How Clinical Trials Work

Protocols: 

Protocols are essential guidelines for conducting clinical trials. They outline the trial's purpose, procedures, and the criteria for participant eligibility, ensuring that everyone involved knows what to expect. These documents detail the schedule of tests, dosages of any treatments, and how long the study will last. Importantly, they define the study’s goals (known as endpoints), such as what health outcomes will be measured. By strictly adhering to these protocols, clinical trials maintain high standards of accuracy and safety, providing reliable results that will be reviewed by health authorities.

Collaboration Between Trial Sponsors and Investigating Sites: 

Clinical trials rely on the collaboration between trial sponsors, such as pharmaceutical companies, and investigating sites, including hospitals and clinics. Sponsors design the trial's structure, provide the necessary funding, and supply resources like medical equipment. Investigating sites are responsible for carrying out the trial according to established protocols, ensuring all procedures are unbiased and meet ethical standards. Effective communication between sponsors and sites is crucial, often supported by digital tools and platforms that manage data and coordinate trial activities efficiently.

Role of the Principal Investigator (PI): 

The Principal Investigator (PI) is a senior medical professional with extensive experience in the field related to the clinical trial, responsible for overseeing the management of the clinical trial at the site level. The PI ensures the trial is conducted safely and according to strict scientific and ethical standards. The duties include overseeing participant safety, maintaining the integrity of the data collected, and leading the site team. They also analyze and report the trial's results. If the need arises due to preliminary findings or emerging risks, the PI has the authority to make necessary changes to the trial, including adjusting study protocols or even halting the trial early to protect participants and ensure compliance with all regulations

Clinical Trial Phases

Clinical trials are structured into four parts, called phases, each with a distinct purpose and specific goals that must be  achieved before moving to the next phase.

Preclinical testing: 

Before a new drug is tested on humans, it undergoes preclinical testing. This step involves laboratory experiments and animal studies to determine if the drug is both safe and potentially effective. Successful results are required before the drug can advance to clinical trials involving human participants. 

Phase I - Initial Safety Checks

• Participants: The Phase I study is conducted on a relatively small group of participants, ranging from ten to a hundred healthy volunteers. In cases where the drug is intended to treat severe conditions such as cancer, patients with the specific disease may be included. 

• Purpose: The primary focus is to evaluate the safety, identify side effects, and in general determine how the body reacts to the drug. A method known as dose escalation determines the highest dose that can be administered safely without severe side effects, known as the maximum tolerated dose (MTD).

• Process: Participants are closely monitored as the dosage is gradually increased. This careful monitoring helps ensure the drug's safety profile is thoroughly evaluated.

• Outcome: If the drug is found to be safe with manageable side effects, the study progresses to Phase II for more comprehensive testing.

 Phase II - Efficacy and Side Effects

• Participants: This phase typically involves several hundred patients with the disease the drug is intended to treat.

• Purpose: Phase II focuses on evaluating whether the drug effectively treats the condition it is designed for while continuing to assess its safety monitoring side effects. The research further refines the drug's dosage to achieve the best balance between efficacy and safety. 

• Process: The drug is administered to patients under conditions that allow researchers to observe and document its effects. This is crucial for determining the optimal therapeutic dose that maximizes benefits while minimizing adverse effects. 

• Outcome: If the drug demonstrates effectiveness and maintains a manageable safety profile, it moves on to Phase III, which involves a more extensive and diverse group of participants to confirm these findings.

Phase III - Efficacy and Monitoring of Adverse Reactions

• Participants: Involves hundreds to thousands of patients who have the condition the drug is designed to treat.

• Purpose: To confirm the drug’s effectiveness based on results from earlier phases and compare it to standard treatments or placebos. This phase also includes a comprehensive assessment of side effects across diverse populations.

• Process:

• Extensive monitoring of the drug’s effects and side effects.

• Comparisons are made with standard treatments or placebos to validate the drug’s efficacy and safety.

• Outcome: Successful completion can lead to the treatment being submitted for regulatory approval, allowing doctors to prescribe it.

Phase VI - Postmarketing Surveillance

• Participants: Several thousand volunteers using the approved treatment.

• Purpose: To collect additional information about the drug’s long-term effect and ongoing safety after it has been approved and is on the market.

• Process:

• Continuous monitoring for any long-term or rare side effects.

• Ensuring that the treatment meets safety standards.

• Outcome: This phase helps refine treatment guidelines and ensures rapid management of any new risks identified, maintaining the drug’s safe and effective use on the broader population.

Compassionate Use Programs (Expanded access)

Compassionate use programs allow seriously ill patients to access new treatments not yet available to the public. These programs are sometimes offered as an option when no other treatments are available, to provide potentially life-saving opportunities.

At Inform Life, we help patients and healthcare providers navigate these opportunities through our platform's search functionality. This functionality allows users to easily find and access trials offering compassionate use options, helping patients connect with innovative treatments when needed.