Creating an optimum patient experience with rare disease populations

By Nareda Mills and Eric Redline

Navigating any disease can be tough. For those suffering with a rare disease, it is even tougher. According to the Genetic and Rare Diseases (GARD) Information Center, one in 10 people in the U.S. are believed to have a rare disease. Despite being a somewhat significant percentage of the overall U.S. population, the fact that these patients are spread over approximately 7,000 known rare diseases makes the actual population for each disease very small. As a result, there is very little, if any, public awareness of any of these diseases. Patients may not even know they suffer with a rare disease, or be able to explain it. Many are accused of faking their symptoms, and even if diagnosed, may feel adrift in a sea of process and information. These patients don’t know where to turn, and are often getting bite-size bits of information and little to no resources to steady their course. 

Patient support for these populations can greatly impact their overall well-being and willingness to adhere to prescribed therapies, but is also critical to alleviating the inherent anxiety accompanying the diagnosis and treatment journey in rare disease. To help ensure an optimum experience, the best patient support programs will address these concerns by blending the empathy of human connection with the scale of technology.

The human connection

No matter what the disease, a patient support program needs to address the “whole” patient and his or her care partners, those spouses and family members responsible for the patient. In so doing, one needs to take into account not just the clinical and functional needs of patients and their care partners, but their behavioral and psychosocial needs as well. These underlying drivers can be far more onerous and difficult to overcome in the case of rare disease than those experienced by patients with other specialty and chronic diseases. Because many rare diseases are based on genetic heredity, these patients and their family members tend to have a high degree of self-blame and/or stronger feelings of victimization. Many have intense feelings of self-doubt in being able to get through the disease journey. Rare disease patient support programs should be designed to help patients and their care partners move past these feelings and deal with the actual disease manifestations and treatment. 

The ideal support program should start with the welcome call by the clinical educator, and it is this call which sets the tone for the ongoing support between the clinical educator and the patient and care partner(s). The importance of this call cannot be stressed enough. It is here that the clinical educator will begin the process of providing supplemental education to patients and their care partners on the facts about their condition, its possible progression, and how they can move forward to make the most of their situation. The clinical educator will also explain the process of obtaining a specialty treatment and what delays might be encountered. Having patients understand these basics from the onset of support is critical to setting appropriate expectations for patients in their therapy. These patients and their care partners need to understand what they are facing and how their therapy will impact their disease, along with what side effects they might experience and what they might do to mitigate those side effects. With this rare disease population, these calls generally last more than an hour due to the complexity of the disease and treatment journey. In contrast, welcome calls in support programs for other patient populations last only 20-30 minutes. Without question, clinical educators tasked with supporting rare disease patients need to be highly skilled and versed in the nuances of the disease and how it impacts the patient, and also be trained to listen and employ empathetic communication strategies and motivational interviewing with this special population. 

For most rare diseases there is considerable testing done up front to identify the genetic component, and then once diagnosis is confirmed, the treatment medication often needs to be titrated and tracked, further complicating education and support. Ideally, one clinical educator or navigator will be assigned to the patient to best build a relationship where the individual patient’s story is forefront, providing much-needed and customized consistency throughout the support program. Since the process of being diagnosed is difficult enough for rare disease patients and their care partners, they don’t need the additional burden of having to explain themselves time and again to those supporting them. The assigned clinical educator will help the patient set goals and get initiated onto treatment, often heavily integrating with physician offices, hub providers and specialty pharmacy stakeholders to get the treatment started. Many programs will employ real-time data transfer or APIs between hub providers, specialty pharmacies and clinical educators, so at a moment’s notice the clinical educator knows where the patient is in their journey and can respond accordingly. 

The scale of technology

With the increasing acceptance of digital channels providing healthcare support and information, omnichannel support has become an even more important component in most patient support programs. It takes on an even greater importance with rare disease. The small patient population of rare diseases, made even more difficult due to their geographic spread throughout the United States, may preclude full-scale support teams. As a result, technology becomes an important tool to augment the human touch. 

