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Key trends to watch:
Bridging the rare disease treatment gap

New treatments are providing hope for patients with rare and hard-to-treat conditions.

Breakthroughs in rare and complex care

Rare and difficult-to-treat chronic diseases affect millions of patients, but only a fraction receives effective treatment. Patients often face years of delayed or missed diagnoses and treatments that address symptoms but not the underlying causes. A better approach is needed.

Pharma is evolving to meet the challenge, leveraging orphan drug incentives, accelerated pathways and collaborative reviews to develop therapies across oncology, neurology, hematology and more, ensuring earlier access, meaningful outcomes and support throughout the patient journey.

Widespread need with few solutions

With thousands of rare diseases but hundreds of treatments, patients often face years without answers. Innovative new therapeutics spanning multiple therapeutic areas are closing this care gap.

>7,000
rare diseases have been identified.

(National Organization for Rare Disorders, 2025)
~500
treatments for rare diseases are approved by the U.S. FDA or EMA.

(National Organization for Rare Disorders, 2025)
29.6%
of the drugs granted rare or orphan drug designations globally in 2025 were for oncology.

(Clarivate data)

Promising treatments for rare and chronic conditions

The evolution of precision oncology and immunology reflects increasing sophistication in targeting disease mechanisms, and the Drugs to Watch in this category signal a paradigm shift in approaching difficult-to-treat diseases.

Depemokimab
Asthma
Icotrokinra
Plaque psoriasis
Sibeprenlimab
Immunoglobulin A nephropathy (IgAN)
Tolebrutinib
Multiple sclerosis