21st Century Cures Act and Real-World Evidence
Maria Harrison
Maria Harrison
VP and Global Head of Real World Solutions

The 21st Century Cures Act, enacted in 2016, provides many potential opportunities for biopharmaceutical companies to advance their development pipelines more quickly and more efficiently in the U.S.

While the act was approved with a budget of about $500 million, annual congressional budget negotiations will further determine the actual amount of additional funds, if any, redirected toward specific areas of focus. Nonetheless, the overall value to biopharmaceutical companies will occur in the coming years in the form of:

  • Practical, streamlined approaches to the U.S. FDA product approval pathway, particularly in the rare disease and pediatric settings
  • Increased FDA willingness to embrace innovations in clinical trial designs
  • The use of real-world evidence to support new product approvals and label expansions
  • Reinforcement of data sharing and academic networks (in adult and pediatric settings)

Some other opportunities for pharma companies include:

  • Comprehensive modifications to the U.S. Food and Drug Administration (FDA) development and approval process, such as:
    • Product development tools, such as biomarkers and clinical outcome assessments
    • Real-world evidence in support of product reviews and approvals
    • Patient engagement in the drug development process
    • Orphan Drug Act compensation extended to cover more of drug development costs for rare diseases
  • NIH (National Institutes of Health) investments in biomedical research, precision medicine neuroscience, and oncology
  • Creation of disease centers of excellence

The magnitude of these opportunities, however, will depend on the actual details of implementation stipulated by various regulatory agencies; congressional funding levels year over year; and the willingness of key stakeholders in the research process to embrace these concepts.

Implementation of the various components of the act will take considerable time (the act is funded through 2025) with the typical process as follows:

  • 180 days for agencies to develop an implementation plan
  • 12-18 months of public comment periods
  • 12-18 months of finalization based on public comments

The biopharmaceutical community will need to remain fully engaged in collaborating with regulatory agencies, policy makers, academic stakeholders, and bioethicists to ensure the details of the implementation achieve their intended goal of effectively and efficiently bringing new therapies to market while continuing to protect patients’ rights and well-being.

PRA’s overall business strategy and current operating model are well aligned with the 21st Century Cures Act. For instance, over the last 10 years, PRA has proactively:

  • Invested heavily in our data science/real-world outcomes solution
  • Built a pediatric research network and operational working group
  • Established significant expertise in rare diseases
  • Created technologies to accelerate the drug development process

PRA’s executive management and related functional groups continue to closely monitor the implementation efforts of the 21st Century Cures Act and we will be prepared to comment on the draft implementation to ensure our operational model remains fully aligned.

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