Real world evidence: The importance for rare disease therapies

Transcript of 'Real world evidence: The importance for rare disease therapies'

 

How is RWE used to supplement data for rare disease therapies?

So rare disease therapies can pose unique challenges for health care decision makers because of the less rigorous clinical trial data that's available to inform their potential benefits and risks. The inherent constraints in rare disease studies, for example, the small number of eligible patients, the heterogeneity of patient group, the short trial duration, pediatric patients, as well as the desire for patients and their caregivers to receive active therapy, can make the conduct of classical RCTs or randomized clinical trials to be implausible.

 

How is RWE used in the pre-launch phase?

So in the pre-launch phase, real world evidence, or RWE, can fill this knowledge gap by providing additional evidence that regulators, health technology assessment or HTA evaluators, and reimbursement stakeholders require for decision making. RWE can therefore be leveraged to help overcome uncertainties and concerns about the value of treatments in pre-market decisions, and therefore positively impacting access to medical innovation for rare disease patients.

 

How is RWE used in the post-launch phase?

So moving from pre-launch to post-launch, RWE can play a key role in evaluating whether and how rare disease therapies deliver on its promised value, both from a clinical as well as a healthcare system perspective. Post-launch RWE can be used to inform long-term safety and efficacy, utilization patterns, gain conditional approval from regulators, implement OBAs or outcomes-based agreements with payers, support pricing negotiation, and help shape value messaging.

 

What is the role of RWE in Canada?

So the Canadian government has committed to investing in national strategy for rare disease treatments, including improving the collection on use of RWE. Therefore, RWE is expected to play an increasingly important role in Canada in supporting various types of healthcare decision making in rare disease treatments.