The ALS Association

Right to Try and FDA Expanded Access

May 31, 2018

 

Federal Right to Try Legislation
The Right to Try Act of 2017 was signed into law by PresidentTrump on May 30, 2018. Each individual pharmaceutical company will decide how it will respond to patient requests under the Right to Try law. It’s too early to tell what individual companies will do, but this piece will provide some background on both Right to Try and the FDA’s Expanded Access program.

How the Drug Approval Process Works 
To understand Right to Try and the FDA Expanded Access program – it’s important to first understand the drug development process. Pharmaceuticals in the United States are regulated by the Food and Drug Administration. When a drug company develops a new compound intended for patient use, the medication goes through several phases that often take years to complete.

Each phase has a different purpose and helps researchers answer different questions.

  • Phase I trials: Researchers test a drug or treatment in a small group of people for the first time. The purpose is to study the drug or treatment to learn about safety and identify side effects.
  • Phase II trials: The new drug or treatment is given to a slightly larger group of people to determine its effectiveness and to further study its safety.
  • Phase III trials: The new drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it with standard or similar treatments, and collect information that will allow the new drug or treatment to be used safely and effectively.

  • FDA Approval

  • Phase IV trials: After a drug is approved by the FDA and made available to the public, researchers track its safety in the general population and seek more information about a drug or treatment’s benefits and optimal use.

Key Provisions of The Right to Try Law

  1. Eligible Patient 
    An “eligible patient” is someone who has been diagnosed with a life-threatening disease or condition and has exhausted approved treatment options and is unable to participate in a clinical trial involving the eligible investigational drug, as certified by a physician.

  2. Eligible Investigational Drug
    The term ‘eligible investigational drug’ means a drug for which a Phase 1 clinical trial has been completed but the drug has not been approved or licensed for any use. The drug must be the subject of an active investigational new drug application under FDA.

  3. Impact Clinical Outcomes for FDA Approval 
    The FDA may not use a clinical outcome associated with the use of an eligible investigational drug to delay or adversely affect the review or approval of such drug unless FDA determines that such clinical outcome is critical to determining the safety of the eligible investigational drug or the sponsor requests use of such outcomes.

  4. Annual Report 
    The manufacturer or sponsor of an eligible investigational drug shall submit to the Secretary of Health and Human Services an annual summary of any use of such drug under this section. The summary shall include the number of doses supplied, the number of patients treated, the uses for which the drug was made available, and any known serious adverse events. The Secretary shall post an annual summary report of the use of this section on the Internet website of the FDA.

  5. No Liability for Manufacturer, Prescriber, Dispenser 
    - No liability shall lie against a sponsor manufacturer, prescriber, dispenser or other individual entity for its determination not to provide access to an eligible investigational drug. 
    - With respect to any alleged act or omission with respect to an eligible investigational drug provided to an eligible patient - no liability in a cause of action shall lie against a sponsor or manufacturer; or a prescriber, dispenser, or other individual entity (other than a sponsor or manufacturer), unless the relevant conduct constitutes reckless or willful misconduct, gross negligence, or an intentional tort under any applicable State law.

Federal Right to Try Law – What Does It Mean for Patients? 
The new federal law supersedes the Right to Try laws that exist in 40 states. The federal Right to Try law gives terminally ill patients the ability to access experimental medications that have not yet been approved by the FDA but have completed Phase I trials. The request does not have to be approved by the FDA.

Nothing in the new law makes it mandatory for pharmaceutical companies to provide these medications or for insurance companies to cover drugs under Right to Try. Thus, patients must still convince pharmaceutical companies to provide drugs outside of trials, and patients must still bear the costs for these experimental treatments.

Terminally-ill patients can work with their doctors to ask pharmaceutical companies for access to medications that have not completed the rigorous testing required by the FDA and without waiting for the drugs to become commercially available. However, a pharmaceutical company may not be prepared to provide drugs to terminally ill patients under the Right to Try law. Giving access to preapproval drugs can be costly, particularly given the limited supply, and almost no medical insurance will cover experimental treatments.

Drug companies also may worry that making a drug available without FDA approval opens them up to lawsuits if the drugs prove ineffective or have unforeseen side effects. The new federal Right to Try law does provide some protections for companies in this regard, but until those protections have been tested in court, it is likely companies will remain cautious.

Lastly, providing the drug to patients outside of clinical trials can also be disruptive to the FDA approval process because it can lead to data on negative clinical outcomes outside the highly controlled trial setting. While the new law prevents this data from being used unless it is deemed "critical to determining safety," undesirable outcomes might give the FDA pause and delay the approval of drugs.

Food and Drug Administration Expanded Access

Expanded access, sometimes called "compassionate use," is the use of an investigational medical product (i.e., one that has not been approved by the FDA) outside of a clinical trial.

According to the FDA, the use of an investigational medical product by a patient as part of a clinical trial is preferable because clinical trials can generate data that may lead to the approval of products and, consequently, to wider availability. However, when patient enrollment in a clinical trial is not possible (e.g., a patient is not eligible for any ongoing clinical trials, or there are no ongoing clinical trials), patients may be able to receive the product, when appropriate, through expanded access.

According to a 2016 study published in the journal Therapeutic Innovation & Regulatory Science about 99 percent of submitted applications for expanded access to almost 9,000 investigational drugs were approved over a 10-year period between January 2005 and December 2014. Emergency requests for individual patients are usually granted immediately over the phone and non-emergency requests are generally processed within a few days.

Requirements for All Expanded Access Uses

Under the Federal Food, Drug, and Cosmetic Act (FD&C Act), a patient may seek individual patient expanded access to investigational products for the diagnosis, monitoring, or treatment of a serious disease or condition if the following conditions are met:

  • The patient and a licensed physician are both willing to participate.

  • The patient's physician determines that there is no comparable or satisfactory therapy available to diagnose, monitor, or treat the patient’s disease or condition.

  • That the probable risk to the person from the investigational product is not greater than the probable risk from the disease or condition.

  • FDA determines there is sufficient evidence of the safety and effectiveness of the investigational product to support its use in the particular circumstance.

  • FDA determines that providing the investigational product will not interfere with the initiation, conduct, or completion of clinical investigations to support marketing approval.

  • The sponsor (generally the company developing the investigational product for commercial use) or the clinical investigator (or the patient’s physician in the case of a single patient expanded access request) submits a clinical protocol (a document that describes the treatment plan for the patient) that is consistent with the FDA’s statute and applicable regulations for the investigational new Drug (INDs) or investigational device exemption applications (IDEs), describing the use of the investigational product.

  • The patient is unable to obtain the investigational drug under another IND or to participate in a clinical trial.

Under the FD&C Act statute, a sponsor or a physician may also submit a protocol intended to provide widespread access to an investigational product for multiple patients. In this scenario, FDA will permit the investigational product to be made available under a treatment IND or treatment IDE if certain criteria are met. Ensuring patient safety is a priority - FDA must determine that the potential patient benefit justifies the potential risk of the expanded access use of the investigational drug, and that the potential risk is not unreasonable in the context of the disease or condition to be treated.

 

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