Innovative Solutions for Fast-Tracking Drugs for Rare Diseases

Achieving regulatory approval alone no longer determines a drug’s or therapy’s commercial success or even guarantees its market launch. Today, each product must be evaluated from a value perspective by payers and health authorities to be placed on the formulary, factored into reimbursement rates, and put into treatment plans before it is available for healthcare providers to prescribe.

Approval processes are also adapting to meet the high unmet needs of patients with rare diseases where treatment options are limited or non-existent.  A disease is considered to be rare when it affects fewer than 1 in 2,000 people. Although each rare disease does not affect a large number of patients, there are estimated to be over 7,000 rare diseases.

The options for fast-track and conditional approvals for products to treat rare diseases are rapidly evolving to get medicines to patients quicker. These options include using a combination of real-world evidence (R2WE) and innovative contracting techniques to give payers confidence to reimburse a novel product. RWE is the clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of real-world data (RWD). Real-World Data are data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources including electronic health records (EHRs); medical claims and billing data; data from product and disease registries; patient-generated data, including from in-home-use settings; and data gathered from other sources that can inform on health status such as mobile devices.

By watching this webinar, you will learn what works and what doesn’t in our rapidly changing regulatory and reimbursement world. Oliver Leatham, Vice President, Global Value & Access at Certara, discussed the following:

  1. Case studies of approved drugs with limited market access – What went wrong?
  2. Analogues of products that have successfully navigated the reimbursement landscape using innovative contracting
  3. How RWE can support payer confidence and market access to sustain premium pricing and drive market share
  4. How sponsors are approaching the changing regulatory and reimbursement environment to stay ahead of the competition