When your furry family member has a disease or medical condition, it can be stressful and difficult to watch, especially if there are no available treatments or the available ones don’t seem to be helping. Enrolling your dog, cat, or horse in a pivotal study can be a way to access new, investigational treatments that aren’t yet available, while potentially benefiting millions of pets in the future. Below is more information that can help you make an informed decision about enrolling your pet in a pivotal study.
First, What’s a Clinical Study?
Before we dive into pivotal studies, let’s explore the broader category of clinical studies. A clinical study is used to gather information about a potential new therapy, treatment, medication, or device. Whether the treatment is intended for animals or humans, a clinical study is essential to helping discover new ways to improve patient health.
A “pivotal” veterinary study is the final study conducted before a potential new pet medication is submitted to the Food & Drug Administration (FDA). These studies are called “pivotal” because they are essentially “make-or-break” studies that will inform the FDA’s final decision on whether to approve the treatment.
A medication being evaluated in a pivotal study is generally called an “investigational” or “test” medication, and the pharmaceutical company paying for the study is known as the “Sponsor”. These studies are conducted to learn more about the potential new treatment under conditions that are as close to the real world as possible.
The Important Role of the FDA
In the U.S., neither human nor animal medications can be sold until they have been approved by the FDA. Pet owners will find it reassuring to know that the FDA holds animal medicines to the same rigorous quality standards as medications for humans. The path to FDA approval is usually a long and rigorous one, requiring many phases of clinical studies and may take eight to 10 years for veterinary medications.
Before reaching the pivotal phase, investigational medications must undergo extensive evaluation, including proof of concept (also called “pilot” studies), safety studies, and efficacy (effectiveness) studies.
Pivotal studies for veterinary medications are reviewed by the FDA’s Center for Veterinary Medicine (FDA-CVM). The submission is called a New Animal Drug Application (NADA).
How Will I Recognize a Pivotal Veterinary Study?
There are many non-pivotal veterinary clinical studies conducted across the U.S. These may include smaller studies that may be conducted at veterinary colleges or by veterinary hospitals for purposes other than achieving FDA approval. While it may be difficult for pet owners to know whether a study is pivotal, here are some ways to recognize one:
- There may be 20 or more veterinary clinics participating. The FDA wants to ensure that the treatment is evaluated by multiple investigators (veterinarians who have received specialized study training to screen and enroll patients) in different geographic regions. It is important to know whether the results observed in the study are identified among all study locations.
- There are hundreds of pets being recruited. Studying the effects of a medication in a large number of animal patients helps the FDA ensure statistically significant study outcomes, which can be expected to be consistent across larger populations of pets.
- The study uses a placebo. The majority of pivotal studies compare the effect(s) of the test medication against a placebo. A placebo is an inactive substance in a nearly identical formulation. This helps ensure any perceived benefits from the test medication are valid.
- There’s no cost to participate. Most pivotal studies offer free participation for qualifying pets (dogs, cats, or horses), and may include diagnostics, veterinary exams and care, plus the test medication (or placebo).
- The purpose of the study is to seek FDA approval. Perhaps the easiest way for pet owners to know if a study is pivotal is to ask the study veterinarian if FDA approval is the goal.
What are the benefits of enrolling my pet in a pivotal study?
When you enroll your pet in a pivotal study, both of you become participants in a long chain of events that may lead to healthier pets in the future. If the treatment is approved and brought to market, it can help improve the health of millions of pets and make the lives of their owners easier. Even if the medication is not approved, the information gathered from the study can advance the veterinary community’s understanding of the disease or condition.
Some other benefits of pivotal study participation include:
1. Access to the latest new treatments that aren’t yet available.
If your pet is struggling with a disease or condition, an investigational medication may provide more relief to your pet than those currently on the market.
2. Access to no-cost veterinary care.
The value of the veterinary care received in a pivotal study can range from hundreds to thousands of dollars and often includes:
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- Diagnostic tests such as urinalysis, blood work, radiographs (X-rays), and MRIs
- Veterinary evaluations from trained veterinarians
- Veterinary exams
- New, investigational medications
3. Advancements in human medicine as well.
Many conditions that animals experience — such as obesity, diabetes, and certain types of cancer — are similar or identical to the same conditions in humans, so enrolling your pet in a pivotal study may also help advance medical knowledge about human health.
