To be eligible for access to an investigational drug outside of a clinical study, a patient must meet the following criteria: Suffers from a serious or immediately life-threatening disease or condition.
Who can participate in a clinical trial?
Clinical trials have strict inclusion and exclusion criteria about who can participate. The criteria can be based on such factors as: Having a certain type or stage of disease. Having received (or not received) a certain kind of therapy in the past. Being in a certain age group.
Who can conduct clinical studies on drugs?
Such studies are typically undertaken by a pharmaceutical or device manufacturing company or other commercial entity. Analogously, there must be a compliance with the requirements that the study does not amount to a commercial distribution or marketing of a new drug.
Are Group C Guidelines part of a clinical trial?
Although treatment is the primary objective and patients treated under Group C guidelines are not part of a clinical trial, safety and effectiveness data are collected. Because administration of Group C drugs is not done with research intent, FDA has generally granted a waiver from the IRB review requirements [21 CFR 56.105].
Do I need an IND for my clinical trial?
Clinical trials that use an FDA-approved drug within the approved labeling do not need an IND. However, clinical investigations initiated by sponsor-investigators frequently make use of FDA-approved drugs in populations or indications not addressed in the approved labeling.
What are the requirements for investigational new drug approval?
The IND application must contain information in three broad areas: Animal Pharmacology and Toxicology Studies - Preclinical data to permit an assessment as to whether the product is reasonably safe for initial testing in humans. Also included are any previous experience with the drug in humans (often foreign use).
What basic criteria must be met by the pharmaceutical company in order for the FDA to approve drug testing in people?
A pharmaceutical company seeking FDA approval to sell a new prescription drug must complete a five-step process: discovery/concept, preclinical research, clinical research, FDA review and FDA post-market safety monitoring.
What is the FDA process for a critically ill patient to obtain investigated drugs outside of the typical clinical trial method?
Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable ...
Should new drugs be given to patients outside of clinical trials?
Ideally, patients who wish to try an experimental drug should do so in a clinical trial. In a trial, outcomes, including adverse events, are monitored, problems identified, and, should the need arise, treatment provided to address side effects or other adverse events.
What are the requirements for FDA approval?
To get FDA approval, drug manufacturers must conduct lab, animal, and human clinical testing and submit their data to FDA. FDA will then review the data and may approve the drug if the agency determines that the benefits of the drug outweigh the risks for the intended use.
What are the 3 phases an investigational drug goes through prior to submitting a new drug application?
1- Preclinical (animal) testing. 2-An investigational new drug application (IND) outlines what the sponsor of a new drug proposes for human testing in clinical trials. 3-Phase 1 studies (typically involve 20 to 80 people). 4-Phase 2 studies (typically involve a few dozen to about 300 people).
Why an IND is required prior to conducting a clinical trial?
The stated purpose of an IND is “to ensure that subjects will not face undue risk of harm” in a clinical investigation that involves the use of a drug. Hence, to authorize a drug study in humans, the FDA requires sufficient information to assess the safety of the intended research study.
What is right to access for investigational products?
The Right to Try Act permits/allows eligible patients to have access to eligible investigational drugs. An eligible patient is a patient who has: Been diagnosed with a life-threatening disease or condition.
What are the 4 phases of FDA approval?
Information ForStep 1: Discovery and Development.Step 2: Preclinical Research.Step 3: Clinical Research.Step 4: FDA Drug Review.Step 5: FDA Post-Market Drug Safety Monitoring.
What can be done to allow therapeutic use under a physician's care of unapproved drug by patients who desperately need the drug even if it is for experimental use?
Because the drug was not yet approved by the FDA, it was not available in pharmacies. However, the FDA may permit the use of an unapproved drug in certain circumstances—a process referred to as compassionate use. For FDA to grant that permission, however, the manufacturer must have agreed to provide the drug.
What program might impact a patient's access to a clinical trial before a drug or biologic is approved?
Expanded Access Program (EAP)An Expanded Access Program (EAP) allows physicians and patients access to pre-approval, investigational drugs outside of the clinical trial setting.
What makes a medical treatment experimental?
Experimental medicine, in contrast to therapeutic medicine, is defined as the study of treatments currently undergoing clinical trials or other forms of testing to understand their effectiveness and safety.
