Four Takeaways from World Orphan Drug Congress

The 2024 World Orphan Drug Congress brought together leading thinkers and advocates from across the healthcare ecosystem to discuss innovative ways we can bust silos, challenge conventional wisdom, and bring breakthrough therapies to patients with rare or ultra-rare conditions. Several members of Real Chemistry’s Analytics and Insights group participated in a panel on the end of Day One: From Patient-Finding to Payer Pull-Through: Leveraging Advanced Analytics to Fuel the Rare Disease Revolution. The panel reinforced several key conference themes around leveraging real-world data and AI in new, promising ways and anchored discussion around the fundamental question illustrated in the graphic below – how well are manufacturers seeing the whole picture as they move a rare disease therapy through the commercialization process? Real Chemistry panelists included:

• Ivan Kouchlev, Managing Principal, Real-World Data Solutions

• Lindsay Murphy, Senior Group Director, Integrated Intelligence

• Christine Juday, Head, Market Access

If you missed WODC, here were the four key takeaways from that panel discussion:

1. Innovative uses of AI and advanced analytics can help manufacturers find the “hard to find” patients and HCPs. Ivan Kouchlev noted how ML-based models can help manufacturers identify potentially misdiagnosed or undiagnosed patients, allowing for more targeted, education-centered HCP messaging and engagement strategies that may accelerate diagnosis and treatment initiation. With new launches, in particular, there’s added value in regularly refining those ML models as well, using the expanding pool of patients on therapy to improve the model’s precision in finding even more potential patients and prescribers. This, in turn, helps maximize the efficiency of sales and marketing spend.

2. Social intelligence can greatly enhance manufacturers’ understanding of individual patients, caregivers, and rare disease communities. Panelist Lindsay Murphy highlighted the value of social intelligence to help rare disease drug manufacturers see patients as whole people, not just individuals with a particular disease. She emphasized the vitality of online communities for rare disease patients, noting that rare disease patients are actually 42% more likely to be active online than those without a rare disease. Social intelligence can help manufacturers better understand individuals’ questions, frustrations, advice, coping mechanisms, and even particular word preferences – all of which can greatly enhance manufacturers’ messaging and engagement strategies for both consumers and HCPs.

3. An ecosystem-wide lens can help manufacturers re-imagine what’s possible as they look to optimize access and coverage. Chris Juday talked about the power of incorporating multiple stakeholder perspectives into an early, integrated evidence-generation plan. This can help guide study designs, data collection efforts, and advocacy goals — increasing opportunities to advocate for new ICD-10 codes, quality metrics, or innovative payment models when appropriate.

4. Rare disease drug launches require performance measurement models that enable continuous learning and iteration. Every rare disease launch is different. There’s no playbook, so teams need access to performance data that can quickly illuminate what’s working and what’s not – allowing for maximum learning and agility.

If you’re interested in learning more about how Real Chemistry’s Analytics & Insights teams can help you maximize the impact of your rare disease therapy, please contact Jared Minton at jminton@realchemistry.com. We’d love to listen, learn, and see how we can help.

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