The Ewing's Sarcoma and Pediatric Cancer
 Support Group--"How to get Drugs not FDA Approved Yet" from The Cure Our Children Foundation

Lainie, Alon, Lilah and Barry Sugarman Alon Sugarman During Chemotherapy


In Honor of Alon Sugarman
14 Years Old, Alive and Well

This site maintained by Barry Sugarman, B.S.ENGR.
Father of Alon Sugarman, Diagnosed March 6, 1998
with Ewing's Sarcoma of the Distal Femur.

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** This site and our organization support the traditional medical approaches to treatment of Ewing's Sarcoma because they are proven effective in numerous long term clinical studies.  Alternative and Complimentary treatment information is provided only for use in conjunction with traditional approaches. **

** This guide has been written for educational purposes only, it cannot be used for
diagnosing or treating a health problem or a disease. If you have or suspect that you or your child may have a health problem, you should consult your doctor. **

Brief Introductory Article and Glossary by Barry Sugarman, B.S.ENGR., President, The Cure Our Children Foundation

The purpose of this page is to let you know more options on how to obtain prescription drugs that may not be approved in the United States yet, or that may be approved for indications other than the disease that is being treated.  First, let's look at some common abbreviations that are used in the pharmaceutical development field:

Clinical Trial: Research studies on human subjects that seek to answer specific questions of safety and efficacy of a particular drug, device, or treatment regimen.
FDA: Food and Drug Administration, the agency which regulates drugs in the United States.
IND: Investigational New Drug Application. What a drug maker needs to file with the FDA to begin the drug approval process and start clinical studies.
CFR: Code of Federal Regulations, the regulations that FDA uses to govern the manufacture and distribution of drugs.
FR: Federal Register, the periodical that the government publishes updated regulations in.  Published daily.
Protocol: A set of criteria rules established under which a Clinical Trial (drug study) is accomplished.
IRB: Institutional Review Board, a group of independent doctors and professionals that reviews and approves/disapproves IND protocols. Most hospitals and universities have their own IRB's.
SOP: Standard Operating Procedure, a set of written instructions on how to perform tasks involved in drug development and clinical trials.

In order to be able to obtain and use a drug in the United States that is not yet approved by the FDA, an IND must be filed with the FDA and approved by the FDA and the local IRB.  IND's may be filed by a drug company or by an individual doctor, and they may be for individual patients, multiple patients, or for a large number of patients.  Below, I describe the numerous types of IND processes.

For many drug and biologic products, the pharmaceutical companies that are developing the product, or the Universities, National Cancer Institute, Phone: 800-422-6237, or Children's Oncology Group, Phone 800-458-6223 or 626-447-1674 have already filed an IND and have received IRB and IND approval for their clinical trials. Contact information for those clinical trials may be obtained by utilizing the clinical trials search resources on our foundation home page at .  For products that you wish have your doctor obtain that are not currently the subject of an approved clinical trial, please read on for more information and procedures to follow.

It should be noted that drugs that are approved for any indication in the United States may be prescribed by a physician for any use, even if that use is not approved specifically by the FDA.  This is called off-label use, and it is legal and very common.  For instances, if your physician decides that he wants to use oxaliplatin for Ewing's Sarcoma, in combination with other drugs, he or she may do so even though oxaliplatin is only approved by FDA for treatment of other types of cancers.  There is even a specific FDA regulation which exempts off-label use of approved drugs.  It is 21 CFR 312.2(d).  FDA has also issued a guidance document to clarify its policy that broadens the exemptions from an IND.  That guidance document is here:  IND Exemptions for Studies of Lawfully Marketed Drug or Biological Products for the Treatment of Cancer  You may click on this link to print out and provide your healthcare provider these FDA regulations and guidances.

Many doctors are familiar with the IND process, but some are not.  For drugs that are not yet approved, it will be essential to have the cooperation of the manufacturer both to provide the supply of the drug manufactured according to pharmaceutical standards, and to support the IND filing with FDA with the data on the drug.  Below, I describe the procedures and policies of how to file IND's to get drugs, and I provide numerous resources to assist doctors, patients, and pharmaceutical development companies to make it easier for cancer patients to obtain new therapies.

All IRB's have procedures for applying for and obtaining approval to use an investigation product; however, if you are being treated by a private doctor, he or she can contact a local university and apply to their IRB for approval, or I have listed an IRB that accepts applications from private doctors below.  Also, most IRB's have a procedure in place already to apply for and receive approval for emergency use of a drug; however, for those IRB's and facilities that do not have such a procedure, I have provided links and information in after the Emergency IND paragraph below to assist.

There is also a foundation that is active in trying to expand investigational drugs for cancer patients.  It is the Abagail Alliance, Phone: 703-525-9266 , Fax: 703-243-2892.  The website is  .

                                                                         Warm Regards.....Barry Sugarman

There are numerous ways to obtain drugs that are being investigated for FDA approval.  These are:

An Excellent Writeup from the National Institute of Health on the Clinical Trials System is here.

The Main FDA Investigational New Drug Application (IND) Page is here.

The Treatment Referral Center (TRC) is a means for the National Cancer Institute (NCI) to provide information to community oncologists and other health care professionals about therapeutic options for cancer patients.  Phone (301)496-5725, Fax (301)402-4870. 


FDA Regulations:

Investigational New Drug Applications (IND's):

IND Regulations Table of Contents (21 CFR 312)

Text File     PDF File     21 CFR 312.34  Treatment use of an investigational new drug.
Text File     PDF File     21 CFR 312.35  Submission for treatment use.
Text File     PDF File     21 CFR 312.36  Emergency use of an investigational new drug.

Institutional Review Board Regulations (IRB's):

IRB Regulations Table of Contents (21 CFR 56)
Text File     PDF File     21 CFR 56.102  Definitions 
Text File
     PDF File     21 CFR 56.104  Exemptions from IRB requirement.

Protection of Human Subjects Regulations (21 CFR 50):

Protection of Humans Subjects Regulations Table of Contents

Text File     PDF File     21 CFR 50.23    Exception from general requirements. 
Text File     PDF File     21 CFR 50.24    Exception from informed consent requirements for emergency research. 

This site last updated on July 3, 2005