What is the FDA regulation for dispense of investigational drugs?
FDA regulations at §312.310 specify the two criteria under which a licensed physician may administer or dispense an investigational drug for treatment use, including emergency use, for an individual patient.
Who is considered an investigator?
A licensed physician who administers an investigational drug for an expanded access use is considered an investigator (i.e., physician-investigator) and must comply with the responsibilities for investigators as itemized below.
What is the purpose of FDA subpart I?
Subpart I of FDA regulations describes the requirements for the use of investigational and approved drugs where availability is limited by a risk evaluation and mitigation strategy when the primary purpose of the use is to diagnose, monitor, or treat a patient's disease or condition (i.e., expanded access uses ). By authorizing such expanded access uses, FDA aims to facilitate the availability of such drugs to patients with serious or life-threatening diseases or conditions when there is no comparable or satisfactory alternative therapy.
What is FDA 115?
115. FDA Regulations for Treatment or Emergency Uses of Investigational Drugs. The Food and Drug Administration's Expanded Access Program allows for drugs that are investigational (i.e., not FDA approved) to be used for treatment use outside of a clinical trial. Sometimes this is referred to as compassionate use.
What is FDA regulation 312.83?
In addition to provisions for expanded access use for treatment of an individual patient and intermediate-sized populations, FDA regulations at §312.83 and §312.320 allow for wide-spread treatment use of an investigational drug via a treatment protocol or Treatment IND.
What is the FDA's definition of "subpart"?
FDA allows flexibility in the expanded use of investigational drugs for treatment for persons with serious or life-threatening diseases or conditions (Subpart I) and for persons with life-threatening and severely debilitating illnesses (Subpart E).
What do investigator sponsors do?
Investigator-sponsors must: ensure physician-investigators are qualified to administer the investigational drug for the expanded access use; provide the physician-investigator with the information needed to minimize risk and maximize the potential benefits of the investigational drug (e.g., Investigator's Brochure);
Why is it important to have investigational interventions outside of clinical trials?
It has the potential to seriously harm individuals and raises important issues of fairness. For these reasons, the National Breast Cancer Coalition (NBCC) believes that access to investigational interventions outside of clinical trials should be allowed only in very limited circumstances.
What is evidence based medicine?
1 Evidence-Based Medicine has been defined as “the conscientious, explicit and judicious use of current best evidence in making clinical decisions about the care of individual patients.” Sackett D et al. Evidence-Based Medicine: What it is and what it isn’t. British Medical Journal 1996;312:71-2.
Is NBCC a public policy?
NBCC believes that public policy should discourage access to investigational interventions outside of clinical trials. But there are a few circumstances in which it would be fair and appropriate to implement an expanded access protocol.
What are the criteria for clinical trials?
Clinical trials have strict inclusion and exclusion criteria about who can participate. The criteria can be based on such factors as: Having a certain type or stage of disease. Having received (or not received) a certain kind of therapy in the past. Being in a certain age group.
What is an experimental drug?
An investigational drug can also be called an experimental drug and is being studied to see if your disease or medical condition improves while taking it. Scientists are trying to prove in clinical trials: If the drug is safe and effective. How the drug might be used in that disease. How much of the drug is needed.
Can you get an investigational drug through a clinical trial?
Before you can be given an investigational drug either through a clinical trial or through expanded access, your healthcare provider must give you additional information about the potential risks and potential benefits of the drug. As promising as an investigational drug may sound.
Is an investigational drug still being tested?
As promising as an investigational drug may sound. It is still being tested in clinical trials to determine if it can be used to treat a disease or medical condition. And not everyone who wants to enroll in a clinical trial will be able to participate. Clinical trials have strict inclusion and exclusion criteria about who can participate.
What is the time gap between clinical trial and market availability of a drug?
There is a time gap between clinical trial and market availability of a drug. To ensure patients have access to experimental, not-yet-authorised or marketed treatments supported by scientific proof of efficacy and safety, frameworks such as medical need programs and compassionate use programs are in place.
When is an approved drug considered off label?
