Seriously ill patients will be allowed to participate in the final stages of clinical trials for drugs that have not been approved by the government starting early next year, a decision made by the health ministry from a humanitarian perspective.
The Health, Labor and Welfare Ministry's new system will make unapproved drugs in trial stages accessible to patients suffering from types of cancer that cannot be effectively treated, as well as those with other intractable diseases. The ministry will introduce the system by revising relevant ordinances by the end of the year.
The envisaged system will allow patients who have not been allowed to participate in conventional drug trials to take part, including patients with other kinds of chronic diseases.
A prominent feature of the system is an eased financial burden on patients. Expanded access to cutting-edge medicine will also allow some patients to expect a better prognosis.
The new system will cover unapproved medicine in the final stages of clinical trials, after the initial stages confirm to a certain degree the effectiveness of the drug and the presence of any side effects.
Under the new system, information about medicine used in clinical trials will be disclosed to the public, in principle, on the website of the Pharmaceuticals and Medical Devices Agency (PMDA) - an independent administrative institution. Currently, details about clinical drug trials are in many cases only known by pharmaceutical companies and attending doctors.
Patients who obtain information about trials will inform their attending doctors if they want to participate in clinical trials. Attending doctors will then consult doctors in charge of the trials, and pharmaceutical companies will decide if the patients can take part.
Ordinary clinical trials generally use unapproved medicine and placebos. Pharmaceutical companies also set criteria for the kinds of patients who can participate in trials, based on factors like age, body condition and whether the patients have other chronic diseases. For example, patients diagnosed with chronic heart disease are not allowed to participate in clinical trials for anticancer drugs.
New support system
Under the ministry's new envisaged system, placebos will not, in principle, be used. Pharmaceutical companies will also in principle not be allowed to automatically reject requests from patients to participate in trials only because they do not fulfil set criteria.
The new system will establish a support system for patients who wish to participate in clinical trials but are rejected by pharmaceutical companies. These patients will be able to file complaints with the health ministry through their attending doctors.
If the ministry's panel of experts deems that a patient's participation in clinical trials is appropriate, the ministry will ask relevant pharmaceutical companies to accept the patient and include them in their clinical trials.
However, clinical trials under the new system must meet the requirement that participation for humanitarian purposes will not negatively affect the development of new medicine.
In this connection, some pharmaceutical companies may end up refusing patients for clinical trials that see an excessive number of applications and patients who are in critical condition.
Starting next April, the government will start rolling out another new system that will allow the concurrent use of conventional and advanced treatments not covered by public health insurance schemes if a request to do so is submitted by a patient.
This system will similarly allow the use of drugs that have yet to be approved, but patients will be required to shoulder most of the costs on their own.
In many cases, pharmaceutical companies cover the costs of clinical trials for unapproved medicine, so if a patient wants to participate in a trial under the system for humanitarian purposes, the request will be handled separately from the other new system set to begin in April.