DCTs a good fit for rare diseases say industry experts

Drug firms developing medicines for rare diseases should consider using decentralized trials according to researchers who point to the recruitment advantages of site-free studies.

The potential benefits were outlined in analysis in the Journal of Rare Diseases, with the authors arguing that decentralized studies can help firm’s developing drugs for rare diseases comply with EU requirements for access to medicines.

“As required by the EU’s Regulation (EC) No. 141/2000 on orphan medicinal products, “patients suffering from rare conditions should be entitled to the same quality of treatment as other patients,” and “it is therefore necessary to stimulate the research, development and bringing to the market of appropriate medications by the pharmaceutical industry.

“DCT, as a patient centric approach, provide more possibility for medical treatment research” the authors write.

Planning

Despite their advocacy, the authors do add caveats. The major requirement being that developers should validate and carry out pilot studies before beginning the full decentralized studies.

“Although DCTs have potential benefits, we should not overlook the accompanying challenges. For example, careful design and implementation of data collection as well as monitoring and validation of data such as effective digital biomarker is required to ensure the data quality and accuracy.”

They also say “More cautious planning and implementation of data management procedures are needed to ensure data privacy and security as data may be transmitted remotely.”

The authors – who include representatives from the drug industry as well as academics working in bioethics – also stressed that all stakeholders should be involved in the trial planning and development process.

“Sponsor clinical trialists and health authorities should work together to ensure sufficient rigor of study endpoints. At the same time, members of the patient and caregiver community should have input into study design.”

They conclude that “These discussions can define which elements of the trial must be done on site, and how DCT elements can be safely incorporated to enhance the trial participant and caregiver experience.”

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