WHAT IS A CLINICAL TRIAL?

The purpose of clinical trials is to investigate whether an investigational medication is safe and effective in the treatment of a disease. Clinical trials must follow strict legal and scientific rules and guidelines. This helps protect the patients' rights and safety.

Only approved medications are allowed for use by prescription. Approval is given by healthcare authorities such as the US Food and Drug Administration (FDA) and European Medicines Agency (EMA). The authorities only approve a medication when it has been proven to be safe and effective. To obtain information about safety and effectiveness of a new medication, clinical trials are performed.

There are four phases of clinical trials.

Phase I

The first study in humans. These are small studies with 20-100 participants, mostly in healthy volunteers. The main objectives are to investigate:

• Safety of the study medication
• How the study medication is absorbed by the body and what dosage should be used
• How the study medication is removed from the body
• Potential side effects

Phase II

Small studies with around 100-500 participants. The main objectives are to investigate:

• Ongoing safety
• Whether the study medication works for a particular disease
• The best dose of the study medication

Phase III

Large studies with around 500 or more participants. These are the main studies for final approval by health authorities. The main objectives are to investigate:

• Safety and side effects in bigger populations
• Whether the study medication works for a particular disease
• How the treatment compares to already existing standard therapies

Phase IV

Large studies in patients after the study medication has been approved by regulatory authorities for prescription use (or public use if it isn't a medicine that requires a prescription). The main objectives are to investigate:

• Side effects during day-to-day use in the population
• Risks and benefits over a longer period of time

Clinical trials must be approved by government health authorities before patients can enter the trial and potentially receive the investigational medication. There are strict rules around how clinical trials must be run, and each trial is different. Patients must meet specific criteria to be included in a clinical trial.

Sometimes patients cannot join a clinical trial if they:

• Have other health problems in addition to the condition being studied
• Are taking certain other medicines that may affect the trial result and/or put the patient at risk

For these reasons, in some cases, these patients may not qualify for a clinical trial. Ask your doctor if participating in a clinical trial is right for you. The study physician will also help determine if a patient is a good fit for the study in question.

1. Eligibility check

To see if the trial is a potential good match for you, you will answer an online survey (also called pre-screening questions) and you will be put in touch with a nurse that will perform a short phone-interview with you about the study. If you are eligible for the study, you will be contacted by a study clinic and invited to a screening visit.

2. Informed Consent Form (ICF) Review

The ICF helps you to make an informed decision about whether or not to participate. The study nurse or doctor is required to review the ICF with you.

The form includes:

• What you need to know about the study
• Patient signature (your signature)
• Doctor signature

Please note that you can always decide to stop your participation in the study.

3. Screening visits

4. Treatment

The treatment period is different for each study. A treatment period can last a few weeks, several months or, in some cases, years. During the treatment period you will go for regular visits to your clinic, where the study staff will monitor your progress.

5. Follow-up

After the treatment ends there may be a follow-up period where your health is monitored by the study doctors. It may include visits to the study clinic for performing final tests or phone calls with the study team. The length of the follow-up period varies between clinical trials.