Clinical Research: Phase 1 - Phase 4

8 Cost Containment Strategies for Rare Disease Clinical Trials

In a world of ever-increasing clinical trial costs, sponsors can feel overwhelming pressure to stretch their budgets without sacrificing the data they need for regulatory approval. In rare diseases, this pressure is intensified with the many challenges that often accompany these trials, such as small patient populations, limited disease data, and patient engagement challenges. 

Here are 8 strategies to help contain clinical development costs in rare disease research, without compromising on quality.

  1. Trial Design Optimization
    Streamline data collection by focusing on essential assessments and flexibility in visit windows to reduce the patient’s burden. Consider options such as home health visits and telemedicine to minimize the need for extensive patient travel.

  2. Data Collection through eSource Options
    Exploit the potential of eSource options, such as electronic patient-reported outcomes (ePRO) and mobile technology, to streamline data collection. Beyond improving data quality, these technologies reduce administrative overhead.

  3. Collaboration with Key Opinion Leaders (KOLs)
    Foster strong partnerships with KOLs and advocacy groups early in the design process to refine eligibility criteria, reducing the necessity for protocol amendments. This collaboration also enhances patient recruitment and retention.

  4. Case Report Form (CRF) Size
    Exercise careful consideration in determining the amount and frequency of data collection which determine the size of the dataset. Not only will this control the visit costs but also the data collection and cleaning costs for the trial.

  5. Sample Size
    Collaborate with experienced biostatisticians to explore alternative trial designs, such as crossover or adaptive trials, or in silico modeling. These innovative approaches can significantly reduce the required number of patients while maintaining statistical rigor.

  6. Technological Innovation
    Venture into new territories with decentralized trial options, wearable devices, and direct data capture via smartphones. These technological frontiers enhance patient compliance and retention, aligning with the ever-evolving clinical research landscape.

  7. Central Monitoring
    Leverage centralized data monitoring as an early warning system that reduces the need for frequent onsite visits. This strategy not only accrues cost savings but also upholds data integrity.

  8. Risk-Based Approaches
    Define acceptable levels of risk across various data types, ushering in a new era of flexibility in data monitoring strategies. This approach optimally allocates resources while safeguarding data quality.

Breaking the Mold Without Breaking the Bank

The challenges of rare disease research are undeniable, from limited patient populations to the weighty burden of high clinical trial costs. However, striking a balance between innovation and budget constraints is possible. To learn how Premier can help you find this balance in your next rare disease trial, contact us.