It is justified if there is scientific evidence that the approved drug is safe and beneficial to patients suffering from an illness or disease that the drug is not approved for, or if no other satisfactory treatment is available for a patient.
Do private clinics have GMP?
Some private clinics and physicians provide off-label and investigational drugs outside of a clinical trial that are not supported by scientific evidence and/or not compliant with GMP regulation. There is no guarantee that patient rights, safety and well-being are respected by these treatments.
Is it safe to use experimental products?
The use of experimental products that are not yet approved or licensed is justified if there is scientific evidence that it is safe and likely beneficial to patients , if its production complies with the Good Manufacturing Practice guidelines, or if no other satisfactory treatment is available for a patient.
Can you use unauthorised medicines?
Unauthorised medicinal products are sometimes authorised to use out of compassionate reasons, upon request of individual pharmaceutical companies. Patients with a debilitating or life-threatening disease that cannot be treated by an authorised product can use an unauthorised medicinal product, as long as the product has applied for marketing authorisation or is currently undergoing clinical trials for the indication.
When to use investigational medicine?
Investigational medicines are used when you are seriously ill but not enrolled in a clinical trial. These medicines are being studied but have not yet been approved as safe and effective by the FDA. It is sometimes called compassionate medicine use. Normally, investigational medicines have strict testing through many stages ...
How to contact NCI for cancer?
Call NCI's Cancer Information Service at 800-4-CANCER (800-422-6237) or visit www.clinicaltrials.gov to search for clinical trials for your condition. If you don't qualify for a clinical trial, your healthcare provider can ask the trial's sponsor about requesting a single-patient exception.
What is expanded access medicine?
Expanded access uses medicines that have been well-tested and are nearing FDA approval. The medicine must show in studies that it is effective in treating a specific cancer. The program is offered by a medicine company and the medicine is given out to a group of people.
How long does it take for a drug to be tested?
They are also tested to see how well they work and at what doses, and with what conditions or diseases they are to be used. The process may take many years. In certain cases, these medicines can be used outside of a clinical trial. Investigational medicine use is legal.
Does everyone qualify for a clinical trial?
The FDA closely regulates clinical trials, and people taking new medicines are carefully monitored. Not everyone will qualify for a clinical trial. Because the use of new, unapproved medicines is risky, only certain people who are not enrolled in clinical trials can get investigational medicines for compassionate use.
Who must correct any problems that occur during the study?
Investigators must also correct any problems that occur during the study or terminate the study and notify their IRB, the FDA, and other investigators . CONCLUSIONS. Meeting the regulatory requirements for conducting drug studies is an essential part of doing clinical research.
What is the primary set of federal laws establishing FDA authority as well as codification of the regulations?
The primary set of federal laws establishing FDA authority as well as codification of the regulations is the Federal Food, Drug, and Cosmetic Act. The specific section of these laws covering an IND is in Part 312 of the Code of Federal Regulations (CFR).
What is an investigator IND?
An “investigator IND” is a research IND submitted by an investigator who initiates and conducts the study including the immediate supervision of the use of the study drug. This would typify the studies conducted by sponsor-investigators.
What is an IND in medicine?
Studies using a drug that has not been approved by the Food and Drug Administration (FDA) or for indications not in the approved labeling may require filing an Investigational New Drug (IND) application with the FDA. If a study meets specific regulatory exemption criteria, then an IND may not be needed.
How long does it take for an IND to be approved?
If the IND is approved, the study may begin 30 days after the FDA acknowledges receipt and assigns an IND. If the FDA requires additional information or if the study is placed on a “clinical hold,” the study must not proceed.
Do you need an IND for a clinical trial?
Clinical trials that use an FDA-approved drug within the approved labeling do not need an IND. However, clinical investigations initiated by sponsor-investigators frequently make use of FDA-approved drugs in populations or indications not addressed in the approved labeling.
Do drug investigators have to comply with FDA regulations?
Therefore, to conduct drug studies, an investigator must comply with FDA requirements. Failing to meet the FDA’s regulations can have legal and financial implications for the individuals conducting the research as well as the institutions associated with the research activities.
Who can stand in for a patient who cannot give consent?
