Access to Investigational Medicines: Our Approach
Dr Amrit Ray, Chief Medical Officer, Janssen, explains our approach to access to investigational medicines.
At Janssen, the well-being of patients is the reason we come to work each day. Our goal is to help them by developing effective and safe medicines.
We are often asked how patients with serious diseases can get medicines not yet approved by government health authorities. These medicines are still being studied and are also called "investigational medicines." In the United States, the government health authority is the Food and Drug Administration (FDA).
While government health authorities have made great advances in shortening the time it takes to make new investigational medicines available, some seriously ill patients are still in urgent need of new treatment options. We understand that urgency, especially for patients with medical needs that are not met by treatments available now.
We follow three important principles when providing access to investigational medicines:
- that all patients are treated fairly and equally
- that we conduct thorough scientific studies to understand the potential risks and benefits of any investigational medication. These studies provide the information needed to seek approval from government health authorities and bring new medicines to all patients who need them.
- that we are not putting patients at risk of unnecessary harm.
We cannot provide an investigational medicine to patients until we have enough information to believe that the risks associated with a particular medicine are reasonable in light of the potential benefit, and that we have preliminary proof that the drug may work. The information below provides details about available options for gaining access to investigational medicines and how to request more information.
One method for gaining access to Janssen's investigational medicines is to enroll in a clinical trial. Clinical trials are scientific studies in which investigational medicines are tested to make sure they are safe and effective for people to take. They are one of the most important steps in bringing new medicines to patients.
Clinical trials allow pharmaceutical companies like Janssen to get the scientific proof we need to ask a government health authority to approve our medicines. Getting that approval is important because it means that doctors can then prescribe the medicine to patients who may need it.
Researchers design clinical trials to answer specific scientific questions. Trials must be conducted as fairly and ethically as possible. Clinical trials are designed:
- based on accepted principles of medical research
- to meet the laws and regulations of government health agencies
- to make sure investigational medicines are safe and effective for people to take, and
- to obtain the government health authority approvals needed to bring new medicines to the largest number of patients in need.
Clinical trials must be approved by government health authorities before patients can enter the trial and receive the investigational medicine. There are strict rules around how clinical trials must be run, and each trial is different. Patients must meet specific criteria to be included in a clinical trial. For example, to enter a clinical trial, the patient must have the type of disease that is being studied or patients may need the type of disease that is being studied AND have already tried other medicines that did not work as well as expected.
Sometimes patients cannot enter a clinical trial if they have other health conditions or problems in addition to the type of disease being studied or are taking certain other medicines that might interfere with the trial results or put patients at unreasonable risk. For this reason, in some cases, these patients are not allowed to join a clinical trial.
Ask your doctor if participating in a clinical trial is right for you. All of Janssen’s trials are listed publicly at www.clinicaltrials.gov.
In addition to speaking with your doctor, additional information about clinical trials is available on the following sites:
Expanded Access Programs (EAPs)
If a clinical trial is not available or right for you, there may be another option. Patients may sometimes obtain access to an investigational medicine through "expanded access programs," or EAPs. EAPs are programs that provide a way for pharmaceutical companies to provide some investigational medicines to patients before the medicines have been approved by the government health authority in the country in which they live. EAPs also need to be approved by government health authorities.
EAPs are only authorized by the U.S. FDA for:
- patients with a serious or life-threatening disease or condition,
- patients with no comparable or satisfactory other treatment options available
In addition, there needs to be sufficient information to believe that the risks associated with a particular medicine are reasonable in light of the potential benefit.
At Janssen, generally we consider opening an EAP in the US when our clinical studies are done and we are waiting for approval of our investigational medicine from government health authorities. However, an EAP is not opened for every investigational medicine before it is approved.
Janssen does not offer EAPs when investigational medicines are in early testing because there are still too many unknowns and we believe providing the medicine to patients at this point would put them at undue risk. Keep in mind that when an investigational medicine is first being tested in people, we are still learning which specific patients might benefit from the treatment and what types of side effects the medicine may cause.
Ask your doctor if an EAP is right for you. We list our EAPs publicly at www.clinicaltrials.gov.
How to Get More Information
The best and fastest way to get information about whether or not you may be able to gain access to an investigational medicine being studied by Janssen is to have your doctor call Janssen Medical Information at 1-800-Janssen or email us at firstname.lastname@example.org.
Trained professionals are available at this toll-free number who can talk with doctors and know how to get the exact information needed to help with the process. They have an understanding of which trials or EAPs are open for enrollment, the location of study sites and the types of patients who are eligible to participate. They are also trained to share requests and coordinate decisions within Janssen. Each call is answered by a live operator and each case is evaluated individually. Our Medical Information staff members are professionals; however they cannot make specific recommendations about a patient's treatment.
Sometimes, loved ones and will reach out to multiple people at Janssen requesting access to an investigational medicine. This can actually delay the process. Any request received still needs to be handled by Medical Information staff directly.
The fastest way to make these requests is to have your doctor reach out directly to Medical Information at 1-800-Janssen or email us at email@example.com.