Compassionate Use Program

Compassionate Use Program

Providing access to care, serving overlooked populations and working to save lives is at the core of AVM Biotechnology’s mission. As a part of that mission, we are pleased to be able to offer our compassionate use program.

Compassionate use or expanded access programs are designed to make investigational medical products available as early as possible to patients without therapeutic options, because they have exhausted or are not a good candidate for approved therapies and cannot enter a clinical trial. Expanded access refers to the use of investigational drugs outside of a clinical trial, where the primary intent is treatment, rather than research

The purpose of our compassionate use program is to make AVM0703 available to those patients who have exhausted other treatment options and who do not meet the criteria to enroll in a clinical trial. It is intended to improve access to AVM0703 for patients with serious or immediately life-threatening diseases or conditions who lack other therapeutic options and may benefit from it.

It is important to remember that investigational medicinal products have not yet received regulatory approval; thus, their potential risks and benefits are not yet established. Physicians and patients should consider all possible benefits and risks when seeking access to an investigational drug.

Physician Instructions for Expanded Use Program aka Compassionate Use

Thank you for inquiring about an expanded use program otherwise known as compassionate use through AVM Biotechnology. We have put together some basic documents to help guide you and ensure compliance with the regulatory agencies. You will need an Expanded Access Single Patient IND. Our goal is to make this as easy and efficient as possible. Below we have outlined this process into 7 easy steps. Prior to starting the process, please keep the following in mind:

As the requesting physician you are the sponsor and principal investigator of expanded use and you must apply to the FDA for a single-use Investigational New Drug number (IND).

• Your request must meet the following criteria to qualify as described in 21 CFR 56.102(d)
  • the patient is in a life-threatening condition that needs immediate treatment;
  • no generally acceptable alternative for treating the patient is available; and
  • because of the immediate need to use the drug, there is no time to use existing procedures to get FDA approval for the use.
• If the above criteria are met, you can easily apply to AVM for access to our treatment by completing a Compassionate Use Application and download the Non-Disclosure and Confidentiality Agreement. Print and sign these documents. Please scan and email to: compassionateuse@avmbiotech.com or fax them to AVM at 206.424.8701. Once we receive the signed Non-Disclosure and Confidentiality Agreement, we will supply you with our Investigator’s Brochure and Pharmacy Manual.

The rest of the application process is fairly simple and we are here to help facilitate this application. The process is split into the following steps.

1. Contact the FDA for a single patient compassionate use Investigational New Drug (IND) number

AVM0703 is under the Division of Hematological Malignancies II (HMD2). E-mail: ONCProjectFacilitate@fda.hhs.gov The general phone number is ph: 301-796-7550 or fax: 301-796-9845. Address: Food and Drug Administration, Center for Drug Evaluation and Research, Division of Hematologic Malignancies 2 (DHM2), 5901-B Ammendale Road Beltsville, MD 20705-1266.

If for some reason, you can not get through, here is some general contact information for the FDA: 301-796-3400 or 855-543-3784; fax: 301-431-6353; or e-mail: druginfo@fda.hhs.gov. For questions about expanded access for emergency use for investigational biologics, contact CBER’s Office of Communication, Outreach, and Development at phone: 240-402-7800 or 800-835-4709; or e-mail: industry.biologics@fda.gov. For questions about non-emergency individual expanded access for biologics, contact CBER at: 240-402-8010 or 800-835-4709. More information can be found here.

2. Complete FDA form 3926

This will normally only take 45 minutes and we can help with any details. We have a few documents to help you understand the mechanism of action and rationale for treatment. You can see a sample outline to the Brief Clinical History question. A sample protocol can be provided upon request. You should also submit the first two pages of your CV with this form.

3. Obtain an Informed Consent from the patient or patient advocate

A generic form of an informed consent can be found at INFORMED CONSENT TEMPLATE. This is to ensure that both you and your patient understand the risks and potential benefits. Keep a copy of the consent for the IRB submission.

4. Set up an Institutional Review Board meeting

You can request emergent use from the FDA by checking the box “Alternative IRB Review” on form 3926, however, you must still have an expedited review by the IRB chairperson as soon as possible. If you do not have access to an Institutional Review Board we can expedite your application through a third-party IRB. Please note this on the application form. The emergency use provision is an exemption from prospective review and approval by the IRB. The exemption, which may not be used unless all of the conditions described in 21 CFR 56.102(d) exist, allows for one emergency use of a test article. These are typically situations in which the intent is not to conduct research but to act in the best interest of an individual patient. If you have a true emergency request, we can work with you to ensure your patient receives this treatment very quickly.

5. Request a Letter of Authorization (LOA) from AVM Biotechnology

We can normally send this within 24 hrs. You will need to include this with your FDA 3926 form.

6. Send the following to the FDA

FDA Form 3926, Letter of Authorization, and any additional documentation requested by the FDA. These documents can usually be provided by fax or email.

7. Send all information to AVM Biotechnology and set up a date and time for the treatment

We will need the FDA approval letter, IRB approval with approved Informed Consent, and confirmed shipping information. Please allow up to 2 business days from the time of approved IND to the delivery of the drug to your institution or practice. We will provide you and/or your staff with a tracking number to ensure the drug is delivered prior to the scheduled treatment date. The date and place of the treatment as well as information on the key contact people involved in the treatment should be provided in the initial application.

As a physician sponsor and investigator, you are required to report any adverse events to AVM Biotechnology immediately and to the FDA as well as using standard acceptable forms of reporting as required by the FDA (Use FDA Form 3500A together with Form 1571). In addition, at the conclusion of the treatment, you are required to report the outcome of the treatment to the FDA and your IRB. If the treatment lasts over 12 months, you must submit an annual report.

We have found an excellent resource to help you and your colleagues navigate the compassionate use for your patients and their family members. Kids vs. Cancer publishes the Compassionate Use Navigator which is an excellent resource if you ever have questions.

Our goal is to help provide compassionate use of our product to your patient in the safest, most efficient manner and with the least amount of stress upon an already stressful situation. The cost of this medication will thus be provided free of charge for the patient you are sponsoring. We can also provide support to properly understand the dosing, potential side effects and the potential methods to prevent and or treat the side effects. If you have any questions or concerns about the treatment AVM Biotechnology has provided, please contact us any time of the day or night. We are always available to answer any questions.

Patient / Family Instructions for Expanded Access Program aka Compassionate Use

Thank you for inquiring about an Expanded Access Program otherwise known as compassionate use through AVM Biotechnology. The process starts with finding a physician sponsor either currently treating or willing to treat your or your loved one’s condition with AVM0703.

AVM0703’s mechanism of action (MOA) is understood to be the stimulation and release of supercharged highly efficient natural killer T cells (NKT) and cytotoxic T cells  which provide an organized enhancement of the immune response. There are 4 steps to this process:

Step 1

Ask your physician to read the Physician Instructions for Compassionate Use.

Step 2

Encourage your sponsoring physician to complete the FDA form 3926 and schedule a review by the Institutional Review Board (IRB) of your treating facility.

Step 3

Ask your sponsoring physician to complete the Compassionate Use Application.

Step 4

Ask your sponsoring physician to contact AVM Biotechnology for a Letter of Authorization.

Kids vs. Cancer is a wonderful non-profit organization with very useful information to help you through this process at Compassionate Use Navigator.

Our goal is to help provide compassionate use of our product in the safest, most efficient manner and with the least amount of stress upon an already stressful situation. The cost of this medication is provided free of charge. We can also provide support to properly understand the dosing, potential side effects and the potential methods to prevent and or treat the side effects. If you have any questions or concerns about the treatment AVM Biotechnology has provided, please contact us anytime of the day or night. We are always available to answer any questions.

Expanded Access Policy

AVM Biotechnology development resources are focused on conducting clinical studies required by regulatory authorities to fully answer important scientific questions about the potential risks and benefits of our investigational products, and to obtain regulatory approval.

AVM Biotechnology is committed to making investigational products available to seriously ill patients who have exhausted other treatment options. A treating physician, who is able to comply with the requirements that are stated in this document, may request information about how to apply for access to AVM Biotechnology’s investigational products by contacting the Company.

The purpose of this policy is to describe the requirements for Expanded Access to AVM Biotechnology investigational products to patients outside of a clinical study.

Scope

This policy applies to the provision of access to an AVM Biotechnology investigational product that is not approved for any purpose in the country from which the request is intended to be used. This also includes the time period between regulatory approval of an investigational product and its commercial availability in a country.

Policy Statements

Any use of an AVM Biotechnology investigational product outside a clinical study in a country must be in accordance with local laws and regulations governing such programs, including AVM Biotechnology policies and procedures.

In general, where permitted by local regulation, the investigational product supplied via Expanded Access may no longer be provided by AVM Biotechnology when it becomes available via the local healthcare system. AVM Biotechnology may decide not to provide an investigational product under this policy if the Company does not intend to market the product in the country.

A. Patient Eligibility Criteria

To be eligible for access to an investigational product, patients must meet the following criteria:

• Suffer from a serious or immediately life-threatening disease or condition.
• Have undergone appropriate standard treatments without success and no comparable or satisfactory alternative treatment is available or exists to treat the disease or condition.
• Are ineligible for participation in any ongoing clinical study of the investigational product, which includes lack of access due to geographic limitations.
• The patient has a disease for which there is sufficient evidence of a projected benefit from the use of the investigational product and the benefit outweighs the known or anticipated risks.
• There is adequate information to support appropriate dosing for a special population of patients such as pediatric, elderly, renal or hepatic disease, etc.
• Any other pertinent medical criteria for access to the investigational product, as established by the AVM Biotechnology clinically or medically responsible individual.

B. Investigational Product Criteria

In addition to the patient eligibility requirements, the investigational product must meet the following criteria:

• The product is under investigation in one or more clinical studies.
• There is sufficient evidence to expect that the investigational product will have an acceptable safety profile for the intended patient population.
• The provision of the investigational product will not interfere with or compromise the clinical development of the product.

C. Treating Physician Criteria and Responsibilities

The physician(s) attending to the patient(s) who is/are receiving an investigational product through compassionate use access is (are) properly licensed and fully qualified to administer the product. The physician must agree in writing to comply with:

• Any applicable country-specific legal and regulatory requirements related to providing an investigational product under Expanded Access.
• Any AVM Biotechnology requirements in terms of medical criteria, reporting of patient outcomes, safety reporting, drug supply/use and protection of intellectual property. A treating physician may submit questions or requests regarding expanded access to AVM by calling 206-906-9922 or by e-mailing compassionateuse@avmbiotech.com.