Therapeutic Areas in Clinical Trials – BioPharma Services

Recent decades have brought about important innovations in drug development, including novel clinical trial designs, digital endpoints, and regulatory changes. These factors have contributed to the increased complexity of clinical trials. However, the wide range of therapeutic areas in clinical trials also contributes to their complexity. Moreover, different therapeutic areas may pose specific challenges regarding volunteer recruitment, clinical trial design, adherence to regulatory guidelines, required medical procedures, and data analysis.

To address the complexity of clinical trials across diverse therapeutic areas, it can be very beneficial to collaborate with an experienced clinical research organization (CRO) that has the know-how of a multidisciplinary team of experts.

What are Therapeutic Areas?

Therapeutic areas are fields of research and therapeutic development for groups of similar medical conditions. Globally, oncology is the therapeutic area with the highest clinical trial volume. It is followed by mental health and behavioral disorders, endocrinology and metabolic diseases, cardiovascular and circulatory diseases, and nervous system diseases. 

As a full-service Phase 1 CRO, BioPharma Services is involved in drug development programs across a variety of therapeutic areas, including cardiovascular diseases, oncology, neurology, psychiatry, pain, infections, digestive system diseases, metabolic disorders, reproductive diseases, blood disorders, urological diseases, and respiratory disorders.

Drug development trends across therapeutic areas 

Innovative research and clinical tools, such as the use of engineered human cells, preclinical modeling, real-world data, and artificial intelligence, continue to find applications in drug development.

During the preclinical stage of drug development, animal models have long been established as the gold standard to characterize the safety and pharmacological effects of NCEs. However, challenges in extrapolating animal-derived data to humans and concerns about animal welfare have emerged. To address these concerns, there has been a trend toward decreased use of animal studies and greater implementation of engineered human cell lines and preclinical modeling in drug development programs. Human disease models with the potential to provide insight into the pharmacological and safety characteristics of NCEs include 2D-cell cultures, organoids, bioengineered tissue models, and one-organ and multi-organ models.

As clinical trials are limited in their duration, they can deliver safety and efficacy data concerning a fixed period. Real-world evidence (RWE), that is generated during routine clinical practice, can provide additional data regarding the use, safety, and efficacy of a drug. Sources of RWE may include patient health records, registries, pharmacy claims, and social media. Even though the broadest application of RWE has been in collecting safety data about a drug, it can also help instruct the design of future clinical trials.

In addition, there is a tendency towards the adoption of artificial intelligence and new technological platforms in clinical drug development programs. The implementation of artificial intelligence can help to improve testing efficiency and accuracy and promote the fast delivery of reliable data. More specifically, artificial intelligence may optimize clinical trial design, helping to transition clinical trials faster from the planning to the delivery stage. In addition, artificial intelligence tools may aid in the practical aspects of clinical trial delivery and provide novel simulation tools.

Regulatory aspects of drug development across therapeutic areas

The drug development process is highly regulated and should adhere strictly to the quality standards of Good Clinical Practice (GCP) and Good Laboratory Practice (GLP). Accordingly, regulatory affairs professionals play an important role in drug development. In addition, certain therapeutic areas may be associated with specific regulatory requirements. For example, central nervous system (CNS)-active compounds are subject to human abuse potential (HAP) studies that should adhere to a set of regulatory guidelines. Therefore, for the success of drug development programs, it is critical to work with a team of regulatory professionals with extensive expertise across relevant therapeutic areas in clinical trials.

As numerous diseases across therapeutic areas still lack sufficiently effective and safe therapies, there is a need for accelerated therapeutic development. To enable the faster development of therapeutics, Accelerated Approval Programs have been established by regulatory agencies. Accelerated Approval Programs allow the approval of an NCE targeting a serious condition with an unmet medical need based on its effect on a surrogate endpoint. Surrogate endpoints are laboratory, radiographic, physical, or other markers that are thought to predict clinical benefit but are not themselves a direct measure of clinical benefit. 

BioPharma Services adheres strictly to all relevant regulatory guidelines and to the principles set forward by the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH). Our regulatory inspection history is flawless and includes inspections by the US FDA, Health Canada, World Health Organization, Austrian AGES, Danish DKMA, Dutch CBG, French ANSM, United Kingdom MHRA, and ANVISA. We are also GLP-certified by the Standards Council of Canada.

