What are Expanded Access Studies?

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What are Expanded Access Studies?

Introduction:

Many of you might be unaware about the expanded access programs which are otherwise known as compassionate use studies. Knowing about these programs will be important to you as the number of disease conditions are increasing day by day and such programs will be helpful to you and your friends/relatives in some way. This article will let you know about the expanded access programs in detail...

Defining Expanded Access:

This program would refer to the use of a particular investigation new drug outside of a clinical study. As such, the investigational drugs will be administered to the patients who carry life-threatening disease conditions.(1) In simple terms, an expanded access program will use an investigational drug and/or biologic in order to treat the patients with serious disease conditions who otherwise do not have any satisfactory alternative therapy to treat the existing disease condition.(2)

Why Expanded Access Programs?

The approved drugs will not always be available for some serious illnesses. And, not every patient will respond to the approved drugs for a number of reasons. (3) Only at these conditions, an expanded access program comes into play wherein a promising experimental drug/device made available to these patients. It is to be noted that, just as in the clinical trials, the investigational drug/device that is being used in these programs has not yet been approved. Also, these drug candidates have not been proven to be safe or effective. (4)

Various Types of Compassionate Use:

The regulations of the U.S. Food and Drug Administration have allowed the patients to access the investigational drugs or biologics through various expanded access programs since the year 1987. At present, the FDA approves the expanded access for the following situations:

- On case by case basis for a particular patient.

- For intermediate size groups of subjects with similar treatment requirements and who otherwise will not qualify to participate in the clinical trials.

- For larger groups of patients who do not any other treatment option available.(4)

Criteria for Considering Expanded Access:

It is important to keep in mind that the company which is developing the drug or device must approve your request for expanded access. Without the corresponding company's approval, your expanded access to an investigational drug will not happen. Following are the criteria for considering your expanded access:

- Drug/Device must be in Active Development: In order to get approval from the company, the investigational drug must be part of an active development program. One must remember that, once the FDA approves a drug/device, an expanded access program or compassionate use will not be available.

- Access must not Interfere with Clinical Trial or Approval: Granting access to any investigational drug by the company must not interfere with the completion of ongoing clinical trials that can support the approval of the particular drug. It must not also compromise the potential development of the experimental drug/device.

- Patients should not Qualify for Clinical Trials: An important criterion for gaining access to the investigational drug will be that the patient must not qualify for ongoing or planned clinical trials of the investigational drug.

- Risk-Benefit Assessment: The potential benefits to the patients seeking access to the investigational new drug will always be considered to outweigh the collective risks to the patients including the outcomes of disease itself.

- Patients with Safety Risks: Patients with any underlying medical condition that might pose safety risks that have not been characterized and/or studied will not be eligible for expanded access.

Besides the above mentioned criteria, the expanded access can also be approved in an emergency situation which is known to be an 'emergency IND'. Such an emergency IND could be requested over phone by your physician and will be approved by a FDA authorized official. (4), (5)

What needs to be done Before Requesting an Expanded Access?

Before requesting for an expanded access, you will need to do the following:

- Searching for possible clinical trials you may be eligible for by visiting the major clinical trial registries.

- Searching for specific expanded access programs by using a search engine online.

- Calling drug/device Company directly for asking about their policies.

- Contacting some patient advocacy organizations in order to check whether they have information on the expanded access programs.

You are advised to consult with your healthcare provider in order to see whether the use of an experimental drug/device for treating your medical condition will be right for you. Also, be sure to consider the following aspects:

- How much is known about the particular experimental drug/device?

- Severity of your medical condition

- Likelihood that the use of the particular experimental drug/device will be effective

- Kind of illness you have

- Stage of the disease

- Any other condition you may be experiencing

- Organ functions (Example: Liver, Kidney, etc., ) (4)

Benefits of Expanded Access Programs:

Following are some of the important benefits of an expanded access program:

- Through an expanded access program, we can provide access to patients with life-threatening diseases who otherwise have no other alternatives.

- Through such programs, we can provide the patients the measure of autonomy over their own health decisions.

- The compassionate use could help bridging the gap between the later stages of drug development and approval.

- The expanded access could help fostering the development of additional applications of a particular investigational drug.

- This could help offering the patients the hope with no other options available.

Risks Associated with Expanded Access Programs:

With regards to an expanded access program, there are unknown risks associated with the access to an investigational drug or device for which there is only limited information about the safety and efficacy available. Through an expanded access, some patients might get benefitted; some patients might experience no effect and some other patients might be harmed. (4)

Conclusion:

A patient may decide to seek access to an investigational drug/device for various reasons. All the patients are advised to carefully read the informed consented that is being presented to you and make sure that you understand the risks that are associated with the drug or device before signing. (3), (4)

References:

(1) http://en.wikipedia.org/wiki/Expanded_access

(2) http://amda-pompe.org/downloads/transcripts/FDA_expanded_access_slides.pdf

(3) http://www.fda.gov/ForPatients/Other/OffLabel/ucm20041767.htm

(4) http://www.fda.gov/ForPatients/Other/ExpandedAccess/ucm20041768.htm

(5) http://www.gene.com/patients/investigational-medicines/criteria

About the Author

Tharani Rajamanickam's picture

I am Tharani. I have Bachelors degree in Biotechnology. I am passionate about the healthcare and medical industry and continue to explore, learn and share and I bring in a wealth of knowledge and expertise to help you keep updated with current trends and developments. I have gained a lot of experience in clinical field by working in clinical trials arena. Stay tuned..

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