Gold Rush: Rare Diseases

Explore the future of rare disease and the market potential with in-first-class!

This in-depth ARTO whitepaper unpacks a strong sense of optimism about the Rare Disease market in 2025, with analysts projecting the rare disease therapeutics market to exceed $400 billion by 2030, growing at ~12% CAGR​ – a testament to both the high unmet medical need and attractive incentives (e.g. Orphan Drug exclusivity, tax credits) driving investment. We cover a range of topics including:

Talent Hurdles
This section explores the critical challenges Life Sciences companies face in attracting and retaining specialised talent in the rare disease space, particularly across regulatory, medical affairs, and clinical development functions.

Drug Launches
We examine recent and upcoming drug launches in the rare disease market, assessing commercial strategies, regulatory milestones, and the organisational readiness required for successful market entry.

Biggest Players
An overview of the leading pharmaceutical and biotech companies investing heavily in rare diseases, including analysis of their pipelines, market share, and strategic acquisitions driving their dominance.

First-in-Class Drugs
This section highlights groundbreaking first-in-class therapies, showcasing innovation in mechanisms of action and the impact these drugs have on previously untreated or poorly managed rare conditions.

Clinical Trial Recruitment of Patients
We address the complexities of enrolling patients in rare disease trials, focusing on recruitment bottlenecks, patient engagement strategies, and the ethical and logistical challenges of working with ultra-small populations.

Download the whitepaper now to stay ahead of the curve.