of personal care is vital to ensure access, manage preauthorizations and help individuals continue to take their medication as prescribed.  

Clinical experts simply don’t have the first-hand perspective to understand that every patient has a different experience and requires care precisely tailored to it. As a result, a manufacturer seeking clinical expertise can inadvertently disrupt the quality and continuity of care.  

Here’s how it happens: to help ensure access, a manufacturer may choose to work with several SPs that claim to have experts in a particular disease state (remember, they don’t). 

As the life circumstances of a patient change—switching jobs, for example—that patient may change SPs several times. So they don’t have a consistent experience, because the same team isn’t working closely with the patient year after year. 

As the patient changes SPs, the drug itself may change as well (if there’s more than one available for the condition). There’s the potential for adverse events that could hinder compliance—and waste all of the progress made to date. 

The better way: find the patient expert 

If the condition is truly rare, virtually none are expert in the disease state itself. However, they are expert in what matters more: caring for people who have a rare or orphan condition. Patient experts determine how those in their care can more effectively use the prescribed therapy to treat the condition and improve quality of life. 

A patient expert starts by identifying the “pain points” in each person’s life. Was it in the diagnosis? Two months after starting therapy? Three years later when they felt fine and stopped taking their medication? By getting involved early and asking the right questions, a patient expert can better help an individual overcome these pain points and take better advantage of the therapy.  

Over time, the patient expert can acquire a significant amount of data and understanding of how the therapy serves the broader patient community, which in turn can help the manufacturer maintain and grow market share. Indeed, a patient-focused SP gains first-hand knowledge of how a disease state impacts individual patients and their families in the real world, right in their homes.  

Contrast this with SPs accustomed to serving large patient populations and attempting to apply that approach to rare and orphan drugs. It doesn’t work as planned, because the program isn’t geared specifically to the needs of individual patients with rare and orphan conditions.  

These SPs don’t understand the unique needs of these patients because they haven’t had meaningful conversations with them, their families and caregivers. In addition, these SPs aren’t involved in the clinical trials, so they don’t have intimate experience with the issues patients have. 

The value of seeking the right expertise 

If you choose an SP that’s expert at the patient—not the disease state—you will be in a better position to gain the advantages you seek: 

  • Patients will get on the therapy faster and stay on it longer 
  • Patient and physician satisfaction will grow 
  • You will more likely achieve sales and market share goals (if there is more than one product for the condition)  

Physician surveys conducted by manufacturers reinforce this value. When asked which was more important to them when choosing an SP, physicians overwhelmingly chose patient-focused services over disease state expertise. 

How do you find a patient expert? First, it’s important to understand that clinical knowledge about a rare or orphan disease can be learned. The far greater challenge is having the patient-first focus to take care of patients who have these conditions. 

Rather than ask an SP if it’s expert in a rare or orphan condition, learn whether the SP is expert at treating patients who have it. Put another way, is the SP patient-oriented or process-oriented? Instead of touting its robocall capabilities and hub efficiencies, is the SP focused on understanding your individual patients and their unique experiences with the rare or orphan condition? From there, you can tailor an approach to better serve each patient while achieving your overall business goals.  

To learn more, download our white paper, “How to create a patient-first strategy.” Or contact me directly at or call 888-287-2017.