Can real-world data transform the rare disease research landscape? 

Cytel and Pulse Infoframe have collaborated to create a solution that marks a new direction in research for rare disease. This partnership will support drug development sponsors seeking to implement real-world evidence solutions where the data generated can be used for a range of purposes, from synthetic controls and comparative effectiveness to pricing reimbursement. 

Their approach  

The companies will combine their expertise to channel real-world evidence toward new treatments in rare disease. Rare disease research and drug development have historically been confronted with limited sample sizes, so they have required innovative approaches to clinical development and evidence generation. The companies therefore consider the use of real-world data to support regulatory and payer submissions essential to ensure that stakeholders review the most comprehensive evidence package, enabling faster access to new treatments in areas of high yet unmet need. 

Official comments 

Dr. Radek Wasiak, Chief Data Officer and the head of Real World and Advanced Analytics at Cytel, said: “Advances in precision medicine enable life sciences companies to develop better and more targeted treatments. However, in rare diseases, this translates to practical difficulties in executing the necessary research activities. Combining Cytel’s pioneering advances in efficient clinical trial design with Pulse Infoframe’s healthie platform and therapeutic area expertise will accelerate our ability to provide our life sciences partners with research needed to obtain regulatory and payer approval.” 

Dr. Femida Gwadry-Sridhar, the CEO and founder of Pulse Infoframe, said: “Rare disease patients can wait years for even the smallest advancement in treatment. In many cases, those advancements may not come at all. By collaborating with Cytel, a company renowned for their quantitative methods for improved research outcomes and exemplary trial design, we can help accelerate drug development for rare diseases.” 

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