Frequently asked questions

A clinical trial (also known as a clinical study) is a type of research that evaluate effects on human health outcomes. People volunteer to participate in clinical trials to test investigational medical interventions (this could include drugs, cells and other biological products, surgical procedures, radiological procedures, devices, behavioral treatments and preventive care). Clinical trials help answer important questions about an investigational therapy or treatment like, "Does it work?" "What are the potential side effects and potential risks?” and "Does it work better than an existing option?" Clinical trials are an important step in the process of developing investigational treatments and getting them to patients.

Clinical trials are carefully designed, reviewed and conducted. They need to be approved before they can start and are monitored while they are running. People of all ages can take part in clinical trials, including children.

Regulatory authorities around the world oversee all clinical trials. In the U.S., the Food and Drug Administration (FDA) regulates clinical trials. In Europe, it’s the European Medicines Agency (EMA). Health Authorities in each country or region look at the information from clinical trials to determine if the investigational therapy or treatment gets approved for patients or not.

Many different types of people from around the world participate in clinical trials. Some trials include healthy volunteers, while other trials include patient volunteers. Some trials include both healthy and patient volunteers. Vertex is committed to supporting clinical trials with diverse participant representation.

When you participate in a clinical trial, you are contributing to research that could bring an investigational therapy or treatment one step closer to reaching patients who need them. All investigational therapies or treatments have to go through a series of clinical trials before being approved. Thousands of people take part in clinical trials, globally, every year. Without clinical trial volunteers, medical progress and innovation would not be possible.

If you’re interested in participating in a Vertex clinical trial, either as a healthy volunteer or as someone with a medical condition, we encourage you to speak with your health care provider first about your eligibility and what is involved.

Before clinical trials are conducted in humans, preclinical studies, also sometimes called laboratory studies are conducted. Clinical trials are done only after collected and reviewed preclinical data findings suggest that the investigational therapy or treatment is likely to be safe and may work in people.

Phase 0 clinical trials use only a few small doses of an investigational drug in a few people, and the drug is given only for a short time. These types of trials are not a required part of the investigational drug approval process, but they could be conducted to help speed up and streamline the approval process.

Phase 1 clinical trials answer the question, "Is the investigational treatment safe?"

Phase 1 trials of an investigational treatment are typically the first trials that involve people. Phase 1 trials aim to find the highest dose of the investigational treatment that can be safely given to trial participants without causing severe side effects. These trials also help researchers to decide the best way to give the investigational therapy or treatment to patients.

Phase 2 clinical trials answer the question, "Does the investigational treatment work?"

If the Phase 1 trial shows the investigational treatment is safe, a Phase 2 clinical trial is conducted. Phase 2 trials involve a larger number of people usually who are living with the medical condition or disease that the potential investigational treatment is being developed to treat. This research will determine if the investigational product is effective and continues to evaluate safety.

Phase 3 clinical trials answer the question, "Is the investigational treatment better than the standard of care?"

If an investigational treatment is shown to work in Phase 2 clinical trials, a Phase 3 trial will be conducted to compare safety and effectiveness of the investigational treatment with standard of care to patients. An investigational treatment must have clinical data evidence from all Phases before it will be considered for approval.

Phase 4 clinical trials answer the question, "What else do we need to know?"

Despite testing an investigational therapy or treatment on many people, the longer-term effects of the treatment cannot be fully understood. Treatments approved by Health Authorities, such as the FDA or EMA are often observed for longer periods of time in what are called Phase 4 studies. These studies are conducted to answer questions about ongoing safety and efficacy.

All clinical trials have guidelines about who can and cannot participate. These guidelines are found in the clinical protocol and are called eligibility criteria (sometimes also called inclusion and exclusion criteria). The eligibility criteria is determined by a clinical trial sponsor (like Vertex) and the regulatory authority. Researchers develop an idea of the group of participants that have qualities that they want to learn more about and test the investigational therapy or treatment and compare its effect, if any, in participants who have similar characteristics. Eligibility criteria help researchers conduct a trial safely and efficiently with participants to get reliable results and also helps researchers identify individuals who can safely participate in a specific clinical trial.

If you’re interested in participating in a Vertex clinical trial, either as a healthy volunteer or as someone with a medical condition, we encourage you to speak with your health care provider first about your eligibility and what is involved.

The decision to participate in a clinical trial is very personal, but things you should consider include:

• What do I hope to gain from participating in a clinical trial?
• What is the main purpose of the clinical trial I am considering?
• Who is eligible to participate?
• What will happen during the clinical trial?
• What are the potential risks and possible benefits?

If you’re interested in participating in a Vertex clinical trial, either as a healthy volunteer or as someone with a medical condition, we encourage you to speak with your health care provider first about your eligibility and what is involved.

