Deep Dive: Moving Away From RCTs for Rare Disease Treatment Evaluations

Deep Dive: Moving Away From RCTs for Rare Disease Treatment Evaluations

Join Dr. Janet Woodcock and Dr. Marshall Summer as they discuss their two review papers in Clinical and Translational Science on rare disease trial design: “Supply and Demand in the Mathematics of Rare Disease Drug Development: Why Choosing the Right Model is Crucial [https://ascpt.onlinelibrary.wiley.com/doi/10.1111/cts.70551]” and “The Placebo Effect in Rare Disease Clinical Trials: Measurement Impact and Statistical Approaches for Patient as Own Control Designs [https://ascpt.onlinelibrary.wiley.com/doi/10.1111/cts.70550].” They delve into why traditional randomized controlled trials can be infeasible for small, heterogeneous rare disease populations, how patient-as-own-control and other alternative statistical designs can add rigor while reducing false negatives, as well as practical ways to address placebo effects, bias, and endpoint selection, emphasizing natural history studies and patient-centered input. You can find more information about The Haystack Project mentioned in this episode here: https://haystackproject.org/ [https://urldefense.com/v3/__https:/haystackproject.org/__;!!N11eV2iwtfs!tZixA9DmHh63HRBj_53QVYCPA2_pXE4R2kJARDvlDSedaFmLPX-JyBADbxscnpMbg5Of9lYr7i_SLQ$].

CTS Call for Papers: Celebrating 10 Years with ASCPT

Join Clinical and Translational Science editors Erica Woodahl and Liewei Wang and Editor-in-Training Mai-Uyen Nguyen to hear about CTS’s call for papers for a special collection [https://urldefense.com/v3/__https:/www.ascpt.org/Resources/ASCPT-News/View/ArticleId/28778/CTS-Call-for-Papers-10-Years-of-Translational-Science-Driving-Precision-Medicine__;!!N11eV2iwtfs!reGhnLo1cJbeYepw633Ebwt5DP1Z7cwGYyjfzzWOWFpeakjhHB_mNfJSc6O1E407gE5tFl6NKxDyqQ$] on how translational science can advance precision medicine, marking the 10th anniversary of CTS joining the ASCPT Journal Family. The editors highlight desired submission areas including translational approaches, new tools and technologies from preclinical models to clinical implementation, trial design and real-world evidence, regulatory/ethical/policy considerations for genomic data and testing costs, and pharmacoequity. Submissions are due October 1, 2026.

CPT Call for Papers: Understudied Populations

Working on research focusing on an understudied population? Consider submitting to the CPT themed issue “Advancing Therapeutics for Understudied Patient Groups [https://urldefense.com/v3/__https:/www.ascpt.org/Resources/ASCPT-News/View/ArticleId/28743/CPT-Call-for-Papers-Understudied-Populations__;!!N11eV2iwtfs!qgfvX-f03-EFijJJVyzq0BQbrmvarmBH89bU248vld1cQBjxtfqAMKd59nQSE1W10x_zBdLSTGMmdA$].” This episode dives into the call for papers and what aspects of this topic editors are really hoping to see highlighted in submitted research. Submission deadline: June 30, 2026.

CPT Call for Papers: Advances in New Approach Methodologies (NAMs)

Check out the latest Clinical Pharmacology & Therapeutics call for papers on “Advances in New Approach Methodologies (NAMs): From Bench Development and Validation to Regulatory Adoption.” The submission deadline is June 1, 2026, so submit your research soon!

End of an Era: CPT Says Farewell to Piet van der Graaf and Kathy Giacomini

Listen in as former Clinical Pharmacology & Therapeutics Editor-in-Chief Piet van der Graaf and former Deputy Editor-in-Chief Kathy Giacomini discuss their journeys to clinical pharmacology, ASCPT, and the CPT editorship. They reminisce about mentors and influential people in their lives, challenges and milestones faced at CPT, and how scientific publishing continues to evolve and change.