Drugs

Lessons Learned from Rare Disease Trials During Covid

Two CDER Office of Orphan Products Development researchers say lessons learned from rare disease product development during the Covid-19 pandemic can be applied to future trials in rare diseases. Writing in Contemporary Clinical Trials, the authors say the two important lessons were:

virtual capabilities, when appropriate, can be an important trial component because they decrease the travel burden on participants and reduce in-person risks, which should increase patient recruitment and retention; and
building flexibility into clinical trials is critical in the post-Covid era and could include increasing the use of multi-site trials, clinical networks, and innovative designs and collaborations to speed trials without compromising study data.

The researchers studied all 62 grants that were being funded by the Orphan Products Grants Program between 3/2020 and 3/2021, gathering data from grantees and describing the effect of the pandemic on multiple aspects of the studies, including enrollment, patient follow-up, protocol, and budget.

The study says that the need to use virtual capabilities in rare disease clinical trials was essential to continue studies for vulnerable populations and was the highest reported pandemic effect in the analysis.

“Rare disease research, regardless of the status of the Covid-19 pandemic, could be further supported by increased flexibility in trial design and decreased need for patient travel, when appropriate,” the paper concludes. “Moving to decentralized trials where feasible or incorporating increased virtual capabilities in study designs can reduce the burden for rare disease patients, families, and caregivers in enrolling these studies, and promote efficiency and reduced costs for these trials. Planning for flexibility in the early stages of trial development is essential to adapt to changes while limiting effects on study data quality.”

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome