One of the central goals of the newly passed ACT for ALS is to increase access to experimental drugs for people living with amyotrophic lateral sclerosis (ALS). This is an extremely important development for the many people living with this 100% fatal, often fast-progressing disease with no effective cures or treatments.

The way the new law aims to increase access to experimental drugs is by providing funding that will make it easier for clinical trial sponsors to establish Expanded Access Programs (EAPs). EAPs, also sometimes referred as compassionate use programs, are a pathway for people who do not qualify or cannot participate in a clinical trial to access treatments that are not yet approved by the FDA. EAPs are reserved only for cases where a disease is immediately life-threatening, and there are no other available therapeutic options.

Why are EAPs Important?

In the US, the system of clinical trials and FDA approval is designed to prevent harm to patients, as well to keep them from wasting precious time and money on treatments that will not have any effect on their condition. Phase 1, 2, and 3 clinical trials are conducted to demonstrate that a potential treatment for a disease is safe and effective. FDA review exists to make sure that the data gathered in these trials is accurate and reliable.

For many people with terminal and/or debilitating illnesses with few treatment options, one of the only ways to access a potential treatment is by participating in a clinical trial. However, this option is not available to everyone – some may not qualify for a trial based on its enrollment criteria, while others may not have a trial site near them. For some, there just might not be enough spaces available in a trial. This is a particularly challenging position for people with a fast progressing, fatal disease like ALS – the average lifespan after an ALS diagnosis is 3-5 years, while the clinical trial process and its three phases often takes close to a decade to complete.

Applying for EAPs

EAPs allow people who wish to try treatments that are not yet approved a way to access them. To apply for an EAP, a person living with a disease must first seek approval from the physician handling their case. The physician then reaches out to the manufacturer or trial sponsor to request access to the drug or treatment.

If the company is willing and able to provide the drug to the patient they must then seek approval from the FDA, as well as their Institutional Review Board (IRB), a panel of experts authorized by the FDA to monitor their research. When deciding to approve an application for an EAP, each individual and organization in this process must consider:

  • whether there are any alternative treatments available
  • whether the potential benefits of accessing the treatment outweigh the risks for the patient
  • and, whether providing access to the drug will interfere with any clinical trials.

Once approval has been granted, the patient (or patients) are given an authorization called a “investigational new drug” (IND), and may be granted access to the drug under their supervision of their physician, as well as the manufacturer.

The Types of EAPs

FDA regulations outline three different categories of EAPs, differing by the number of participants. The smallest are single-patient INDs – EAPs established so one individual can access an experimental treatment. If a drug company anticipates more than one patient, but fewer than would be involved in a typical trial, they can apply for an intermediate-size IND. When an EAP involves more than one patient, more robust safety information about the drug in question is usually required for approval.

Treatment INDs are the largest tier of EAP – the enrollment size can range from hundreds to tens of thousands. This is usually reserved for treatments with a large amount of evidence already available supporting their safety and efficacy. This means that they mostly involve drugs that are already in phase 3 clinical trials, or that have completed a phase 3 trials but are still awaiting FDA approval. Unlike individual and intermediate size EAPs, these INDs may only be requested by the treatment’s manufacturer, rather than an individual’s physician.

EAP Challenges

When an individual requests access to an investigational treatment through an EAP, the treatment’s manufacturer has no legal obligation to grant the request or pursue an IND – it is solely at their discretion. Similarly, even for drugs that show promise in the later phases of clinical trials, there is no requirement for a company to request a treatment IND to make the drug widely available.

For many companies the cost of these programs might be considerable – and take resources away from the clinical trials that are needed to get the treatment in question approved to bring it to market. Historically, there have been tough ethical questions involved in considering EAPs running in parallel to clinical trials because many trial participants will be on placebo – raising the question of whether it is fair to provide the drug to people outside of the trial before those in the placebo group have access to it.

EAPs and the ACT for ALS

A major motivating factor behind ACT for ALS was to address some of these issues and make it easier for companies with potential ALS treatments to run EAPs. It aims to do this by providing government-issued grants for EAPs for drugs in phase 3 clinical trials. These grants will be administered by the U.S. Department of Health and Human Services (HHS) and will be available for any clinical trial sponsor or clinical trial site that provides access to treatments to people who are not eligible to enroll in a clinical trial for ALS.

Any company or group applying for one of these grants will need to demonstrate that the data generated from this expanded access program will be used to advance ALS research and will not interfere with the conduct of ongoing clinical trials. The grants will cover the sponsor or site’s expenses related to providing access to the drug to people with ALS that are ineligible for trials.

ALS TDI believes the passing of ACT for ALS is a testament to the power of the ALS community and will offer hope to many living with ALS. We know that we will need to discover many more drugs to meet the needs of everyone with ALS. So, as we celebrate this accomplishment, ALS TDI will continue to build on this hope by inventing and discovering more drugs for ALS - until everyone living with ALS has access to effective treatments.

Key Takeaways: 

  1. The ACT for ALS' main mechanism for increasing access to investigational treatments for ALS is by providing grants to help pay for Expanded Access Programs (EAPs).
  2. EAPs are programs run by trial sponsors that make experimental treatments available to those who do not qualify or cannot participate in a clinical trial.
  3. An EAP can be run for a single patient, a small group of patients, or groups of up to hundreds or thousands of people.
  4. Someone suffering from a terminal or debilitating illness with no alternative treatments may request an EAP through their Doctor – but companies have no obligation to grant these requests.

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