A case in point is a program we have in place serving infants suffering with a severe respiratory condition. This program uses text reminders to parents/care partners of these infants to ensure that they are scheduling follow-up dosing visits and completing the appropriate course of therapy. In addition, the program sends motivational messages via text and images and videos in between these reminder notices to help parents through this difficult experience. The program is also looking to create an alert system for parents, using publicly available data sources, pinpointing when cases are arising in specific locations so that parents can be vigilant around symptom identification.

Another example of leveraging technology to augment support of rare disease patients are outreach programs we have developed with Sobi for patients suffering with a chronic rare liver disease coupled with a specific blood disorder. “At Sobi, we know the ability to listen to the customer and quickly adapt to meet their needs is critical to provide support for those who need it most, especially in rare disease,” says Chris Weber, associate director, customer experience, Sobi North America. “To deliver on that, we’ve developed patient, caregiver, and HCP journeys that provide relevant and customized content to these important external stakeholders across various digital platforms and live engagements.”

These personalized patient outreach programs have been designed to serve two specific patient classifications. We determine these patient types from the answers provided to some basic on-boarding questions presented in a form on the therapy website. All patients completing the form receive a thank you email, acknowledging them for being a “goal-getter” and informing them that they will receive a welcome kit in the mail. 

The subsequent outreach programs are then designed to specifically address the characteristics of the patient type. Examples include guiding them to reach a treatment goal, or offering a multitude of ways to continue to track progress and take record of goals and milestones achieved. Educational information about their medication and how it can help with their condition is also provided where needed. To optimize engagement in the email program, we are using data and AI to do subject line testing in order to deliver the most eye-catching wording to drive engagement with the email.

To round out the program for this therapy, our customer engagement team has developed a program to send out emails to the HCPs within this community in order to create awareness of the treatment options with this disorder. For those already prescribing the therapy, we provide more detailed information highlighting the medication’s benefits, and also educate them on the existing support programs to help their patient remain adherent to their therapy.

Ideally, rare disease patient support programs are supported by a technology platform such as our proprietary engagement system – Trak360. This platform facilitates what rare disease patients need for support while providing a 360° view of the treatment journey in real time. The patient-centric platform provides information to the patient via their channel of preference – mobile, email, texts, live chats, video conferences, websites, printed materials, and in-home education. At the same time, it tracks activity such as medication administration, engagement points with the attending physician and even data from wearable technology. Having this real-time data and resulting analytics available to the clinical educator allows for faster responses to the patient’s needs, ultimately improving adherence and persistence. 

Regardless of the technology platform underlying the patient support program, giving rare disease patients a self-service portal, where they can access information when and how they are ready to absorb it throughout their treatment journey, gives the individual a sense of empowerment rare disease patients often lack. Developing community portals where patients and care partners within a rare disease category can securely and confidentially connect with one another would be the next logical step. In such a portal, the patient and care partner would be able to communicate with others dealing with the same condition, share experiences and recognize they are not alone. Advocacy groups and associations can also participate and provide educational and support resources.

Putting digital and technology front and center for such a community, one could envision a gated self-service patient portal on a particular therapy allowing for connectivity with other like-minded patients and care partners and moderated for compliance. This portal would be augmented by text and email touchpoints to check in on patient progress and send prescription refill reminders, but also provide for human outreach in situations where the patient may not be responding or updating information around refill status, check-in, etc. The front end would be driven by a humanized bot, either voice or text driven, but would have a flow that can be augmented by live interactions depending on the criteria. 

Such a scenario and ecosystem will combine the best elements of technology, people and process. It builds flows for specific touchpoints within the treatment experience blending the empathy of human connection with the scale of technology, thereby maximizing the constrained budgets rare disease brands often find themselves facing.

There is a massive amount of work to be done in rare disease. Improving awareness of the conditions and the support programs and resources available continues to evolve slowly as the industry grapples with the complexity of the diseases and limited resources. Payers are beginning to take notice and are looking to establish educational support programs, which can only help. Wherever the awareness and support originate, it cannot come too soon for those suffering with a rare disease.

Nareda Mills is global president, patient solutions, Ashfield Engage; Eric Redline is senior VP, Engage Digital (North America).