The life sciences industry is a modern marvel. It is producing the most dramatic scientific breakthroughs in history and life-altering treatments and cures for diseases are emerging rapidly. Artificial intelligence is being used to diagnose cancer. Leading-edge gene therapies are being used to cure children with previously untreatable, lifelong disorders. In fact, a record-breaking 22 orphan drugs were approved in 2019 for rare diseases.
Regularly, the FDA is approving groundbreaking treatments for rare diseases that didn’t have any options before. It’s exciting but it also introduces new hurdles for the industry. Medical Science Liaisons (MSLs) for instance, have historically identified key opinion leaders based on their volumes of clinical trials or peer-reviewed publications. However, this historical information doesn’t exist for many rare diseases, where research has been limited, or at least not in the quantity needed to reliably denote expert status. Finding experts in rare disease often requires MSLs to do more detective-work, searching more sources for those who align with their scientific programs.
Like all things, as the industry evolves, so must the professionals working on the front lines – especially medical affairs teams. Here are five key steps that will help pave the path to success for MSLs working in rare diseases.
Step 1: Collect as much quantitative data as possible.
Rare diseases are defined as those that impact fewer than 200,000 people and more frequently, the numbers are much smaller. Today, providers of advanced data analytics are aiding the life sciences industry by aggregating vast volumes of publicly available data sources, real-world data, and other proprietary collections of information and running sophisticated algorithms that allow MSLs to identify small, specific pools of potential experts.
Monocl’s data system, for example, aligns its search functionality with the relevant lexicon of terms for each rare disease, allowing MSLs to efficiently research topics across clinical trials, conferences, publications, organization involvement, and other key activities, like social media. Monocl also offers a constantly evolving an updated data set in real time, that will capture and index relevant information for these diseases as fast as they are evolving.
Step 2: Take the time to do qualitative research.
Secondary data can only reveal certain types of experts. Many clinical experts are important contributors but are not prominent trial investigators and or prolific publishers. Qualitative research, essentially talking to key subject-matter experts, can reveal the informal networks that connect various community experts and also help map the average patient’s journey. Map the patient journey. How are patients connecting with experts – are they referred by a general practitioner? Are patients finding them online? Are they being guided by their insurance companies? Do patients remain in their care throughout their journeys or are they referred elsewhere?
Step 3: Map the influencer networks.
Informed by both quantitative and qualitative data, MSLs should next begin mapping experts to reveal the key institutional hubs connecting research and treatment experts with patients. With rare diseases, patients frequently are treated at larger institutions, surrounded by a full team of experts including diagnostic experts (i.e., pathologist, radiologists, geneticists); treatment experts (i.e., specialty physicians, surgeons, research scientists,); and complementary care experts (i.e., nurses, physician assistants, dieticians, physical therapists). Each expert might need specific information from the MSL based on where they are with their own disease understanding, awareness of the treatment landscape, and perspective on patient needs.
Step 4: Personalize every engagement.
MSLs are excellent at talking through the science of new therapies and how each fits into the treatment landscape but it is important to share this information in the course of a dialogue with experts. Where is the expert in his or her own professional journey? What are her likes and dislikes? What are her communications preferences? What is he curious about? Even finding out what they do for fun can improve your ability to engage in a meaningful and memorable conversation. Personalized relationships will build the trust required for experts to share deeper insights, observations, and questions.
Step 5: Be a sponge, and bring experts’ insights back to the organization.
Successful MSLs can absorb insights and communicate them back to those driving the medical strategy within their organizations. Once you’ve established a trusted relationship with an expert (step 4), you now have an open line of communication. Listen. Ask questions. Capture real world insights. Don’t waste a single conversation. These types of MSL insights can inform scientific programs, impact clinical guidelines, define patient sub-populations, and change labels. They are especially important in rare diseases, where products are more frequently securing expedited approval status by the FDA.
In 2019, 60% of CDER’s novel drug approvals (29 of 48) used one or more expedited programs, bringing new therapies to patients months, or even years, sooner than expected. This is great news for patients but also limits the time companies have in the controlled research environment to learn more about the full impact of their novel drug. The real-world data brought back from MSLs engaging with experts in the field, therefore, is incredibly important to the ongoing development of the product and the label.