What else do I need to consider before enrolling my pet in a pivotal study?
Before considering a clinical study for your pet, you should be aware of a few other things. These include:
- Your pet may not be eligible to participate. Each study has “inclusion and exclusion criteria” — conditions that must be met to be included and factors that would make a pet ineligible.” Only the study veterinarian can determine if your pet is eligible to enroll.
- You may need to temporarily forego other treatments. Most studies involve treatments that may provide an incremental benefit over existing therapies. This means you will likely not be allowed to administer any existing treatments for the disease or condition while your pet is enrolled in the study.
- You will need to dedicate time to the study. If your pet is screened and qualifies for enrollment, you will be expected to bring in your pet for monitoring at specified times. You may also be asked to keep a logbook or complete other types of documentation. Each study has different requirements and varying lengths of participation. You should ensure that your schedule can accommodate the commitment.
- The treatment may have adverse effects or no effect. All medications have a risk of adverse effects. However, since pivotal studies are the last studies conducted before a test medication is submitted for FDA approval, more is known about the risks to patients. The study veterinarian will explain the known risks to you so that you can make an informed decision. If your pet receives a placebo instead of the test medication, there may be no benefit.
- The treatment being studied may successfully help your pet but may not be available after the study ends. As mentioned above, the FDA approval process is lengthy and rigorous. Even if the investigational medication is found to improve your pet’s condition, it may not be approved (and made available to the public) for years, if at all.
Enrolling My Pet In a Pivotal Study: What’s Next?
If there’s a pivotal study in your area and you believe your pet qualifies, then discuss it carefully with your veterinarian. Make certain you understand what will be required and anything else that will help you make an informed choice. Pivotal studies, like all clinical studies, are completely voluntary.
When our furry family members have a disease or condition, we want to do anything in our power to help them. Participating in a veterinary pivotal study may be one of the best ways you can do that, while at the same time providing information that may lead to FDA approval for treatments that can help future generations of animals live healthier, longer lives.
FAQs
1. What’s the difference between a veterinary clinical study and a pivotal study?
“Veterinary clinical study” is an overarching umbrella term that includes many types of studies. By contrast, a “pivotal study” is the final study conducted before a test medication is submitted for potential FDA-CVM approval by a pharmaceutical sponsor.
2. What is a “placebo”? Why do most pivotal studies use a placebo in addition to the test medication?
A “placebo” is a treatment that generally matches the appearance of the test medication. It contains no medication, and therefore has no therapeutic benefits. It can take the form of a sugar pill, salt-water (saline) injection, or even a fake surgical procedure.
In the course of the study there may occur what is known as “the placebo effect.” This happens when a patient’s physical or mental health appears to improve, even though the medication (in the form of a placebo) had no active medicinal properties. Therefore, most veterinary clinical studies will compare a group of patients that has received a placebo against a group that has received the test medication to ensure that any benefits observed from the study are valid and repeatable.
3. What is a “double-blind” study?
A “single-blind” study is one in which the participant (or the participant’s owner/parent/guardian) does not know which treatment, if any, the participant is receiving. A “double-blind” study is one in which neither the researchers nor the participants know which patients have received the placebo. This helps minimize potential bias and maximize the validity of the research results. A double-blind placebo study is the gold standard in both human and veterinary clinical research.
4. How can I tell if the FDA has approved a product I’m considering for my pet?
All FDA-reviewed and approved medications, whether for humans or animals, contain a label or package insert. Confirmation of approval will be found near the bottom of the label, and it will be written as “Approved by FDA under NADA #XXX”, the unique number that the FDA has assigned the product. If your veterinarian provided your pet’s medication in a pharmacy bottle, rather than the original package from the manufacturer, it’s possible that you may not have received the package insert. Simply ask your veterinarian’s office for a copy of the package insert.
About Visionaire Research & Education
For over 20 years, Visionaire Research & Education has helped veterinary medication sponsors bring new treatments to market. Our number one priority is helping dogs, cats, and horses — and their owners — experience a better quality of life through innovative treatments and solutions. The pivotal studies that we support can be a great way to gain access to quality diagnostics and veterinary care — and investigational therapies that often aren’t available anywhere else — all at no cost. If you have questions about the work we do, please feel free to contact us.