Mapping the pathway

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:

  1. Cost savings: Setting up a study — any study — is the biggest cost related to a clinical study., including a patient registry. Once it’s up and running, it is just a matter of maintenance and periodic exploratory analyses.
  2. Time savings. Building a living registry allows biopharma companies to leverage economies of scale, tapping the same resource over and over again for new and more robust data. Without it, biopharma companies will find themselves in a perpetual state of ramping-up new research studies to accommodate the next HTA review.
  3. Identify delayed benefits and risks. Ongoing registries enable biopharma companies to track long-term patient outcomes, which they can use to validate claims about the long-terms benefits of their treatment and to identify any risks that may be important. This is particularly important for some interventions that may not show an immediate benefit, or for which there is concern about long-term side-effects.
  4. Fill information gaps. Rather than gathering disparate data from multiple disconnected databases, a registry offers a single source of information, which reduces the risk of data gaps and offers a more holistic picture of the total patient experience.
  5. Address comparisons. HTA assessments compare treatments to what’s already in the market and the issues that of the greatest concern. These issues and competing treatments can shift over time, making it difficult to know what data you will need for the next submission. An ongoing registry gathers data about multiple factors and indicators, which means researchers have a robust collection of data from which to make their case.
  6. Support other research. Registries offer rich data to support exploratory studies, and to answer early clinical questions that can help shape future trials and even to provide ideas for label extensions, for example, to different age groups or in combinations with other treatments such as are often encountered in oncology.

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. 

Topics in this blog post: Market Access, HTA, Registries