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Investigational Drug Study

Definition An Investigational Drug Study, in the context of VA benefits, refers to a clinical trial or research study conducted to evaluate the safety, efficacy, and/or dosage of an experimental drug, typically in a controlled environment. These studies are pivotal in the development and eventual approval of new medications for various conditions and diseases. Participants, […]


An Investigational Drug Study, in the context of VA benefits, refers to a clinical trial or research study conducted to evaluate the safety, efficacy, and/or dosage of an experimental drug, typically in a controlled environment. These studies are pivotal in the development and eventual approval of new medications for various conditions and diseases. Participants, including eligible veterans, may receive the investigational drug under FDA guidelines, with the purpose of advancing medical knowledge and potential treatment options.

Key Takeaways

  1. Investigational Drug Studies are clinical trials conducted by the VA to test new treatments and medications, with the goal of improving the health and well-being of veterans.
  2. Veterans who participate in these studies can potentially gain access to cutting-edge therapies and contribute to the advancement of medical knowledge for the benefit of fellow veterans and the general public.
  3. Participation in investigational drug studies is voluntary, and veterans can choose to withdraw at any time. The VA maintains strict guidelines to ensure the safety and well-being of the participants during the study.


The VA benefits term “Investigational Drug Study” is important because it refers to the research and evaluation process of new, unapproved drugs or therapies on a selected group of participants, often veterans.

These studies play a crucial role in developing innovative treatments and enhancing the healthcare provisions available to veterans.

By participating in investigational drug studies, veterans help advance science and medical knowledge, improving future healthcare services for themselves and fellow veterans.

Moreover, such studies become a significant addition to their VA benefits, potentially granting them access to cutting-edge and potentially life-saving treatments before they become widely available.


The purpose of an Investigational Drug Study in the context of VA benefits is to provide veterans with access to cutting-edge drug therapies that have the potential to significantly improve their health outcomes or quality of life. These studies are carried out to evaluate the safety, efficacy, and, in some cases, effectiveness of new, promising, or previously approved medications. This targeted program ensures that the brave men and women who have served our country are afforded every opportunity for medical advancements and treatments that could help them combat and manage their various health conditions.

Participation in these studies allows veterans to contribute to furthering medical science and finding novel therapies for illnesses affecting the entire veteran community. Investigational Drug Studies within the VA system follow strict protocols and are in line with the standards set by the Food and Drug Administration (FDA) for such studies. Participating veterans may benefit from early access to potentially life-changing treatments before they become widely available to the general public.

Additionally, veterans in these studies will typically receive close monitoring and support from a team of dedicated healthcare professionals, who will ensure that any potential side effects or adverse reactions are addressed with care. It is worth noting that while investing in Investigational Drug Studies could be immensely helpful for veterans, it may not always guarantee a substantial benefit. Nevertheless, these studies remain an essential tool in our collective efforts to improve health outcomes and quality of life for our esteemed veterans.

Examples of Investigational Drug Study

AVXS-101 Study: In 2016, the Department of Veterans Affairs (VA) announced the AVXS-101 study, an investigational drug study for spinal muscular atrophy (SMA) in SMA1 patients from neonates to children up to 8 years of age. The study assessed the safety and efficacy of AVXS-101, which is designed to target the underlying genetic cause of SMA compared to standard symptomatic therapy. The investigational drug study aimed to improve motor function and survival rates in SMA patients and potentially offer a new treatment option.

PTSD and MDMA-Assisted Psychotherapy Study: In 2020, the VA partnered with the Multidisciplinary Association for Psychedelic Studies (MAPS) to conduct a clinical trial evaluating the safety and effectiveness of MDMA (ecstasy) as an adjunct to psychotherapy for post-traumatic stress disorder (PTSD) in veterans and first responders. The investigational drug study aimed to determine whether MDMA-assisted psychotherapy may offer a potential treatment option for treatment-resistant PTSD cases.

ADCS A4 Study: The Alzheimer’s Disease Cooperative Study (ADCS) A4 study, a nationwide clinical trial, was conducted at multiple VA research centers in partnership with other research institutions to test the efficacy of an anti-amyloid investigational drug called solanezumab on individuals who have evidence of amyloid plaque in their brain but have not yet developed symptoms of Alzheimer’s disease. The goal of the study was to determine if the drug could slow down memory loss and cognitive decline associated with Alzheimer’s, thus potentially preventing the onset of the disease in at-risk individuals.

FAQs: Investigational Drug Study

What is an Investigational Drug Study?

An Investigational Drug Study is a clinical research trial designed to evaluate the safety, effectiveness, and dosage of new medications or treatment approaches on volunteer participants. Such studies might be sponsored by pharmaceutical companies, medical institutions, or government agencies like the Veterans Affairs (VA) to ensure the best treatment options are available to veterans.

Are veterans eligible to participate in Investigational Drug Studies?

Yes, veterans are eligible to participate in Investigational Drug Studies approved by the VA. However, specific eligibility criteria may vary depending on the study and the health condition being targeted. It’s important for interested veterans to discuss the study and their eligibility with their healthcare providers.

How does the VA protect the safety and well-being of veterans in these studies?

The VA follows stringent safety guidelines and adheres to ethical standards to protect the well-being of veterans participating in Investigational Drug Studies. Researchers must obtain informed consent from participants, and studies must be reviewed and approved by Institutional Review Boards and other regulatory entities to ensure the study is safe and ethical.

How can a veteran participate in an Investigational Drug Study?

Veterans interested in participating in an Investigational Drug Study should first consult with their VA healthcare provider. The healthcare provider can discuss whether a specific study is suitable for the veteran’s condition and provide guidance on how to enroll in the study if eligible.

Does the VA cover the costs related to participation in an Investigational Drug Study?

Generally, the VA covers the costs associated with Investigational Drug Studies, including medication, tests, and treatments related to the study. However, it’s essential for veterans to discuss the financial aspects of participation with their healthcare providers and study coordinators. In some cases, additional support from sponsors or grants may be available to cover other costs.

Related VA Benefit Terms

  • Clinical Trial
  • Experimental Treatment
  • Control Group
  • Placebo Effect
  • Informed Consent

Sources for More Information

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