Real-World Evidence (RWE) In The HTA Process
With the significant rise in treatment cost, an increase of the number of drugs and the pressure of the treatments for special conditions and rare diseases, the regulatory and reimbursement systems in Canada, as one of the pioneers in this area, are going to collaboratively publish a framework for integration of RWE in the drug approval processes. Such a collaboration would be efficient for all stakeholders including the patients, payers, manufacturers, physicians, HTA and regulatory bodies (The full report).
Considering the increase of the interest in targeted therapy, treatment improvement, increase of patient’s QOL and survival rates by new products particularly large molecule drugs in the last two decades, it would be expected that health systems pay significantly more compared with the drug expenditure in the past. However, the decision-makers need to be more efficient in their decisions on recommending health technologies. The current reimbursement recommendations are mainly based on the evidence from RCTs. The conditions in RCTs is different from what the patients experience in the real world. The patient population in RCTs unlike RWD is smaller and causes greater uncertainty in safety, efficacy and effectiveness data even the RCT has already met all methodology requirements.
Integration of the RWE into the HTA systems has a substantial impact on the drug approval process and improves paying and using health technologies. To reach this goal, several challenges are needed to be addressed in advance such as:
- How to choose the best and most relevant data source with the highest level of quality?
- How to distinguish between higher and lower quality RWE for different conditions?
- Which RWD would be robust, transparent, reproducible?
- How to harmonize the data if different sources are used?
- How and in which step of the product approval process should we consider RWE?
- How to create a win-win situation among stakeholders to reduce the resistance to change?
- When and how an RWD analysis can issue causality?
- How much does it cost to protect the data produced by different stakeholders?
- How to protect patients’ privacy and data security?
- Do we need to monitor the market as long as the health technology patent has not been expired or it should be re-evaluated in a certain time period or just once at a certain time after the first-time recommendation?
and many others to successfully integrate RWE in the HTA process.