Gene Therapy

Stay on Track  

The paradigm of regenerative medicine development is changing. Expedited regulatory approvals can help get your much-needed therapy to patients earlier, but only if you are timely with your strategic planning.

Being ready to move forward expediently after regulatory approval means you need to fully explore endpoints, start planning for reimbursement, and begin developing a market access strategy prior to your gene or cell therapy or tissue engineered product obtaining regulatory approval. 

If your advanced-therapy product has received fast-track designation, like the US FDA’s Regenerative Medicine Advanced Therapies (RMAT) or the EMA’s Advanced Therapy Medicinal Products (ATMP) designations, getting a jump on this planning is even more critical.

We Can Help

Our experienced team can help you stay on track. We’ll put together an integrated team of experts to help you ask relevant questions and gather the right data to build an informed strategy for regulatory approval and post-approval activities - giving you the best opportunity to obtain market access for your product as quickly as possible after approval.

A Wealth of Experience

We have collaborated with our clients on projects to research a variety of cell and gene therapies. Our experience with these therapies includes several disease areas:

  • Sickle cell disease
  • Neuronal ceroid lipofuscinosis type 2 (CLN2) disease
  • Duchenne muscular dystrophy
  • Spinal muscular atrophy

    Types of Projects

    We have implemented studies to help our clients develop strategies and gain market access for regenerative therapies. Projects have included:

    Our Experts

    Amy Barrett
    Senior Director, Patient-Centered Outcomes Assessment
    Carla DeMuro
    Executive Director, Patient-Centered Outcomes Assessment
    Carol Mansfield
    Senior Economist and Head, Health Preference Assessment

    Selected Recent Gene Therapy Publications