Today, there are more than 7,000 rare diseases affecting nearly 30 million Americans, half of whom are children. While there has been significant progress with modern medical science and the development of treatments, only about 5 percent of rare diseases have an approved treatment option.

Gene therapies target and correct rare disorders caused by errant genes. They are the frontier for potentially transformative, durable treatments and are targeting many unmet medical needs. Innovation in the gene therapy pipeline is thriving but more needs to be done to ensure these therapies make it to market.

Because gene therapies are complex and costly to develop, they can carry large upfront price tags. These high prices combined with the current US reimbursement system, which is largely based on volume of sales and standards of care rather than outcomes achieved, means that accessing gene therapies can be extremely challenging for patients. We need a system that encourages more innovative payments to ensure patient access and affordability without forsaking innovation.

Join the Campaign for Transformative Therapies for a virtual briefing to learn more about:

• The burden of rare disease across the US
• What it’s like to develop gene therapies and how they work; and
• Federal barriers that need to be fixed to ensure future patient access.

Speakers

• Annie Kennedy
  EveryLife Foundation
• Will Hubbert
  Hemophilia Patient Advocate
• Glenn O’Neill
  Cure Sanfillippo Foundation
• Dr. Steven Pipe, MD
  University of Michigan
• Sloane Salzburg
  CTT/CAHC, Moderator

Click to View Slides