PharmaVOICE Blog Post

Leveraging Real-World Evidence for Regenerative Medicine and Advanced Therapy Success Beyond the Regulator

Posted By: Dan Limbach
December 7, 2017

In the Fall issue of The Evidence Forum, one of the many informative articles covers the promising opportunities of regenerative medicine in reversing or significantly impacting the progression of – and costs associated with – serious illnesses. These treatments have the potential to transform the healthcare landscape, offering transformative, durable, and (in some cases) even potentially curative outcomes targeting many of our highest unmet need scenarios, including life-threatening acute and chronic conditions, injuries, degenerative diseases, genetic disorders, and cancer.

The 21st Century Cures Act establishes an optimized FDA approval pathway for regenerative medicine therapies, encouraging innovation while balancing patient safety and accelerated access.  This article addresses how these treatments are defined, how they differ from conventional therapies, and important factors in demonstrating their value to optimize their success.

Here is a sample of what you will see in the article…

The global market for small molecules and biologics, gene therapy, and cell therapy is expected to grow to $67.5 billion by 2020 (a more than fourfold increase from $16.4 billion in 2013). Regenerative medicine saw venture capital investment nearly quadruple from ~$200 million in 2010 to ~$800 million in 2016, signifying a 34% average year-over-year growth rate during that period. The strong, consistent investment and market growth in the regenerative medicine space signals a future intensely-competitive landscape where differentiating product value will be key.

Clinical trials for regenerative medicine therapies are often insufficient to capture the total magnitude of potential benefit to the patient, the payer, and the healthcare system overall. Contributing factors to this hurdle include rapid evolution and variability of early regenerative and advanced therapy platforms; the need to demonstrate longer-term benefits of transformative and potentially curative treatments versus historical trial considerations; and, unknown side effects associated with these truly novel therapies. Use of real-world evidence (RWE) approaches will be critical to establishing the transformative benefit, durability, and safety outside of the pivotal studies needed for regulatory approval.

Developing a strong value story for regenerative and advanced therapies requires demonstrating evidence of long-term effectiveness and safety, and clearly showing that pivotal outcomes translate into longer-term transformative benefit.

Lessons Learned: Opportunities to Position Regenerative and Advanced Therapies for Success

  1. Plan to build a comprehensive and long-term value story
  2. Understand the patient (that will be included in the study) and patient journey
  3. Establish a foundation for rationale for positioning and pricing; ensure outcomes and value story are clear and meaningful
  4. Characterize the resources and economic impact associated with the therapy

More details about the lessons learned are included in the full article.

The article also contains seven valuable charts which help explain the complex issues and help you develop your roadmap to success.

Download the full article pdf at

Download the full issue with numerous articles at

Provided by Evidera

About the Blog Poster: Dan Limbach

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