Pre-Clinical and Clinical Trials

A Practical Approach to Clinical Trial Diversity

A 2021 study in the Journal of the American Medical Association found that minority groups were underrepresented in US-based vaccine clinical trials, with white people accounting for ~80% of participants (1). Although researchers broadly acknowledge that trial representation should mirror the many different populations who could benefit from a drug, lack of diversity continues to hamper clinical-trial effectiveness. To address this problem, the pharmaceutical industry must increase awareness about clinical trials and reduce barriers to participation in them. Benefits of…

eBook: Rare Diseases — Biopharmaceutical Challenges Presented By Relatively Small Patient Populations

By definition, an orphan disease affects a small percentage of the population. However, with some 7,000 such conditions identified so far, they collectively have a significant impact on global health. An estimated 350 million people are affected worldwide by a rare disease — altogether more than the population of the world’s third largest country (the United States). Some well-known biopharmaceutical companies are devoted to developing treatments for rare diseases. However, the vast majority of such diseases have no treatments approved by…

The Promise of Artificial Intelligence in Healthcare

The term artificial intelligence (AI) has become pervasive in conversations about the future of healthcare. AI has the potential to transform medicine through novel models of scientific discovery and healthcare delivery, ultimately leading to improved individual and public health. Yet misunderstanding and miscommunication abound. Thus, concepts related to AI need to be defined and explained to elevate our general level of understanding and our discourse around the topic. The Promise of AI in Healthcare AI has been studied by computer…

Bioengineering for “Benchtop Clinical Trials”

Animal studies can be poor predictors of human drug response. Species-specificity of organs is a concern especially for the heart. Many drugs that enter clinical trials will fail ultimately because of unexpected cardiotoxicity. Drug developers would love to mitigate such risks through in vitro human cardiac testing, but human heart biopsy materials and donor organs do not survive well in a laboratory setting. Breakthroughs with human stem cells offer an alternative. It is now possible to take a simple skin…

Emerging Treatments for Spinal Cord Damage

Injuries to the spinal cord can cause permanent paralysis and even lead to death, with little or no hope for patients to regain lost function after such trauma has occurred. News of my spinal cord research first came to prominence in the 1980s with a front-page story that chronicles a presentation at the annual Society for Neuroscience meeting (1). That reported the first time that crushed peripheral nerves had been regenerated back into a patient’s spinal cord. Regeneration of spinal…

Is the QbD Toolbox Ready for Cell and Gene Therapies? Integrating Patient Outcomes into Manufacturing Cell and Gene Therapy Bioproducts

In their lifecycle development and manufacturing models, biotechnology products and the biopharmaceutical industry have been founded on principles originating from the pharmaceutical small-molecule industry. Such principles define clinical programs that establish risk benefits of a dosage and its delivery system on healthy individuals and patients. A company then develops a process to manufacture that product consistently over several years. Product quality attributes set through manufacturing controls are expected to ensure patient outcomes in terms of safety and efficacy and deliver…

Partnerships in Immunotherapy for the Future of Cancer Treatment

Immunotherapy seeks to harness the power of our human immune system to fight disease. In this rapidly evolving field, collaboration among different stakeholders is essential to bringing new treatments to market. Patient advocacy groups, researchers, hospitals, manufacturers, and government entities all are working together to translate promising new research into life-saving products. Types of immunotherapy include monoclonal antibodies (MAbs) and antibody derivatives, checkpoint inhibitors (immune-modulating proteins), cancer vaccines, T-cell therapies, and cytokines — so the approach involves a range of…

Elucidation: Better Biomarkers Could Power Precision Medicine for Autoimmune Disease

Precision medicine has long been a tantalizing goal for the pharmaceutical and healthcare industries. Fields such as oncology and rare diseases have benefited greatly from implementation of genome-guided care. Drug developers likewise have made strides with biomarker-aided discovery programs and stratification for clinical trials. Current indications suggest that autoimmune diseases will be the next major focus of personalized medicine. The goal of precision medicine often is described as “getting the right drug to the right patient at the right time.”…

Your Brand Is the Patient’s Experience

The future success of biopharmaceutical businesses will depend at least partly on their ability to create meaningful brand experiences from the start of a drug program. By “brand,” I don’t mean logos and taglines. I’m talking about meaningfully unique experiences that directly affect clinical and patient needs — specifically, to address the growing demand for self-administered injectable therapeutics. Whether you are a biosimilar developer trying to carve out differentiated value or a market leader looking at your patent protection in…

Reverse Translational Medicine: The Promising Future of Failure

The drug development landscape is awash with candidates that have shown enormous promise and efficacy in preclinical models but failed when administered to clinical trial subjects. Although such failures occur for different reasons, one of the most pervasive causes is the inability of preclinical models to recapitulate human physiology accurately. Despite advances with both in vitro and in vivo models, improving those toward a more accurate avatar of the human physiological process remains a challenge. Central to that effort will…