Som Sarkar, Director, Innovative Medicines US, Novartis 

In 2026, rare disease launch success is defined by precision, not reach. With small, hard-to-find patient populations and complex access dynamics, pharmaceutical leaders must orchestrate disease education, patient identification, access strategy, and advocacy partnerships into a single, integrated launch motion. Excellence comes from removing friction across the care pathway - before, during, and after approval.

• Aligning medical, access, and marketing around the full rare disease care journey, from diagnosis to long-term support.

• Prioritizing disease awareness, AI-enabled patient finding, and HCP decision support to unlock demand before launch.

• Building pricing, evidence, and patient services into day-one launch planning instead of as post-approval fixes.

• Partnering early and transparently with patient advocacy groups to build credibility and sustain momentum.