Saiqa Khan
Head of Growth, Marketing, Avalere Health
Dr Saiqa Khan, Head of Growth, Global Marketing, at Avalere Health, explains why success in rare disease can no longer be measured by strength of data or time to diagnosis. It must be measured in the number of lives transformed.
Rare diseases represent one of healthcare’s most complex frontiers. The umbrella term rare disease covers extraordinary diversity: some shorten life; others primarily affect quality of life; many bring complex social and emotional challenges. Each is unique, demanding its own tailored pathway.
As a strategic partner for the healthcare industry with one mission, to reach every patient possible, Avalere Health is redefining what a rare disease launch strategy means today.
Decades of progress and a future of possibilities
The rare space has seen a remarkable transformation, from limited understanding to unprecedented innovation, with competitive landscapes emerging. Yet, 95% of rare diseases still have no treatment.1
“The challenge is no longer just scientific innovation,” says Dr Khan. “Equally, it’s about ensuring new therapies, once launched, reach every patient who needs them.” Far from discouraging, this is a powerful call for continued collaboration to empower healthcare professionals, patients and the industry to be the pioneers of positive change.”
If no two rare diseases are
the same, then no launch
can follow a playbook.
Every rare disease launch must be unique
If no two rare diseases are the same, then no launch can follow a playbook. Instead, launches must be distinctly grounded in evidence, insight and human connection. “Launching a rare disease therapy is not a single moment,” explains Dr Khan. “It’s a holistic continuum, where success depends on engaging every person, early, and with uncompromising authenticity.”
Avalere Health takes an integrated approach, uniting its Advisory, Medical and Marketing. capabilities to create a comprehensive picture of the landscape, including the patient experience. “Here, more than ever, patients are not just recipients of care,” says Dr Khan. “They are experts, advocates and partners in research. Their perspectives add immeasurable value, and we must engage very early on.”
Reaching every patient possible
“Every new therapy is a milestone worth celebrating — a step closer to transforming lives across more than 7,000 known conditions,” says Dr Khan. Leveraging its unique ‘SenseMaking’ approach, Avalere Health connects silos across society, systems and science, uncovering missed opportunities to create bold, effective models of engagement grounded in the lived experiences of patients.
Dr Khan explains: “The impact is beyond commercial success. It’s putting therapies in the hands of people who might otherwise remain unseen. By launching with integrity and purpose, we can truly transform people’s lives.”
[1] The Lancet Global Health, 2024. The landscape for rare diseases in 2024. The Lancet Global Health, Volume 12, Issue 3, e341.
