Future-proofing patient identification and engagement
225-30 million Americans are living with a rare disease and a subset of them are living with an ultra-rare disease; defined by those conditions affecting fewer than 20 individuals per million population. Thankfully, the last decade has seen a dramatic shift in the awareness, understanding, and prioritization of rare and ultra-rare diseases. What once was a mysterious and economically challenging category, causing frustration among healthcare providers, patients, and caregivers, has now become a core and even singular focus among biotechnology companies, marketing agencies, patient advocacy groups, and other organizations.
Scientific and technological advancements have enabled improved disease understanding and treatment development, having empowered a greater commitment to identifying and engaging with affected patients, caregivers, and their healthcare providers. Precision medicines have offered the prospect of alleviation to millions of patients, with more than 50 novel therapies receiving FDA approval in 2021 alone. By 2023, 51% (28 of 55) of novel drugs obtained orphan-drug designation and were approved to prevent, diagnose or treat a rare disease or condition. Now is the time to harness that potential and optimize the impact these treatments can have on the patients who are living with these conditions.
As the drug development timeline for rare and ultra-rare diseases continues to expand, it is essential for companies to embrace new technologies and methodologies to reach patients who have an unprecedented opportunity for relief, and to engage with them in a way that provides comfort, confidence, and empowerment. While economics can pose a challenge due to incredibly small disease populations, there are several ways of identifying and engaging with patients, caregivers, and healthcare providers that are financially feasible to deliver impact. The application of sophisticated approaches to data science, bolstered by strategic patient advocacy and center of excellence engagement, represent three of the opportunities we have to take rare and ultra-rare disease patient identification and engagement to the next level.
Precision Methods for Patient Identification
Precision medicine in a broad sense is tailoring treatment to the specific genomic, environmental, and lifestyle characteristics of an individual patient. Patient identification is paramount to the development, availability, utilization, and outcomes potential of rare and ultra-rare disease precision therapeutics.
Historically, these patients have been in the dark, challenged and alienated as an “n” of 1, or oftentimes facing diagnostic delays or misdiagnoses. Scarcity of provider expertise, compounding disease complexity, and a lack of discrete diagnostic codes for ultra-rare diseases and some rare diseases can further complicate the identification of appropriate therapies for patients.
Fortunately, strides made in the application of data science have empowered the discovery of so many more needles in the proverbial haystack, allowing exponentially more patients to participate in the development of and benefit from treatment advances. These strategies include:
– Claims-based research that can enable greater insights to patient pools and locations.
– Proxy models (known and hypothesized subpopulations) that support the identification of patients with rare and ultra-rare conditions.
– Triangulation of data and the use of multiple datasets including claims, prescriptions, and more can identify potential areas of patient activity.
– The use of data analytics and predictive modeling to identify a broad range of appropriate healthcare providers who have the highest likelihood of treating patients.
– Geospatial analytics and visualizations that can ensure maximum access and targeting based on various metrics. Identifying geographic clusters and longitudinal trends can aid in pinpointing patient activity most aligned with the disease profile.
There are also several specific outreach and communication methods that can support the identification of rare or ultra-rare patients:
– Cross-functional engagement with healthcare providers, patient advocacy groups, and caregivers is a critical aspect of ensuring appropriate patients are not lost. The sharing of common hallmarks of disease as potential flags for suspected rare and ultra-rare diseases may drive testing where it has been previously overlooked.
– The sponsorship and amplification of genetic testing programs may quicken the path to diagnosis with patients suspected of disease. Although misuse or overuse of sponsored programs can lead to financial challenges for sponsors, there are strategies to support mitigation of this risk, through targeted amplification with highly specific target audiences.
By investing in these methods, we can endeavor toward earlier diagnoses, interventions, and drive improved outcomes and quality of life for impacted individuals.
Advocacy Integration
There is significant opportunity in the formation of partnerships between industry, patient advocacy groups, and professional organizations. Such collaborations have the potential to effectively reach and engage patients with rare and ultra-rare diseases, their caregivers, and the healthcare providers who treat them. Unlike disease conditions with larger patient populations that typically have highly organized advocacy landscapes, the advocacy universe for rare and ultra-rare diseases tends to be smaller and less formalized, with more grassroots efforts. Advocacy groups have often been founded by a patient or caregiver and are highly personal in nature.
Therefore, there is a tremendous opportunity for industries to support rare and ultra-rare disease advocacy groups, but this is an endeavor that must be approached sensitively and thoughtfully. Due to the small and personal nature of several of these groups, it is critical that any collaboration or engagement be designed in a patient-conscious manner, centered around patient benefit rather than for a company. Co-creation efforts are a great way of engaging with these advocacy groups, empowering those affected by the condition to be in the driver’s seat. Early engagement, with a focus on patient and caregiver voices, can forge stronger ties with the whole organization and optimize the authenticity of communication created in partnership, amplifying impact with individuals and communities aligned with the cause.
In addition, professional societies present another avenue to connect with healthcare providers treating rare and ultra-rare diseases. Healthcare provider engagement can prove challenging for organizations, given the low likelihood that a healthcare provider might encounter a patient with such conditions. The motivation to learn about a condition with which providers are not likely to interact may also be low.
Therefore, professional societies represent a focused, cost-effective strategy to reach the appropriate specialists who can be valuable resources to foster knowledge exchange and disseminate information. Societies are motivated to amplify disease awareness, especially in the case of orphan designation, treatment availability, and can also serve as resources as it relates to driving new clinical guidelines or updates to existing guidelines.
Center of Excellence Treatment Models
Center of excellence provider models for the treatment of rare and ultra-rare diseases can be highly efficient and effective if implemented appropriately. Predictive analytics can help identify key metrics that empower the establishment of these centers in a patient-centric manner, including the consideration of geographic location and the appropriateness of the multi-disciplinary approach required by many rare and ultra-rare conditions.
Through the integration of center of excellence treatment models, marketers can design highly targeted, cost-efficient materials, initiatives, and communications that will empower patients and foster a sense of community. This will help them feel connected and understood, rather than isolated by their condition. Moreover, these hubs enable personalized care and encourage extensive cross-disciplinary specialization, which ensures that patients and caregivers have confidence with the insights from their care teams. This reduces patients’ perceived or actual responsibility to educate their care teams about their condition. These hubs also further empower professional knowledge sharing, which can improve diagnostic confidence, quality of care, and outcomes as well as ongoing collaboration and research.
For marketers, the challenge to appropriately identify, engage, and connect with patients with rare and ultra-rare diseases is ongoing. With each breakthrough treatment that is developed, so too does the need to connect with communities impacted by the condition increase. Luckily, technology advancements and innovation are also continuing to evolve and enable opportunities to address these challenges even more effectively in a patient-conscious manner, presenting significant potential to continue to improve the impact we can have in this space.
