When a patient does not respond to current approved treatments for a variety of reasons, other options still may be available. For serious and life-threatening illnesses, some patients may want to talk to their healthcare provider about other options of trying an investigational drug through “Expanded Access” or “Right to Try” applications.
1.Understanding Investigational Drugs
2.Understanding “Expanded Access” Information
3.Understanding “Right to Try “ Information
4.“Right to Try” FAQs
1. Understanding Investigational Drugs
An investigational drug, also referred to as an experimental drug, is a drug that is still being studied and has not yet been approved by regulatory agencies, so drug manufacturing companies are required to evaluate their safety and effectiveness in clinical trials and submit trial results to regulatory agencies. To participate in a trial, patients must meet certain criteria. It may be helpful for you to understand what an investigational drug is before deciding if one might be right for you.
(See FDA page: Understanding Investigational Drugs)
2. Understanding “Expanded Access”
Information about the FDA’s current expanded access
policies, requirements for enrolling in expanded access programs, and steps you
can take to get more information:
Expanded Access is a potential pathway for a patient with an immediately life-threatening 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 or satisfactory alternative therapy options are available.
(See FDA page: Expanded Access)
3. Understanding “Right to Try”
The Right to Try Act, or the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act, was signed into law on May 30, 2018. This law is another way for patients who have been diagnosed with life-threatening diseases or conditions who have tried all approved treatment options and who are unable to participate in a clinical trial to access certain unapproved treatments.
“Right to Try”
Right to Try The Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act, or Right to Try Act, were signed into law on May 30, 2018. This law is another way for patients who have been diagnosed with life-threatening diseases or conditions who have tried all approved treatment options and who are unable to participate in a clinical trial to access certain unapproved treatments.
Clinical trials provide information about whether a product is safe to use and can effectively treat or prevent a disease. People may have many reasons for participating in clinical trials. In addition to contributing to medical knowledge, some people participate in clinical trials because there is no treatment for their disease, treatments they tried have not worked, or they are not able to tolerate current treatments.
Building on FDA’s long-standing efforts to help patients and families who are facing life-threatening diseases or conditions, the FDA is providing information for patients on the Right to Try Act. The FDA’s role in the implementation of the Right to Try Act is largely focused on those obligations outlined in the law, which is limited to the receipt and posting of certain information submitted regarding Right to Try use.
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
- Exhausted approved treatment options and is unable to participate in a clinical trial involving the eligible investigational drug (this must be certified by a physician who is in good standing with their licensing organization or board and who will not be compensated directly by the manufacturer for certifying)
- And has provided, or their legally authorized representative has provided, written informed consent regarding the eligible investigational drug to the treating physician
An eligible investigational drug is an investigational drug:
- For which a Phase 1 clinical trial has been completed
- That has not been approved or licensed by the FDA for any use
- For which an application has been filed with the FDA or is under investigation in a clinical trial that is intended to form the primary basis of a claim of effectiveness in support of FDA approval and is the subject of an active investigational new drug application submitted to the FDA
- Whose active development or production is ongoing and has not been discontinued by the manufacturer or placed on clinical hold by the FDA
If you are interested in Right to Try, you should discuss this pathway with your licensed physician. Companies who develop and make drugs and biologics, also known as sponsors, can provide information about whether their drug/biologic is considered an eligible investigational drug under Right to Try and if they are able to provide the drug/biologic under the Right to Try Act.
Ultimately, sponsors developing drugs for life-threatening diseases or conditions are responsible for determining whether to make their products available to patients who qualify for access under the Right to Try Act.
The full text of the Right to Try Act, Public Law 115-176, can be found on the US Congress’s website: Public Law 115-176.
4. “Right to Try” FAQs
1. If I’m a patient or a physician and I have a question about the Right to Try Act, who do I ask?
A: The FDA recommends that patients first consult with their physician and that physicians consult with the sponsor of the investigational drug or biological product. The sponsor is in the best position to provide information about whether the drug or biological product meets the criteria to be considered an eligible investigational drug for use under the Right to Try Act.
2. If I identify a drug that I would like to use under the Right to Try Act, how do I know if it is an “eligible investigational drug”?
A: The FDA recommends that you consult with the sponsor of the investigational drug or biological product. The sponsor is in the best position to provide information about whether the drug or biological product meets the criteria to be considered an eligible investigational drug for use under the Right to Try Act.
3. Does the FDA review or approve Right to Try Act requests?
A: The FDA does not review or approve requests for Right to Try Act use. The FDA’s role is limited to the receipt and posting of certain information submitted under the Right to Try Act.
4. What does the FDA do with the annual summaries submitted under the Right to Try Act?
A: The FDA will receive annual summaries from manufacturers or sponsors on the use of an eligible investigational drug under the Right to Try Act. The FDA will post a consolidated annual summary report of Right to Try Act use.
5. Does an IRB review and/or approve Right to Try Act requests?
A: Individual Right to Try Act requests do not require IRB (institutional review board) review or approval; however, eligible investigational drugs under the Right to Try Act must meet certain criteria.
6.Is written informed consent required for the Right to Try Act?
A: Yes, a physician is responsible for getting written informed consent from the eligible patient or their legally authorized representative.
7.Does the Right to Try Act obligate a sponsor to provide an eligible investigational drug to an eligible patient?
A: No, the Right to Try Act does not require a sponsor to provide an eligible investigational drug to an eligible patient.
8.If the IND for a drug is on clinical hold, is the drug an eligible investigational drug for use under the Right to Try Act?
A: No, a drug under IND clinical hold is not an eligible investigational drug and a sponsor cannot provide the drug for use under the Right to Try Act.
The FDA is available to answer questions regarding the Right to Try Act.
Patients with specific Right to Try requests should consult their physician.
To request “Right to Try,” Golden Biotechnology Corporation welcomes eligible patients or physicians in the U.S. to contact us to discuss and process applications.
Golden Biotechnology Corporation
101 Hudson Street, Suite 2100 Jersey City, NJ 07302 USA
<NOTICE> These websites may contain general information relating to various medical conditions and their treatment. Such information is provided for informational purposes only and is not meant to be a substitute for advice provided by a doctor or other qualified healthcare professional. Patients should not use the information contained herein for diagnosing a health or fitness problem or disease. Patients should always consult with a doctor or other healthcare professional for medical advice or information about diagnosis and treatment.