Decentralized Clinical Trials for Rare Disease Treatments

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Decentralized clinical trials offer the potential to democratize rare disease research, advancing drug development and improving patient experiences.


In rare disease (RD) research, traditional site-bound trials face unique challenges, including restricted patient populations, participant identification difficulties, and a lack of understanding of the natural history of the disease. However, the emergence of decentralized clinical trials (DCTs) presents a transformative avenue for trials. The increase of DCTs facilitated through remote technologies has been amplified by the COVID-19 pandemic. These trials have the potential to advance the research landscape by enabling patients to engage in their own environments and reducing the need for frequent site visits.

The shift toward decentralized trials extends beyond trial logistics; it has the potential to reshape the entire approach to clinical trials, evidence generation, and data communication. This transition aligns with the pharmaceutical pipeline’s increasing focus on RDs, spanning gene and cell therapies, mutation-specific oncology, and RNA-interfering technologies.

The modernization of clinical trials and RD research provides unique opportunities for stakeholders, including patients and manufacturers. However, several key factors must be considered.

Enhanced Patient Access and Participation

The dispersed nature of RDs can make decentralized trials a logical approach for certain manufacturers. Global research, advanced digital tools, and access to real-world data can potentially allow for more discoveries and provide effective treatments. This method leverages expertise from researchers and healthcare institutions worldwide, which allows for geographical barriers to be overcome and enables patient participation from homes or local healthcare facilities. By removing the barriers of physical distances, DCTs can bring diverse populations into the research fold, offering more understanding of disease impacts across ethnicities and regions.

Integrating digital health technologies like monitoring devices and electronic patient-reported outcomes supports patient engagement through this research. In turn, this engagement can support increased study retention and generate real-time data. Manufacturers can support this through user-friendly apps and remote monitoring devices, which can foster continuous communication with researchers and promote adherence to treatment protocols.

Accelerating Market Access

Meeting regulatory expectations requires robust clinical evidence across various domains. For RD therapies, the complexity of the payer landscape with its emphasis on cost-effectiveness and real-world evidence (RWE) adds an additional challenge. DCT implementation can help address these challenges.

Streamlining data compilation from DCTs can facilitate RWE collection, potentially supplementing or diminishing the need for traditional clinical trial data. RWE can provide payers with comprehensive insights into treatment outcomes and real-world scenarios, potentially enabling informed, data-driven decisions during negotiations. RWE showcases real-world cost data, potentially strengthening the treatments case and expediting market access negotiations by supplying evidence such as reductions in hospitalizations or emergency room visits. Moreover, robust data reinforces treatment efficacy and safety, potentially expediting regulatory approvals.

Furthermore, the embrace of RWE by the Food & Drug Administration (FDA), exemplified by the Pre-Approval Information Exchange Act, signifies a shift toward evidence-based evaluations. This act enables payers to assess clinical trials and RWE before FDA approval, ensuring a parallel evaluation process.

Regulatory Recognition and Integration

Regulators are collaborating with patient advocacy groups and stakeholders to gauge the practicality and implementation of DCTs, heralding new regulatory guidelines. The FDA has outlined regulatory expectations for implementing DCTs in their draft guidance published in May 2023. The COVID-19 pandemic accelerated these efforts by adapting trials to virtual formats in response to distancing measures. The draft guidance paves the way for greater involvement and clarity in decentralized trials, addressing persistent challenges in RD research, such as recruitment hurdles.

The integration of streamlined data collection, compelling RWE, and alignment with regulatory and payer interests mark a paradigm shift in RD research and treatment access.

Connect with Us

Cross-functional engagement and strategy are vital in ensuring the evolution and optimization of each of these variables—patient access and participation, accelerating market access, regulatory recognition and integration—to allow for maximum benefit to patients. To learn more about how Avalere applies its robust expertise at the intersection of digital health technologies and patient and market access strategy to help stakeholders maximize opportunities for the implementation of DCTs, connect with us.


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