The options must be clearly presented and for those who cannot give consent the parents, guardians or relatives should be able to stand in for the patient(s). This should not be jettisoned even in the face of an emergency. The export country concerned should also consent to the use of the drug in the receiving state.
What is the FDA's focus?
The patient, however, is the central focus and such uses are without control groups .25The US Food and Drug Administration (FDA), which regulates drugs administration and evaluates clinical trials sponsored by US agencies, also allows the use of untested drugs on compassionate ground.
What is monitored compassionate use?
Monitored compassionate use has the goal of saving human life, reducing the suffering of the patients and promoting human dignity in health care systems. To deny access on such ground would amount to a grave injustice.
What is the categorical imperative?
The commands of morality must be categorical imperatives, by which he means a moral law that must apply to all rational beings, regardless of their wants and feelings.11His formulation of the categorical imperative is to establish that a morally good action is based duty and not on consequentialist considerations.
Why is preventive health important in Africa?
Africans need to be pro-active and more health conscious just as the failing states in Africa should see as urgent, overhauling of their existing health care systems.
Do you need to submit an application to the National, State, or Institutional Health Research Ethics Committee for prior review and
In its advisory capacity, the Committee advised the government that researchers may not need to submit an application to National, State or Institutional Health Research Ethics Committee for prior review and approval. There is a strong call for proper documentation of the efficacy and protocols of such trials.33.
Expanded Access (Treatment) Uses of Investigational Drugs
Investigator and Sponsor Responsibilities For Expanded Access Uses
- Investigators’ and sponsors' responsibilities (Subpart D of FDA regulations) apply as follows to the extent the requirements are relevant to the expanded access use: 1. A licensed physician who administers an investigational drug for an expanded access use is considered an investigator (i.e., physician-investigator) and must comply with the responsibilities for investigators as itemized b…
Types of Expanded Access Uses of Investigational Drugs
- Single Patient Treatment Use of an Investigational Drug by a Licensed Physician
FDA regulations at §312.310 specify the two criteria under which a licensed physician may administer or dispense an investigational drug for treatment use, including emergency use, for an individual patient. 1. A licensed physician determined the probable risk to the person from the in… - Treatment Use of an Investigational Drug by a Licensed Physician in an Intermediate-sized Popu…
Under specific conditions (see §312.315), FDA allows use of an investigational drug for the treatment of more than one patient when the population is smaller than that typical of a treatment IND or treatment protocol (as described below). For example, when a significant number of requ…
Requirements For FDA Approval of A Treatment Ind
- Trial status.The drug must be under investigation in a controlled clinical trial under an IND designed to support a marketing application for the expanded access use or all clinical trials of the d...
- Marketing status.The sponsor of the IND supporting marketing must be actively pursuing marketing approval of the drug for the expanded access use with due diligence.
- Trial status.The drug must be under investigation in a controlled clinical trial under an IND designed to support a marketing application for the expanded access use or all clinical trials of the d...
- Marketing status.The sponsor of the IND supporting marketing must be actively pursuing marketing approval of the drug for the expanded access use with due diligence.
- Evidence. Sufficient clinical evidence must be available to provide a reasonable basis for concluding the investigational drug may be effective for the expanded access use without exposing patients...
Additional Resources
- How to submit an expanded access request (form)provides step-by-step instructions for expanded access submissions for investigational drugs and biologics
- Form FDA 3926 and instructions for requests for individual patient expanded access to investigational drugs and biologics, including those for emergency use. It can also be used for certain submiss...
- How to submit an expanded access request (form)provides step-by-step instructions for expanded access submissions for investigational drugs and biologics
- Form FDA 3926 and instructions for requests for individual patient expanded access to investigational drugs and biologics, including those for emergency use. It can also be used for certain submiss...
- Quick guide on how to complete Form FDA 1571 and 1572.
- Forms FDA 1571 and 1572are required for other expanded access submissions (e.g., intermediate access or treatment INDs) and for IND submissions by industry.
Guidance Documents
- FDA Final Guidance: Expanded Access to Investigational Drugs for Treatment Use — Questions & Answers
- FDA Final Guidance: Individual Patient Expanded Access Applications: Form FDA 3926FDA Final Guidance: Charging for Investigational Drugs Under an IND — Questions & Answers