Compassion Corner: Compassionate Use, Expanded Access Part Two: The Terminology Is Different But the Unmet Need Is the Same

Continued From Part One

Compassion [kuhmpash-uhn] noun
A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering.

Compassion Corner is a weekly series from Patient Worthy that will focus on the subject of compassion in the healthcare and rare disease space. In this series, we explore the role of compassion in this field and what it means for caregivers, patients, and others.

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About the Right to Try Act

Dissatisfaction with the Expanded Access Program (EAP) led to patient groups aligning with a think tank named the Goldwater Institute and initiating a movement they called Right to Try. Their efforts led to a law called the Right to Try Act. Although the new federal law gives patients in the U.S. an alternative that allowed accessing drugs prior to FDA approval, the law created a considerable amount of controversy.

Healio’s HemOnc Today, in its March 2020 issue, gave details from both sides of the Right to Try issue. Those who oppose the new law claim it seeks to undermine the FDA and fuels the Goldwater Institute’s mission to push the government to the sidelines in these matters.

Speaking on behalf of the proponents of the Expanded Access Program, Professor Alison Bateman of NYU told HemOnc Today that in emergency situations, the FDA may only take days for completion of its portion of the EAP process.

On the other hand, clinicians point out that the Right to Try Act does not have the same protections and oversight as the FDA’s program and thus could be harmful to cancer patients. The clinicians also note that the FDA approves the majority of requests it receives through its Expanded Access program.

Colorado Passed the Act in 2014

Colorado was the first state to adopt the Right to Try Act. Colorado was followed by Louisiana and Missouri and then the Act was passed at the federal level. The new law allows patients to have access to drugs before the completion of a phase 1 trial when a determination is usually made as to the drug’s efficacy and safety. Opponents were quick to object, saying that the law will harm people by giving them access to unproven drugs.

Early Access: A Timeline

In 2014 the Ebola Crisis in West Africa resulted in a number of governments advocating for investigational drugs by way of the Expanded Access Program.

The Early Access to Medicines Scheme ( EAMS) began in April 2014 in the U.K. EAMS allows doctors to use EAMS under life-threatening conditions. EAMS allows access to off-label and unlicensed drugs for patients with unmet needs.

Then in 2016 new policies emerged in Washington, D.C., and Europe. The FDA streamlined the Investigational New Drug (IND) application in response to patient advocates claiming the IND paperwork was excessive. In addition, the FDA changed the approval process by requiring only one Board member to approve the treatment.

The Right to Try Act was followed by the 21st Century Cures Act requiring disclosure by drug developers of their Expanded Access Policy to be uploaded on their website and on the Clinical Trials database.

About the same time, the EMA initiated the Priority Medicines scheme allowing patients access to novel therapies earlier.

In 2017 the FDA revised guidance for adverse events and clarified how data would be included in its review process.

Help was provided by the Reagan-Udall Foundation to give physicians and patients a better understanding of the Expanded Access programs.

During this period a rare disease organization in Europe issued a report urging that medicines should be widely available for patients who are urgently in need of help.

The Italian government issued rules for Expanded Access stating that access may be requested for products that were not authorized as well as medicinal products used off label (approved for other disorders). Provisions were also included to allow the use of medicine approved abroad but not yet approved in Italy.

In 2018 the Right to Try Act became law. The basic elements are similar to Expanded Access minus the FDA’s oversight and protections. About the same time, the report on the Expanded Access Program was released indicating that absent standard policy, an environment of confusion now surrounded Expanded Access. Drug developers were not certain how the FDA makes use of data pertaining to adverse events it receives from the Expanded Access Programs.

2019 the FDA launched a program called Project Facilitate which is a call-center used by physicians for their Expanded Access applications specifically for oncology patients. This was followed by Post-Trial Access. Developers would utilize the Expanded Access process and allow patients who completed a clinical trial to continue the use of drugs that show potential.

Looking Forward

The Ebola health emergency and the COVID-19 pandemic have incentivized the World Health Organization and other organizations to recognize the importance of Expanded Access. Although regulatory authorities worldwide may use different approaches to Expanded Access programs, patients will benefit most if everyone participates.

 

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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