Access to Investigational Therapies
Access to Investigational Therapies
Cerecor’s goal is to provide potentially life-saving therapies to patients who may benefit from them, while minimizing potential risk. Cerecor believes that clinical trials are the most effective way for patients to access investigational products, as clinical trials can generate data that may lead to the approval of new medicines and wider availability to patients. However, in certain situations, Cerecor will assess requests for access to unapproved products, via a Compassionate Use program.
Compassionate Use (or Expanded Access) is a process that may help qualified patients obtain investigational therapies that are not yet approved by the country’s regulatory agencies (e.g., the Food and Drug Administration (FDA) outside of a clinical study where there is a compelling need.
Clinical Study Information
To participate in a clinical study, you/your child must meet certain eligibility criteria. These criteria identify similar disease characteristics which can allow researchers to better evaluate the investigational therapy. You should discuss participation in a clinical study with your physician who knows your medical history and current status and can help to determine whether you may be able to join a clinical study.
For information on ongoing Cerecor clinical studies:
- Visit ClinicalTrials.gov and search registered clinical studies for the disease. To search by company, Cerecor, click here.
- If you locate a clinical study with a participating site that you are interested in, you or your physician may contact that site directly for more information.
- For a list of ongoing Cerecor clinical studies contact the Cerecor Patient Advocacy group at PatientAdvocacy@cerecor.com.
At Cerecor, we recognize that some patients with serious or immediately life-threatening diseases may not be able or eligible to participate in clinical studies and may not have other treatment options for their condition. Cerecor carefully evaluates requests for individual patients to receive investigational therapies outside of a clinical study on a case-by-case basis.
Cerecor recognizes that patients with serious or life-threatening disorders may seek medical products that are not yet approved or available in their country. The company has established the following compassionate use policy to guide patients, their families and treating physician who seek to be considered for such access.
Cerecor (the "Company") will consider requests from licensed treating physicians for access to investigational products in development for patient treatment purposes on a case-by-case basis. Treating physicians interested in potential access to investigational products may request more information by contacting Cerecor via email at ExpandedAccess@cerecor.com.
Receipt of a request for information will be acknowledged within 10 business days.
Some of the factors that will be considered upon receiving a request include:
- Whether the patient has a serious or life-threatening illness
- Whether the patient has undergone standard treatment without success or there is no viable alternative therapy
- Whether the potential benefits of the experimental product will outweigh any potential risk to the patient
- Whether the patient is ineligible for or is unable to participate in a clinical trial for the investigational product
- The impact of providing access to an investigational product on the development program for that product
- The impact of providing access to an investigational product on the available inventory and supply for that product
Information regarding our ongoing clinical trials may be found at www.clinicaltrials.gov.
The Company’s decision on whether or not to grant access to the investigational product under a Compassionate Use program should be communicated to the requestor within 15 business days of receipt.
Application under this policy is not a guarantee of access to any Company investigational drug. Cerecor reserves the right to revise or revoke this policy at any time.
Additionally, Cerecor reserves the right to deny any request at the discretion of the company.
Decisions for early access will be made on a fair and equitable basis for each investigational therapy. Each disease we are studying may have different specific criteria due to the differences in urgency and known data on safety and efficacy.
For Patients/ Caregivers/ Patient Advocates
Requests for early access to investigational therapies must be made by a qualified physician. You and your family should discuss your situation with your treating physician including current status, available treatment options and reason for considering treatment with an investigational therapy. Your physician should make the request for early access on your behalf. It is important that early access requests are made by medically trained personnel in order to avoid delays.
For more information on Early Access programs you may visit the United States Food and Drug Administration website.
For Physicians / Healthcare Professionals
All requests for early access for a single patient or patients should be submitted by the patient’s treating physician.
The patient’s treating physician must provide adequate information to demonstrate that the patient meets the outlined considerations for early access.
The patient’s treating physician must be appropriately licensed, fully qualified to administer the investigational therapy and have the expertise and facilities for monitoring, managing and reporting any adverse events during the course of treatment. The patient’s treating physician must be willing to meet and follow all applicable legal and regulatory requirements for early access while caring for the patient receiving investigational therapy and must comply with all applicable reporting requirements.
Qualified physicians can submit a request by contacting Cerecor at CompassionateUse@cerecor.com.
For more information on Early Access for Patients and Physicians you may visit the United States Food and Drug Administration website.
Cerecor will review the request and supporting documents from a treating physician to determine the urgency and need of the request. We are committed to acknowledging receipt of a request within 10 business days. We aim to respond to the request including our rationale as promptly as possible so that you and your family can explore all possibilities.
When evaluating the request, we will consider the best option:
- First determine if an appropriate Cerecor sponsored clinical study or an Investigator-initiated clinical study is regionally available.
- If no study is available, then we will consider early access outside of a clinical study:
- If the disease has been treated with the investigational therapy and shown to be safe and effective, we will respond promptly and provide guidance to the physician to ensure all applicable regulatory and legal requirements are met.
- If the disease or condition is one we have not treated or are not studying, our ability to help may be limited. In general, prior treatment of the disease with the investigational therapy is needed for Cerecor to consider early access.
As a company, Cerecor is committed to supporting expanded use, when possible.