The world of medical affairs thrives on innovation and progress in the healthcare landscape. But what about diseases that affect a fraction of the population? Rare diseases, while less common, present a unique set of challenges and remarkable opportunities for medical affairs professionals.
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 denote expert status reliably. Finding experts in rare diseases 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. In this blog, we’ll explore five steps that will help pave the path to success for MSLs working in rare disease medical affairs.
What are the challenges of rare diseases?
Rare diseases present a variety of challenges for all types of healthcare professionals across the care continuum.
Unlike common conditions, rare diseases affect a small percentage of the population. This translates to fewer patients for clinical trials and a smaller overall market. Additionally, the scarcity of patients makes it difficult to find specialized healthcare providers (HCPs) and researchers–the very people medical affairs rely on for collaboration. These obstacles can sometimes limit funding for research and development efforts, further impacting the resources available to medical affairs teams.
Despite these headwinds, there is still hope for patients with these conditions and there are strategies medical affairs professionals can use to find and engage the experts they need to help bring treatments to market faster. Let’s dive into five of the steps MSLs can take to find new opportunities for growth in the rare disease field.
1. Collect as much quantitative data as possible
Unlike common diseases, rare diseases affect a small portion of the population, fewer than 200,000 people to be exact.
This means every piece of information you gather is crucial for understanding your specific therapeutic area. Even if patient populations for your rare disease are small and data is scarce, any insights could contribute to a significant advancement. To find success, MSLs need to be skilled at working with limited datasets and extracting the most value from them. Types of valuable quantitative data include:
- Patient demographics. This includes data on things like age, gender, geographic location, and ethnicity of patients diagnosed with the rare disease. This can help identify potential risk factors and inform targeted outreach efforts.
- Disease prevalence. While the overall number of patients with a rare disease might be small, understanding the prevalence (how many people are affected) can help prioritize research efforts and resource allocation.
- Clinical trials: Data from clinical trials, even small-scale ones, can offer crucial information on the effectiveness and safety of potential treatments for rare diseases.
- Registry data. Patient registries can be a goldmine of quantitative data for rare diseases. These registries collect information on many patients over time and can reveal important information about the signs, symptoms, and variations associated with a rare disease and how patients respond to types of treatment.
Our Monocl ExpertInsight platform offers medical affairs teams an unrivaled amount of relevant, useful data across all therapeutic areas and geographic regions. This empowers MSLs in the rare disease space to efficiently research topics across clinical trials, conferences, publications, organization involvement, and other key activities like social media to deepen their understanding of the scientific landscape and more precisely locate and engage with key experts.
For an even deeper dive into leveraging quantitative data, read our blog on how to use data to accelerate rare disease drug development.
2. Take the time to do qualitative research
Quantitative data can only reveal certain parts of the rare disease landscape. Qualitative data, on the other hand, can illustrate the hidden aspects of the rare disease that might have otherwise been overlooked or neglected by treatment data or a clinical trial.
In-depth interviews, focus groups, and other types of qualitative research can help MSLs deepen their understanding of the financial, social, and emotional burdens associated with a rare disease by hearing directly from patients and their families. MSLs can take these insights back to their organization (more on that later) to ensure that patient perspectives are incorporated into treatment development and care strategies and that their needs are met.
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.
Of course, it’s important to do due diligence and start your mapping with experts at large hospitals and academic medical centers. Within the rare disease space, patients are frequently treated at larger facilities that staff specialists and have the equipment and technology that can best address their needs. Suppose the rare disease requires a full team of specialists. In that case, MSLs need to recognize which types of healthcare providers interact with the patient, and at what point in the journey those interactions happen. The specific information about the rare disease and the treatment landscape each professional will need.
However, the most influential figures might not necessarily be those with the most publications. Expand your search beyond major hospitals and academic centers and look for patient advocacy groups, smaller clinics, or even individual physicians who are deeply involved in the rare disease community and can offer invaluable insights, connections, and patient perspectives.
Don't be tempted to build a massive network of loosely connected influencers. Focus on cultivating strong, genuine relationships with a select group of individuals who possess deep expertise and hold significant sway within the rare disease community. Prioritize building trust and open communication.
4. Personalize every engagement
MSLs rely on strong relationships with healthcare professionals to ensure they have access to the latest information on rare diseases. However, each rare disease has its own set of challenges, symptoms, and treatment options, making a one-size-fits-all approach to communication with healthcare providers impossible.
Because of these circumstances, it’s crucial for MSLs to personalize their communications and interactions with the expert they’re engaging with. Ask your MSLs to do their homework and research the expert’s specific interests they might have in rare diseases and actively listen to their questions, concerns, and experiences. This allows for a more genuine, focused conversation that can foster a sense of partnership and collaboration and ultimately lead to better patient care for those living with rare diseases.
5. Bring expert insights back to the organization
In recent years, life sciences companies have increasingly recognized the strategic power of medical affairs teams. MSLs play a critical role in their organization, acting as the bridge between the company and the scientific community.
However, the true impact of MSLs lies not just in their ability to engage experts and gather knowledge, but also in their ability to share that expertise across the organization. In today’s fiercely competitive healthcare landscape, collaboration and communication across teams have become increasingly important. MSLs can serve as that connective tissue, sharing expert insights with other internal teams, including marketing, sales, R&D, and more, to make strategies more robust and effective. This collaborative approach is especially crucial when it comes to rare diseases, where a comprehensive understanding of the challenges and opportunities is vital for success.
Learn more
Like other areas of the healthcare industry, the journey to success in the rare disease space isn’t easy, but the market is ripe with opportunities for organizations and medical affairs teams to drive positive change.
By embracing the steps discussed above, MSLs can help their organization push the boundaries of research and drug development, and potentially improve the lives of patients living with rare conditions.
Interested in learning more about the rare disease landscape? Check out our blog on identifying opportunities in the orphan drug market or start a free trial with Definitive Healthcare today to access the tools, analytics, and intelligence you need to find the experts for your therapeutic area.
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