To find a Vertex clinical trial, visit the Search page of this site. Clinical trials (sponsored by Vertex and other organizations) are also listed on clinicaltrials.gov.

Each trial has its own rules about who can — or cannot — participate. This is called "eligibility." Eligibility may be based on a person’s geographic location, age, gender, biological sex, overall health, type and stage of a disease, treatment history, and other conditions. Not everyone is eligible to participate.

The exclusion criteria for a clinical trial state who cannot take part in the trial, and these criteria vary for each trial. For example, people who are already taking certain medicines may be excluded as these may affect the investigational treatment being studied. If you do not qualify for a particular clinical trial, consult with your health care provider as there may be other trials you will be eligible for in the future.

Each trial is different and has a unique set of criteria, including age requirements. Vertex conducts clinical trials with the goal of having the treatments approved for the youngest age groups possible.

Before a clinical trial begins, persons wanting to participate will be asked to sign an Informed Consent document, then the medical team running the trial may review medical history with you and run tests. Persons wanting to participate can ask as many questions as they want before the trial begins or at any time after the Informed Consent document is signed.

Informed consent is the idea that a patient should have as much information that they need before making decisions about their medical care or in this case, whether or not to participate in a clinical trial. Organizations that conduct clinical trials have a responsibility to ensure that the consent a participant gives is informed. Vertex has an "Informed Consent" document that outlines all known risks of participating in a clinical trial. Trial staff will walk you through the trial schedule and design. You should ask questions to be comfortable deciding whether or not to participate. Before starting a clinical trial, an Informed Consent document must be signed.

When a clinical trial is over, your commitment comes to an end. The information that has been gathered during the trial will be examined by Vertex and considered alongside information from other trials to see if the potential investigational treatment works, what the possible side effects are and if there is a real benefit for patients.

Information about the trial and its results will be publicly posted in accordance with regulatory requirements and your identity will be protected.

Depending on the type of trial and the trial design, participants may be assigned to one or more treatment option: placebo, the investigational medicine being tested or an already approved medication. The participant will not know which option they are receiving.

At every stage of the trial, the health and well-being of the participants is the highest priority. Participants are carefully monitored throughout and regular health checks may be conducted at the clinical trial site, at home or over the telephone.

When a trial is designed, the way the medicine is given and the tests that need to be conducted decide where clinical trials take place. Locations can include specialist treatment centers, hospitals, universities, doctor’s offices, health clinics, research centers and even your own home. Some trials may require volunteers to travel longer distances; please take this into consideration before signing the Informed Consent form.

A clinical trial may be very short or may take many years; it depends on what is being studied and how long information needs to be gathered to draw a conclusion. The expected length of participation in the trial will be clearly outlined in the Informed Consent form.

A protocol is developed that describes exactly how a clinical trial will be conducted and how to help ensure volunteers are protected. Before a trial can start, the protocol must be reviewed and approved by an institutional review board (IRB). An IRB is made up of physicians, a scientist, a non-scientist, and a representative from the community. Throughout the trial, regular checks are made to make sure that the protocol is being followed and the trial is being conducted safely.

Before agreeing to participate in a clinical trial, the trial staff will walk you through the trial schedule and design and talk about any known risks. You should ask questions so you can decide if you are comfortable moving forward. By reviewing and signing an Informed Consent form you are confirming that you understand all the information you have been given and agree to participate in the research trial. Participation in clinical trials is completely voluntary. You can change your mind and stop participating by withdrawing consent at any time.

Placebos have been used in clinical trials for many years and are an important part of research. A placebo is an exact visual copy of the therapy being investigated that does not, in fact, contain any active drug. In a trial called "Placebo Controlled", some of the participants will receive the investigational medicine and some will receive the placebo. Researchers compare the effect of the investigational medication against the effect of the placebo to find out if the medicine has a real benefit.

It is rare to be paid for taking part in a clinical trial, but in most cases, your costs associated with the clinical trial may be reimbursed. Potential stipends and reimbursements are explained during the informed consent process.

There may be costs associated with your clinical trial, and in some cases, the trial sponsor may reimburse you for your costs. Potential costs and reimbursements are explained during the informed consent process.

Vertex is committed to advancing medicine and improving patients' lives. This includes the responsible sharing of clinical trial data with qualified researchers, but your identity will always be protected when sharing any clinical trial data.

Data from the clinical trial will be published according to the regulations in the region where the clinical trial is conducted.

Vertex conducts clinical trials with the best interests of patients participating in trials, including preparing and sharing clinical trial data with third-party researchers while ensuring individual patient confidentiality and privacy. In the U.S., this means complying with the Health Insurance Portability and Accountability Act (HIPAA), which establishes national standards to protect individual medical records and other personally identifiable health information, where applicable.