At Johnson & Johnson, we are often asked how patients who have exhausted all other treatment options can gain pre-approval access to investigational medicines. These medicines are considered investigational—that is, they are still being studied and evaluated and are not yet approved by a government health authority like the U.S. Food and Drug Administration (FDA).
Johnson & Johnson has an established process that physicians may use to request investigational medicines on behalf of their patients around the globe. Physicians in the United States wishing to discuss submitting a request should call 1-800-JANSSEN or email: firstname.lastname@example.org. Physicians outside of the U.S. should contact the Janssen office in their country.
We recently connected with Beverly Harrison, Head, Patient Support, Janssen Pharmaceutical Companies of Johnson & Johnson, to discuss our pre-approval access approach.
What pathways exist for patients who have exhausted available treatments?
Johnson & Johnson offers three pathways to gain pre-approval access to our investigational medicines—clinical trials, expanded access programs and compassionate use.
1. The primary method for gaining access to Johnson & Johnson’s investigational medicines is to enroll in a clinical trial. Clinical trials allow a pharmaceutical company to develop the scientific data required to seek government health authority to approve its medicines. They are the preferred manner for patients to access investigational medicines.
Clinical trials are critical in bringing new medicines to patients because they support potential approvals by health authorities such as the FDA, allowing physicians to prescribe the medication to a broader population of patients.
For some seriously ill patients, however, clinical trials are not an option due to eligibility or logistical factors. For these patients, other options may be available.
2. Patients may be eligible to obtain access to an investigational medicine through expanded access programs (EAPs). Janssen Pharmaceutical Companies of Johnson & Johnson (Janssen) may open an EAP after having completed its clinical trials and before the FDA approves a new medicine. An EAP, however, is not opened for every investigational medicine.
It’s important to note that EAPs can vary by country and continent. Also, in the United States we use the term EAP, but they are referred to as a “named patient program” (NPP) throughout Europe or as a temporary authorization for use (ATU) in France.
3. Another option for patients who are not eligible for clinical trials or expanded access programs, and for whom no other alternative therapy exists, is “compassionate use.” Physicians, in collaboration with their patient, may determine that compassionate use, or a single or individual patient request, may be appropriate. Such requests may be submitted by the physician on a patient’s behalf to pharmaceutical companies for individual evaluation.
Does a patient submit the request for pre-approval access or does the physician?
Only treating physicians may submit a pre-approval access request on behalf of their patient, as they can provide the medical history and information required to evaluate the request. Physicians in the United States wishing to submit a request should call 1-800-JANSSEN or email: email@example.com. Physicians outside of the U.S. should contact the Janssen office in their country.
How soon will a patient hear whether he or she is granted pre-approval access?
We recognize the urgent nature of these requests and commit to providing a decision as quickly as possible, ideally within five to 10 business days of receiving all required medical information. Janssen notifies the treating physician, who then notifies the patient. Our PAA policy is posted on Janssen.com.
What guides Johnson & Johnson when evaluating pre-approval access requests?
Johnson & Johnson is a company dedicated to advancing patient care and public health. We abide by ethical principles as well as local laws and regulations when evaluating each request—whether to gain access to a clinical trial, an EAP or through compassionate use—to ensure:
- We are not putting patients at risk of unnecessary harm.
- We continue to conduct thorough scientific studies to understand the potential benefits and risks of new medicines to help bring new medicines to all patients who need them.
- We treat all patients fairly and equally.
Can you tell me more about Janssen’s collaboration with CompAC?
Janssen partners with NYU Langone Health to support our review of compassionate use requests made to Janssen according to pre-established criteria. The Compassionate Use Advisory Committee, or CompAC, selected and managed by NYU, provides a consistent, transparent mechanism for compassionate use decisions. Comprising bioethicists, physicians and patients, it provides an independent review to help support ethics-driven assessment of cases and bolsters our decision-making process at Janssen.
Where can I learn more about Johnson & Johnson clinical trials?
Johnson & Johnson sponsors and supports clinical trials in more than 40 countries and we rely on our clinical trial volunteers. Participants gain pre-approval access at the same time as helping others by contributing to medical research. To learn more about how to access a clinical trial, visit Janssen’s website, which provides a global clinical trial database searchable by location and condition. ClinicalTrials.gov also provides information on available clinical trials in the United States.
Is there more information available on Janssen EAPs?
Where can patients, caregivers and healthcare practitioners turn for more on pre-approval access?
Physicians seeking early access to Janssen’s investigational medicines can visit Janssen.com for more information or contact Janssen Medical Information at 1-800-JANSSEN. Physicians outside of the U.S. should contact the Janssen office in their country.