TOKYO -- Japan's health ministry will issue new guidelines next fiscal year aimed at helping new drugs get to market faster, by allowing pharmaceutical companies to use data from outside of clinical trials.
The health ministry will accept medical information for patients that have taken the drug in other settings, in place of, or as a supplement to, data on a placebo group. Requirements for clinical trials, including duration and number of participants, will also be relaxed. The guidelines will be drawn up before this fiscal year ends in March.
The goal is to keep work on promising drugs from stalling in cases where traditional trials might be difficult.
The gold standard for clinical research is randomized double-blind controlled testing, in which one group of participants is given the drug and the other receives a placebo, and neither patients nor doctors know which is which. But with treatments for rare diseases, for example, researchers may not be able to line up the necessary number of participants.
And some people are reluctant to enter such trials when there is a possibility that they might not receive the drug themselves. Double-blind tests of the flu drug Avigan's effectiveness against the coronavirus, for example, had trouble recruiting patients, as the treatment was available in other research settings without the risk of getting a placebo instead.
Japan offers a fast-track drug review option that allows for some of the usual steps to be postponed until later, shortening a process that can normally take up to a year. But this does not let drugmakers skip submitting crucial clinical trial results.
The new Japanese guidelines will express the view that allowing real-world data in the approval process can encourage drug development, while noting concerns about the possibility of drugmakers re-analyzing data in different ways until they get the results they want.
The ministry has begun accumulating real-world data on certain conditions such as cancer and diabetes. The National Institutes of Biomedical Innovation, Health and Nutrition is working on a database of information on coronavirus patients collected from university hospitals and other sources, which will be shared with drugmakers and medical institutions.
Real-world evidence is coming into broader use elsewhere in the world. The U.S. Food and Drug Administration is conducting coronavirus-related research with an American life sciences company, using data from electronic health records and other sources to analyze treatments and complication risks.
There are hurdles to collecting such data in Japan that may bog down drug development. Electronic medical records from different vendors cannot be integrated easily, for one.
"Bringing the data together takes money and effort," said a representative at a company that integrates health information from different hospitals. The U.S. has spent billions of dollars since 2009 to try to standardize its records.