In September 2025, clinical development and clinical operations professionals will gather and discuss the current challenges in running clinical trials for orphan drugs and rare diseases at the 3rd Annual Clinical Trials in Rare Diseases (CTRD) meeting in Princeton, New Jersey, US.
The meeting, which will take place 17–18 September, will bring together pharmaceutical and biotech experts to discuss not only the obstacles and solutions to clinical trial operational issues but also to explore the evolving landscape of clinical trials for rare diseases, with a strong emphasis on innovation, collaboration, and patient-centric approaches.
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At the same venue, the 3rd Annual Clinical Data Management Innovation (CDMI) will explore ways to streamline data entry and collection at trial sites, make better use of real-world evidence, and address the evolving regulatory environment around clinical data. Both meetings will run in parallel at the same venue in Princeton.
The CTRD meeting will kick off with a case study on how AI is accelerating rare disease drug development. During the talk, Fortuity Pharma CEO Bruce Bloom will map out processes for pre-IND packages, working with the US Food and Drug Administration (FDA), and explore both the challenges and successes of applying AI in such trials.
At the CDMI conference, a keynote by Jeff Malavasi, associate director of Information Security and Data Compliance of Massachusetts-based biotech Scholar Rock, will focus on the growing cybersecurity challenges created by the digital transformation of clinical trials. His talk will also provide a practical framework for adopting AI-driven security solutions, including zero-trust architectures and automated response protocols, to safeguard sensitive clinical data.
Building on the theme of AI, Aamir Jaka, global vice-president of the clinical analytics company Saama, will later explore how agentic AI is moving data management beyond traditional rule-based automation.
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By GlobalDataPatient recruitment is a persistent challenge in rare disease research
On the same day, Jamie Switzer, associate director of clinical operations at Insmed, will present a case study on strategies to keep trials on schedule while tackling the issue of patient recruitment. Earlier this month, Insmed’s drug Brinsupri (brensocatib) was approved to treat non-cystic fibrosis bronchiectasis by the FDA based on results from the Phase III ASPEN (NCT04594369) and Phase II WILLOW (NCT03218917) studies.
Switzer will provide practical solutions to patient recruitment problems, while a subsequent panel discussion featuring industry leads from Alexion, Insmed, Asklepion Pharmaceuticals, and BioMarin will take the conversation further to discuss innovative ways to engage patients.
Patient recruitment and patient advocacy will be a recurring theme throughout the conference, with speakers sharing insights and emphasising the importance of having patient advocacy organisations as an indispensable partner to extend trial reach, co-design protocols, and foster genuine patient-centred practices.
Patient centricity will also be a key theme in the CDMI meeting, where Dinesh Pillaipakkamnatt, former director of Merck & Co (MSD), will highlight how to create a more personalised trial experience, while also considering data privacy, adoption barriers, and the safe handling of large volumes of sensitive information.
Tackling operational challenges with clinical trials
The second day of both meetings will commence with interactive roundtable discussions on topics such as the importance of decentralising rare disease trials, strategies to overcome patient retention barriers, best practices for managing external data and addressing issues of identity, integration, and privacy while enabling patient-centric data management across both conferences.
The patient and caregiver perspective with rare disease research will be explored with Leigh Dallow, patient advocate and caregiver, sharing her experience around trial participation, discussing trial enrollment decisions, and patient burden, in the CTRD meeting.
In a subsequent session, an expert from the Rare Disease Diversity Coalition will discuss the issues of inclusion and diversity in rare disease trials, focusing on underrepresented populations and ways to ensure fair access to therapies and research opportunities.
In the afternoon, the focus will shift to the strategic and operational elements of rare disease drug development. In a fireside chat, Fortuity Pharma’s Bruce Bloom will join PTC Therapeutics registry operations senior director Paul Lupo to discuss issues such as how to select a CRO and how to work with small patient populations. Another fireside chat, led by Kinjal Patel and Janine Sampong, both with Mitsubishi Tanabe Pharma America, will examine how to accelerate timelines when working with clinical sites and propose strategies to attract the attention of academic organisations and build strong partnerships.
Concurrently, the second day of the CDMI meeting will feature several sessions on successful strategies for CDM vendor selection and outsourcing models, an important update on ICH E6(R3) guidelines and its implications for clinical data management, and the implications of AI and machine learning for clinical data management. The meeting will conclude with a case study on CRO and sponsor collaboration, emphasising the importance of improving partnerships and aligning financial incentives.
The 3rd Annual Clinical Trials in Rare Diseases and the 3rd Annual Clinical Data Management Innovation conferences are hosted by Arena International Events Group, a B2B events company owned by GlobalData, the parent company of Clinical Trials Arena and Pharmaceutical Technology.
Click here to read the agenda for the conference for the 3rd Annual Clinical Trials in Rare Diseases and the 3rd Annual Clinical Data Management Innovation.
