Ask Question
Sign Guest book

 
About Lymphoma
| Advocacy | Art | CAM & Life Style | Clinical trials | Docs & Centers | Guidelines at Diagnosis | How to Help | Side Effects | Support | Symptoms  | Tests | Treatments | Types of Lymphoma


Experts

Patients Against Lymphoma

 

ClinicalTrials.gov > Lymphoma type | Treatment type | Other | State | Country
About Trials | Expanded Access | Other Trial Resources | PDQ Services | Travel for Treatment

Clinical Trials > Expanded Access (Compassionate Use)

Last update: 06/07/2010

TOPICS
Filing an Application for Compassionate Use
| Commentary on Expanded Access 
Also see Canadian Access Program | FDA background on Expanded Access

 

Compassionate Use of Investigational Therapies
for trial- ineligible patients

Expanded access trials for Lymphoma

NEWS

Oct 2009: FDA Final rule for Expanded Access (PDF - 603KB)  AND 
Charging for Investigational Drugs (PDF - 394KB)

Jun 2010: ASCO: Oncologists, FDA Team Up to Ease Access to Novel Drugs

http://www.medpagetoday.com/MeetingCoverage/ASCO/20482

 

OVERVIEW 

  1. The requested treatment protocol must be considered reasonable by your oncologist and you must be ineligible or unable to participate in a clinical trial that is testing the protocol. 

    The protocol must be deemed reasonable for your treatment circumstance, relative to available treatment options.  

    * Potential for efficacy and sufficient data on safety. 

    * Preliminary evidence (clinical or plausible pharmacological of activity against the disease) 

    * Probable risk of the treatment does not exceed that from disease.

    If you are making the proposal, first do your homework, then write to your doctor and make the case, concisely.  

    NOTE: Safety issues are not always published and available to the patient or the doctor, leading to unrealistic expectations.  Efficacy can be overstated in early reports.)
     

  2. Your doctor must then contact the sponsor of the investigational agent, who must agree to provide the drug - they may not be able or willing. 
     
    This step can be a significant obstacle.  The drug sponsor has little to gain and possible liabilities (financial, insurance, legal) when providing investigational drugs outside the structure and close supervision of a clinical trial. 

    "When a physician would like to request an Investigational New Drug (IND) application to use an unapproved drug or other product for a single patient, the first step is to obtain permission from the manufacturer. Without the consent of the manufacturer, the unapproved product will not be available to the patient. After the manufacturer agrees to provide the product, the recommended procedure is to submit the following information to the appropriate review division. ... "   FDA.gov  

  3. Your doctor completes forms for FDA review & consent as described in the checklist below. The FDA will probably not stand in the way if the requested protocol is reasonable, relative to other available treatment options. 

    The rate limiting circumstance might depend on how your doctor communicates the urgency. 
     

  4. Finally, your doctor must obtain IRB (Institutional Review Board) approval and draw up a consent form. This step is easier if your doctor is also a clinical investigator and is familiar with the process.
     

  5. Your doctor will be responsible for monitoring, and reporting and oversight.  Cost to physician might not be compensated.  Doctor might have to assume some liability.

Compassionate Use Checklist

x
Manufacturer has committed to supplying the investigational drug
x
Letter of authorization from manufacturer included or submitted separately
x
Form FDA 1571 completed and signed (Investigational New Drug Application IND)  PDF 
x
Form FDA 1572 completed and signed (Statement of Investigator) PDF 
 
 for forms 1571 & 1572 (above)
x
CV (first two pages) including documentation of medical licensure Informed consent form completed and signed
x
Commitment to Institutional Review Board (IRB) approval prior to initiating administration of the investigational drug
x
Narrative: Medical history, disease history, prior treatments, response Patient's current medical status
x
Patient or physician's prior experience with the investigational treatment to be administered (if any), rationale for administration of the investigational drug. Proposal for treatment of the patient, including plan for concomitant treatments, administration of the investigational drug, and monitoring of the patient.
 

Expanded Access (EA) and Compassionate Use (CU) - Commentary

I feel that we have a moral responsibility in some instances (when the patient is willing and benefit is feasible) to try investigational agents when normal channels have failed or when standard approaches have not demonstrated benefit.

But, the patient and their loved ones should be informed if the Compassionate Use protocol is likely to increase pain and suffering, and also how likely it is to help. 

GAPS: We need to direct the NCI to fill in the many gaps of our economic-driven system, and conduct more clinical investigations, such as testing first line use of lower toxic immunotherapies as an alternative to observation (expectant management).  

- Karl S 

 
Disclaimer:  The information on Lymphomation.org is not intended to be a substitute for 
professional medical advice or to replace your relationship with a physician.
For all medical concerns,  you should always consult your doctor. 
Patients Against Lymphoma, Copyright © 2004,  All Rights Reserved.