Using registries to support HTA submissions in Japan
By: Nancy Dreyer, MPH, PhD | November 19, 2015
Health Technology Assessment (HTA) is becoming an essential part of market access across the globe — and Japan is no exception. HTA assessments differ from regulatory approval because they take into consideration multiple factors across the healthcare ecosystem to determine the ultimate benefits and cost of a new health therapy or technology. This means biopharma companies must contend with an ever-changing environment in which their drugs will be assessed.
A key component for success in these assessments is providing the Japanese Ministry of Health, Labor and Welfare, or any HTA group, with patient-centric data that include post-marketing studies that provide real-world evidence on safety, effectiveness and value. The Ministry will specifically want to see data that highlight who the treatment benefits and how a treatment will impact a patient given his or her situation, medical condition, gender and other key characteristics. Such data give HTA submissions proof to support for reimbursement, particularly if the sponsor a asking for a relatively high price for the treatment.
The challenge, however, is that most HTA submission data come from non-interventional (observational) studies, and in many countries — including Japan — there is no single database from which all of these necessary data can be obtained. Where databases exist, they are usually fragmented, providing only partial information, such as information about use of ambulatory care but not about hospitalizations and other high-cost events. It is difficult to link databases to track the full continuum of care, even in the best of circumstances. This creates gaps in the value story and weakens an HTA.
Drug developers also face the time and cost related to periodic HTA reviews, which are often required every three years. If sponsors cannot find an efficient way to collect and assemble this data, they often find themselves in a perpetual state of gathering data in preparation for the next HTA review — a costly endeavor!
Biopharma companies can make this process more efficient by creating living registries (on-going studies) of patients with the disease of interest.
A patient registry is an organized system that uses observational study methods to collect uniform clinical, payment and other relevant data for a specified study purpose. Once enrolled in a registry, clinicians and/or patients report on all treatments and key metrics of disease cure or progression. They can also provide quality of life information, such as the ability to perform activities of daily living like the ability to dress themselves, and whether they are well enough to work. They may also provide information on other risk factors that could mitigate the benefits of treatment — certainly important factors to understand when trying to demonstrate the benefit of a treatment.
By creating a registry infrastructure and maintaining data collection, sponsors can generate a steady stream of data that can be used to benchmark their treatment against standard of care or particular competitors. They can also identify subgroups that demonstrate particularly strong benefits and cost-savings, and may isolate some groups that do not show any benefit. This approach will allow sponsors to provide these answers in a lot less time and for a lot less cost than if they begin this data collection process from scratch with each new HTA submission.
Here are six reason registries make HTA submissions stronger:
If a treatment requires ongoing HTA assessments, there is no question that an ongoing patient registry will save substantial time and money while improve the quality of data and reducing information gaps. Once the infrastructure is in place, it is merely a matter maintaining the registry, adapting fields to accommodate new data requirements and mining the data when it is needed.