Emerging therapeutic areas in drug development

Advances in scientific research have been translated into new types of therapeutic targets or NCEs for the drug development process. Gene, RNA-targeted, and cell therapies have emerged as powerful new therapeutic tools. These types of therapeutics hold great promise for diseases lacking effective treatments, such as genetic epilepsies.

Gene therapies aim to modify the genetic code to restore impaired protein functions. They may include gene transfer and genome editing approaches. Gene transfer strategies restore the function of a missing or faulty gene by delivering a normal version of the gene or another gene bypassing the cellular dysfunction. Genome editing introduces targeted changes to cellular DNA to restore normal cellular function. It can remove a disease-causing DNA sequence, turn off a malfunctioning gene, correct a mutated gene, or turn on a gene encoding a required protein. 

RNA-targeted therapies exert their effects by modulating RNA molecules, features, or functions. Classes of RNA-targeted therapies include anti-sense oligonucleotides (ASOs), messenger RNAs (mRNAs), small interfering RNAs (siRNAs), micro RNAs (miRNAs), RNA aptamers, splice-switching oligonucleotides, and CRISPR-Cas9-mediated gene editing. 

Cell therapies transfer healthy cells into the body to replace diseased cells, modulate cells, or remove dysfunctional cells. They may involve stem or non-stem cells as well as autologous or allogenic cells. Even though a number of cell therapies have already obtained FDA approval, cell therapy development still faces challenges, such as the difficult standardization of manufacturing processes, safety concerns, and high costs.

The role of complex medical procedures in clinical trials

With the increasing complexity of clinical trials, the techniques used to deliver NCEs and to assess their efficacy and safety have also evolved. Complex medical procedures play an important role in clinical drug development and can be used in a variety of scenarios, such as the assessment of pharmacokinetic or pharmacodynamic effects of an NCE or for dosing facilitation. As a full-service Phase 1 CRO, BioPharma Services has expertise in the use of complex medical procedures, including lumbar punctures, intraperitoneal injections, muscle biopsies, and ultrasound examinations, for clinical drug development in different therapeutic areas. Read more on our Complex Medical Procedures.

The significance of biostatistics, data management, and data science

During complex clinical trials, a high volume of diverse data is collected. Biostatisticians and data managers are involved in the statistical analysis plan (SAP) design, power analysis, data analysis, data interpretation, and data storage. Moreover, with the implementation of artificial intelligence in drug development, the role of data scientists in clinical trials continues to increase and evolve.

How biomarkers can help drug development across therapeutic areas

Biomarkers are quantifiable and objective characteristics of normal or abnormal biological processes or medical conditions. They can measure different aspects of biological processes or diseases and have been classified into risk, safety, diagnostic, monitoring, pharmacodynamic, predictive, and prognostic biomarkers. In addition to being used as endpoints, biomarkers can be implemented in the design of clinical trials. These biomarker-led clinical trials can stratify study volunteers based on the presence or absence of biomarkers or can use biomarkers to help select the most appropriate therapeutic intervention. 

The role of adaptive study designs

Even when they have been thoroughly planned and designed, clinical trials can deliver unexpected findings. Adaptive clinical trial designs allow investigators to introduce modifications to the clinical trial itself or to its data analysis based on interim data or predefined cut-off values while preserving the clinical trial’s integrity and validity. Due to their potential to contribute to cost and time savings, adaptive designs have found increasing applications in clinical trials across therapeutic areas.

Steps for study sponsors with drug development programs across therapeutic areas

As conducting clinical trials across therapeutic areas requires multidisciplinary expertise, working with an experienced CRO with know-how across scientific functions and therapeutic areas may expedite and streamline drug development programs. Moreover, a competent CRO can help transition NCEs through the various stages of preclinical and clinical drug development and plan strategically for the complete drug development program already during its early stages. BioPharma Services is a full-service Phase 1 CRO with expertise across a number of therapeutic areas in clinical trials.

If you want to collaborate with an award-winning Phase 1 CRO that can support your drug development program across a wide range of therapeutic areas, complete the form below to schedule a discovery call with a member of BioPharma’s scientific and medical team.