Clinical Trials

What You Need to Know

When researchers have completed the rigorous screening and preclinical testing process for a potential new medicine, the company files an Investigational New Drug (IND) or Biologics License (BLA) application with the U.S. Food and Drug Administration (FDA). This application allows the investigational medicine to be tested in human volunteers in clinical trials. A clinical trial is a carefully designed study which tests the benefits and risks of a specific medical treatment or intervention, such as a new drug or a behavior change (e.g., diet).

The FDA requires a multi-phase clinical trials process to be completed before deciding if the medicine under investigation is safe and effective for a broader patient population. Usually, the number of human volunteers in the trial increases as the treatment moves through these phases, which is why innovative medicines cannot be developed without the help of volunteers who participate in clinical trials.

Scientists at the FDA carefully review all the data from all of the studies on the drug under investigation and, after weighing the benefits and risks of the potential medicine, decide whether to grant approval.

Clinical Trials

The Biopharmaceutical Research & Development Process

From drug discovery through FDA approval, developing a new medicine takes at least 10 years on average and costs an average of $2.6 billion.* Less than 12% of the candidate medicines that make it into Phase 1 clinical trials will be approved by the FDA.

Biopharmaceutical Research

The Biopharmaceutical Research & Development Process

Clinical Trial Diversity

Increasing diverse enrollment in clinical trials is a critical step to improving access to medicine development and the biopharmaceutical industry has taken action to enhance clinical trial diversity. The demographics of a clinical trial should, to the extent possible, reflect the intended treatment population. In addition to individual company efforts, PhRMA and its members voluntarily developed and adopted industry wide principles on clinical trial diversity. These principles focus on four main areas: building trust and acknowledging the historic mistrust of clinical trials within Black and Brown communities, reducing barriers to clinical trial access, using real-world data to enhance information on diverse populations beyond product approval and enhancing information about diversity and inclusion in clinical trial participation. Yet despite progress and the steps taken by the FDA and the biopharmaceutical industry, clinical trials do not always reflect the demographics of the intended treatment populations.

Stakeholders must continue to come together to address systemic barriers to participation if we are going to make further progress. That’s why PhRMA, in July of 2022, funded a grant to launch Equitable Breakthroughs in Medicine Development. The initiative is a comprehensive effort to increase diversity in clinical trials and address systemic barriers to participation by communities of color. This effort seeks to help underrepresented patients be more involved in the research and development of potential life-saving medical treatments.

Clinical Trial Diversity

Clinical Trial Data Transparency

Clinical trial data transparency aims to improve access to clinical trial research and results in order to inform medical decision-making, advance scientific discovery and accelerate the development of new treatments to benefit patients. The biopharmaceutical industry has long-established commitments to facilitate appropriate transparency throughout the lifecycle of a clinical trial including registration, results reporting, and data sharing, and the biopharmaceutical industry is at the forefront of initiatives to improve access to clinical trial data in order to advance medical research.

Data Transparency

Research in Your Backyard

Biopharmaceutical companies are conducting clinical trials across the country, connecting patients with potential treatments and working toward new medicines and cures. PhRMA has created reports showcasing clinical trials in a number of states, highlighting their importance to patient health as well as the contribution to the local economy. The reports focus on clinical trials targeting six of the nation’s most debilitating diseases: asthma, cancer, diabetes, heart disease, mental illness, and stroke.

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Clinical Trials

What You Need to Know

The Biopharmaceutical Research & Development Process

Biopharmaceutical Research

Clinical Trial Diversity

Clinical Trial Diversity

Clinical Trial Data Transparency

Data Transparency

Research in Your Backyard

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