The FDA’s Center for Drug Evaluation and Research (CDER) 2024 annual report offers a fascinating insight into the rapid pace of innovation in pharmaceutical therapies. With 50 new drugs approved , 48% of which were first-in-class and 52% targeted at rare diseases , the report reveals a dynamic landscape in which translational science and clinical development are more integrated than ever before.
Key Facts and Figures for 2024
The year 2024 was marked by significant advances:
50 new drugs approved (novel drugs), maintaining the average of the last decade.
26 drugs for rare diseases , representing more than half of the approvals.
24 "first-in-class" drugs , reflecting the focus on innovative mechanisms of action.
66% of approvals used accelerated review programs such as Fast Track and Breakthrough Therapy.
94% of approvals occurred within the PDUFA deadlines, demonstrating regulatory efficiency.
The report also highlights, among the new drugs approved, the significant growth in biosimilar approvals, with 18 new products registered in 2024, including several classified as interchangeable.
Analysis of CDER data reveals trends that go beyond absolute numbers:
Rare Diseases in Focus : The fact that more than half of approvals are for rare diseases reflects the impact of incentive policies, such as the Orphan Drug Act , and the industry’s appetite for niche areas of high unmet medical need. This scenario highlights the potential for financial returns in areas of less competition and the positive impact for patients who previously had no therapeutic options.
Regulatory Accelerators in Action : Programs such as Breakthrough Therapy Designation and Fast Track were decisive for 66% of approvals, demonstrating that the FDA maintains a pro-innovation stance. The use of these fast-tracks is a reflection of the regulatory capacity to adapt to disruptive innovations, without compromising the safety and efficacy of treatments.
The Rise of Biosimilars : The increase in biosimilar approvals demonstrates a maturing regulatory ecosystem. In addition to expanding access, biosimilars play a crucial role in the sustainability of healthcare systems by reducing costs and promoting competition in markets traditionally dominated by high-cost biologics.
Advanced and Personalized Therapies : The advancement of gene and cell therapies demonstrates a maturation of biotechnology. The use of CAR-T cells , for example, continues to expand its indications beyond oncology, showing potential in autoimmune and infectious diseases.
Digital Health Integration : The use of artificial intelligence (AI) tools to accelerate drug development and real-time outcome monitoring has been a competitive differentiator for leading companies. AI has also played an important role in optimizing clinical trials, reducing costs and accelerating development time.
Impacts on the Global Innovation Ecosystem
Pharmaceutical and biotechnology innovation in 2024 is being shaped by factors that transcend the U.S. regulatory environment:
International Collaboration : Many of the FDA’s approved drugs have resulted from global partnerships, highlighting the importance of international research and development networks. Collaboration between pharmaceutical companies, biotechs and academic research centers has been key to accelerating the innovation cycle.
Population Ageing and Chronic Diseases : Increasing global life expectancy is driving demand for therapies for neurodegenerative, cardiovascular and metabolic diseases. This creates additional pressure on the healthcare system and highlights the importance of innovative solutions that offer not only efficacy but also cost-effectiveness.
Sustainability and Innovation : Balancing innovation and financial sustainability of healthcare systems is a growing challenge, requiring new pricing and access models. The introduction of value-based agreements is a growing trend to ensure that costs are aligned with real clinical benefits.
In Brazil, we face major challenges to keep up with the pace of global innovation, but we also have some unique opportunities:
Regulation and Efficiency : The FDA model highlights the importance of agile regulatory processes. ANVISA has made progress, especially with the implementation of the Innovation Policy ( Ordinance No. 1,100/2023 ) , which seeks to promote institutional results, facilitate the development of innovative products and enhance the agency's ability to deal with complex challenges.
Fostering Innovation : Initiatives by BNDES and FINEP to finance biotechnology are essential. BNDES is structuring a R$250 million fund to boost healthcare startups, while also approving billion-dollar investments for the national pharmaceutical industry.
Healthtech Ecosystem : Brazil saw a 16% growth in the number of healthtechs between 2019 and 2022, totaling more than 596 startups. These companies are transforming the sector with solutions in telemedicine, advanced diagnostics, and automation of clinical processes.
The 2024 CDER/FDA report is a reminder that health innovation does not happen by accident; it is the result of an ecosystem that values science, promotes regulatory efficiency, and encourages collaboration across sectors.
At IBIS, we believe that Brazil has the potential to be a relevant global player in healthcare innovation. To achieve this, it is essential to foster an environment that combines efficient regulation, strategic financing and a culture of collaborative innovation. IBIS understands the unique challenges faced by healthtechs, deeptech startups and biotechs in healthcare.
We offer customized services ranging from strategic planning and innovation roadmap creation to regulatory support to meet the requirements of ANVISA and other international agencies. In addition, we connect startups with strategic partners, investors and accelerators, enabling your business to grow sustainably. With our extensive experience in the pharmaceutical and biotechnology industry, we empower your startup with the tools and expertise needed to face challenges and explore its full potential in the global healthcare market.
Let's build this future together?
by Marcio de Paula
Brazilian Institute of Innovation in Health - IBIS
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