Since the Orphan Drug Act was passed in 1983, the number of rare disease drug approvals in the US has significantly risen. Between 1983 and 2020, a total of 5,131 drugs and biologics received orphan drug designation. One-third of the industry’s R&D pipeline is now in rare diseases, and there are nearly 400 therapies approved exclusively for orphan drug indications.

While this expanded the pipeline communities it’s great news for patients who have waited years or even decades for new medicines, challenges remain. Historically, therapies focusing on niche markets face tremendous obstacles, from drug development and clinical trials to drug access.

The following are several key challenges for brand marketers readying to launch products in these small markets, along with solutions for driving better stakeholder experiences.

The challenge of educating physicians who don’t often see a rare disease patient

Physicians are trained to provide care for a broad scope of conditions within their patient population. Rare diseases, by their very nature, are uncommon and even specialists may not always  be able to spot the signs of an illness that they don’t encounter frequently. Rare diseases also can masquerade as common ailments, until more symptoms surface. The combination of uncommon diagnoses and lack of awareness leads to a number of “missed diagnoses” or misdiagnosis.

One way to increase healthcare professionals (HCPs) scope of practice to include rare diseases is to provide educational tools that support a “learn-by-doing” approach. For example, simulation is an educational tool that can help create memorable experiences for physicians. Simulations empower healthcare professionals to encounter real-life medical situations and put controls in place, so they can pause, ask questions, and replay the scenario.

During the pandemic, healthcare systems accelerated investment in simulation technology to provide remote training, integrating the tools into the clinical curriculum. Alongside its role in revolutionizing medical education, the tools have also been used in sales aids, promotional medical education, and even at conferences. However, as augmented and virtual reality move to the mainstream, simulation education is beginning to transition to learning through immersive experiences. Products like Oculus or Sony’s Morpheus bring gamers into the virtual world where they can see, feel, and be in a virtual location that they can control.

Additionally, researchers have started conducting studies into the role of simulation in diagnosing rare diseases. One recent study on Raynaud phenomenon and systemic sclerosis shows the importance of leveraging immersive learning with education to improve knowledge retention.

Offering this type of experience as a diagnostic tool, can help physicians to diagnose and manage treatment for a rare disease patient. Because these devices are driven by artificial intelligence (AI), they may also accelerate the diagnostic process by steering HCPs toward a specific differential diagnosis.

The challenge of building trust in patient communities

The relationship between a healthcare professional and a patient or a caregiver is enhanced when both connect with each other. Biopharmaceutical companies can play a vital role in helping to improve this relationship but doing so starts with empathy and transparency.

Patients living with a rare disease tend to be knowledgeable about their condition. They’ve done hours of research, visited multiple physicians prior to getting an accurate diagnosis, and have communicated with others like them online. As a result, they tend to be skeptical of companies they believe are only “in it” for the profit or “want to capitalize” on the misfortunes of others.

For a marketer to succeed in a rare disease category, they must dig deep to uncover the types of insights that truly show patients and caregivers that they understand them. Behavioral science is emerging in our field and will be an essential component of any future marketing strategy.

Rare disease communities can tell pretty quickly when a company doesn’t get them. The communities are often complex networks. But for us, the application of artificial intelligence (AI) and machine learning has accelerated the way in which we can identify not only patient or caregiver subsegment behavioral patterns, but also why they behave in a certain way. The combination of powerful analytical tools with behavioral science methodologies has enabled us to indentify the interdependencies between HCPs, patients and caregivers, allowing us to make more meaningful connections with the group and to gain the trust with their community.

Developing approaches to improve time to diagnosis

Identifying patients who live with a rare disease can be akin to seeking out the needle in a field of haystacks. Some tools can give us a window to finding patients. The use of ICD10 diagnostic codes, for example, is one way. Social listening to learn about how patients and caregivers are engaging in their own communities is another.

We also apply AI to uncover where the HCP, patient, and caregiver are along their journeys and identify patterns— whether they’re online or offline seeking information. With AI-driven tools, we are able to not only seek them out, but also provide them the right type of content delivered at a specific moment in time, such as when they’re searching for information about a diagnosis or treatment option. Armed with AI data, we build a persona of each individual to create messages that are uniquely personalized to each one. We recently did this for a client and it has yielded remarkable results.

But to accelerate the time to diagnosis, we also need to create a sense of urgency among physicians. When HCPs encounter a patient with a rare disease, that individual is already experiencing a sense of anxiety and fear. Undiagnosed patients know something is wrong; they just don’t know what it is yet. HCPs feel their frustrations and begin the process of ordering tests and examinations to get to a differential diagnosis. However, without enough knowledge about the condition, they’re unable to pinpoint a diagnosis.

HCPs may feel a sense of hopelessness when they are unable to help their patients. Given information about the diagnostic process and emerging or available treatments for a rare disease, HCPs transition from hopelessness to a sense of obligation —  and a responsibility to refer, diagnose, or treat patients quickly. Delivering that information in a way that doesn’t impune the HCP is critical though. Making HCPs feel like they can finally solve the problem for their patients in a way that is relevant to them as an individual is key. It all comes down to personalizing the message and connecting with customers in a way that is as unique as the disease they are experiencing.

Solving these challenges that brand marketers encounter with rare disease brands are not insurmountable, but do require innovative applications, such as the inclusion of AI-driven tools. Irrespective of the tools they apply, marketers must never lose sight of how important it is to invest in rare disease communities, support patients and their families, and unite them with HCPs to build trust and create brand loyalty.

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