Small Things Have a Big Impact: Why Patients are the Most Important Part of Clinical Trials

Each drug that doctors prescribe to patients goes through a lengthy process before gaining approval from the Food and Drug Administration (FDA) and becoming available for patient use. On average, the process of creating a concept for a new drug for FDA approval takes 12 to 15 years. Discovery is the first of several stages each drug passes through on its way to approval. Patients who volunteer their time to test new drugs and offer their input during clinical trials play a critical role in the process.

What Happens during the Prescription Drug Discovery Phase?

The discovery phase is multi-faceted and begins with identifying a biological target. Drug manufacturers choose a target based on what they want to change or duplicate in order to achieve the intended therapeutic effect. Researchers identify biological targets through genomes, which is the medical term for genetic targets. They also use bioinformatics and other targets that they expect to have a positive impact on symptoms of the targeted disease.

Next, medical researchers begin to prepare compounds focused on one or more of the following:

• Method of administration
• Specific protein target
• Therapeutic area

Researchers use a variety of methods when investigating and comparing compounds, including proprietary software and statistical models. The next step is to begin the medicinal chemistry process for small molecules, which involves undergoing chemical synthesis to identify structures relevant to the drug creation process.

In vitro preclinical studies are the last part of the discovery phase. Researchers test cellular disease models, selectivity, efficacy, specificity and optimized properties of the drug under development.

Preclinical Drug Development is a Two-Phase Process

Drug researchers are now ready to begin in vivo models on animal test subjects. The drug under development receives the status of Investigational New Drug (IND), which enables studies on drug safety and the drug manufacturing process.

Clinical Trials with Human Subjects

The purpose of the first three phases of clinical trials is to establish the safety and effectiveness of the new drug in human subjects. The FDA requires most drug manufacturers to complete a Phase IV clinical trial after it has received approval from the government oversight agency.

A drug could not move forward to FDA approval and doctors writing prescriptions for it without the cooperation and feedback of people who volunteer for clinical trials. Researchers obtain input from patients early and often throughout each study. They also request other stakeholders to share their input and observations, such as doctors who would prescribe the drug. Here are just some of the benefits of partnering with human test subjects during clinical trials:

• Using their feedback in the development of future studies that provide detailed and concise answers to questions regarding disease outcomes and the effectiveness of the IND on those outcomes.
• Improved patient experiences with management of the disease the IND treats.
• Practical operation components and the ability to get continual feedback based on patient experience.

Patients and their caregivers help to determine the goals of each study and the information it provides. Other parties involved in each phase of clinical trials include primary care providers and specialists, the product manufacturing team and the clinical team. The latter includes each of the following:

• Clinical operations personnel
• Clinical scientists
• Data management personnel
• Medical and safety personnel
• Regulatory personnel

Statisticians Each person involved with clinical trials should have a specific focus assigned to them. For example, a clinical scientist comes up with a hypothesis and creates a trial to support the IND’s action on specific disease symptoms. The role of clinical operations is to ensure proper administration of the drug study, while medical teams look after patient safety. Statisticians and data scientists supervise data trends and quality throughout the study, starting with design and ending with reporting.

Discussion between stakeholders is vital to the success of any clinical trial, as it increases quality and lowers costs. Ensuring early feedback from study participants reduces dropout rates. Retaining study participants is essential in saving time and expenses associated with recruiting and training new participants.

How Clinical Trial Developers Can Improve Patient Recruitment

Attracting the most qualified study participants requires deep knowledge about the patient population. One effective strategy for understanding patient populations is to interview people who have received previous treatment for the disease in question. Clinical trial managers should also interview primary care doctors, specialists and patient caregivers to gain better insight into who to recruit.

After creating an initial list of inclusion criteria, the clinical team must remain flexible and be willing to accept mistakes in their original assessment. Resolving issues with inclusion criteria is crucial before advancing too far into the trial.

The clinical team also needs to look at the experience from the patient’s perspective to determine if expectations are too demanding and whether the proper patient support is in place. If clinical researchers uncover issues with patient experience, they must address them immediately before the problems have a cascading effect on patient dropout rates.