The relationship between regulators and life science companies is a dynamic and complex interaction. Many think of the role of life science companies as that of pushing the boundaries of innovation, while regulatory agencies are tasked with prioritizing public safety, proceeding with careful deliberation to ensure the highest standards are upheld.
Of late, however, there has been an interesting shift in this dynamic. After an initial adjustment period to the emergence of big data applications, regulators and health technology assessment bodies in key global markets are increasingly recognizing the value of incorporating real-world data, real-world evidence, and patient-reported outcomes to support submissions. This shift is being driven by a renewed emphasis on patient-centered care as agencies seek to capture the patient voice, improve patient outcomes and address healthcare inequalities.
These tools have a number of promising applications, including ensuring more representative clinical trials, using synthetic twin comparator arms and exploring alternative study designs for treatments with small patient populations. Companies are increasingly being challenged to make use of them in order to stay ahead of the competition and ensure equitable access to cutting-edge therapies, but doing so requires early, intentional planning and multifaceted approaches, including robust R&D strategies, strong regulatory and market access planning, effective stakeholder engagement and continuous innovation.
Patient centricity is driving a wave of breakthroughs
- The year 2024 was full of firsts in drug development. Among them:
- The approval of two drugs, AQNEURSA™ and MIPLYFFA™, for Niemann-Pick disease type C in the U.S.
- The approval of REZDIFFRA™ for liver scarring due to fatty liver disease in the U.S.
- The approval of ACENOBEL® for GNE myopathy, a very rare disease characterized by muscle weakness that typically worsens over time, in Japan
- The approval of AMTAGVI™, the first cancer treatment using tumor infiltrating lymphocytes to make it to market as well as the first cell treatment approved for a solid tumor, in the U.S.
- The approval of VYLOY®, the first and only-anti-claudin 18.2-targeted therapy in the world, for gastroesophageal junction cancer in the U.S.
All told, there were more than 50 novel drugs with a first approval in major markets last year (you can find the full list in the Clarivate Drugs to Watch 2025 report). Many had the distinction of being first-in-class, delivered via a new administration route or being the first approved for an indication in decades.
This tide of groundbreaking treatments represents the culmination of years, sometimes decades of patient work by researchers and industry. It also reflects a push by regulators to fill treatment gaps for patient populations with high unmet needs, as evidenced by recent U.S. Food and Drug Administration (FDA) approvals of therapeutics like ADUHELM and ELEVIDYS.
Emerging and maturing modalities promise further firsts
Radiopharmaceuticals is not a new field – the first approved in the U.S., iodine-131, got the green light from FDA way back in 1951. However, a burst of activity around next-generation radiopharmaceuticals combining imaging and therapeutic components is beginning to deliver highly-targeted treatments to patients, and this burgeoning category is just getting started.
Similarly, gene editing treatments are just beginning to make their way to market – CASGEVY® became the first CRISPR-Cas9 gene-edited cell therapy approved globally at the end of 2023, green-lighted in tandem with LYFGENIA™, a gene addition therapy, by the FDA. As with radiopharmaceuticals, the promise of these technologies is inspiring a frenzy of M&A activity and partnerships among large pharmas seeking entry or to bolster their position (e.g., CASGEVY-maker Vertex’s $945m deal with Orum Therapeutics in July).
Perhaps the ultimate gold rush in pharma M&A right now is that around next-generation weight loss treatments, as the shock success of semaglutide and tirzepatide offers new (if expensive) hope for treating a disease with enormous and cascading effects on public health. Even as the effects of Wegovy® and Zepbound® ripple across the healthcare landscape other targets, individually and in combination with GLP-s, are being explored in clinical trials.
Learn more about these key trends, along with some of the most promising therapeutics on the cusp in 2025, in the Drugs to Watch 2025 report.
