More Pipeline Balance
ROI from clinical research can suffer when too many research organizations seek to develop treatments for similar conditions and indications
ROI from clinical research can suffer when too many research organizations seek to develop treatments for similar conditions and indications, making it more difficult for any one pharma company to achieve medical and financial success. Financial analysts note that one-third of R&D spending is going into oncology and inflammatory conditions, and that pipelines also are over-crowded with potential treatments for melanoma and rheumatoid arthritis. Yet research programs have shrunk for new antibiotics, as well as for treatments for heart disease and central nervous systems disorders, which affect larger patient populations.
Much of the enthusiasm for developing new cancer therapies lies in the ability to target population subsets, which often supports designation as an orphan drug and/or breakthrough therapy. Such status reduces regulatory fees and opens the door to added advice from FDA experts and speedy approval, based on small, limited studies.
In some cases, single-arm studies may be sufficient to demonstrate activity, even though such limited trials provide little information on safety, explained Richard Pazdur, irector of the Office of Hematology and Oncology Products in the Center for Drug Evaluation & Research (CDER). Pazdur observed at the FDA/CMS Summit in December that he was not a “big fan” of single-arm studies a few years ago. But for a drug with a 50% response rate for a condition with no existing effective therapy, a limited study may be enough. However, the emergence of more oral cancer therapies for chronic use may require sponsors to look more at safety, Pazdur added, noting that clinical trials for such drugs have to capture toxicities and examine what is a tolerable dose.
FDA Approves Nipocalimab for the Treatment of Generalized Myasthenia Gravis
April 30th 2025Approval is based on results from the pivotal Vivacity-MG3 trial in which IMAAVY (nipocalimab-aahu) demonstrated superior disease control throughout 24 weeks when compared to placebo plus standard of care.
Improving Relationships and Diversifying the Site Selection Process
April 17th 2025In this episode of the Applied Clinical Trials Podcast, Liz Beatty, co-founder and chief strategy officer, Inato, discusses a number of topics around site engagement including community-based sites, the role of technology in improving site/sponsor relationships, how increased operational costs are impacting the industry, and more.
Behind the Buzz: Why Clinical Research Leaders Flock to SCOPE Summit
February 7th 2025In this episode, we meet with Micah Lieberman, Executive Conference Director for SCOPE Summit (Summit for Clinical Ops Executives) at Cambridge Innovation Institute. We will dive deep into the critical role of collaboration within the clinical research ecosystem. How do we bring together diverse stakeholders—sponsors, CROs, clinical trial tech innovators, suppliers, patients, sites, advocacy organizations, investors, and non-profits—to share best practices in trial design, program planning, innovation, and clinical operations? We’ll explore why it’s vital for thought leaders to step beyond their own organizations and learn from others, exchanging ideas that drive advancements in clinical research. Additionally, we’ll discuss the pivotal role of scientific conferences like SCOPE Summit in fostering these essential connections and collaborations, helping shape the future of clinical trials. Join us as we uncover how collective wisdom and cross-industry partnerships are transforming the landscape of clinical research.
