We invite you to join us as we deep-dive into our database and share unique and interesting insights into various therapeutic areas, including the current and potential future state of drug development for particular diseases.
In our monthly series “In Focus: Therapeutic Area Insights,” we’ll select a disease from one of the key therapeutic areas we cover and answer questions like:
- How is this disease generally treated?
- How many industry-led, FDA-regulated interventional clinical development programs are currently ongoing?
- Who are the top sponsors of these programs?
- What clinical development phases are these programs in?
Our series is designed to give you a better understanding of what is going on right now in drug development in specific therapeutic areas and diseases. We’ll showcase diseases from the three key therapeutic areas we cover: oncology, immunology/inflammation and central nervous system (CNS) diseases, while shining a bigger spotlight on various disease awareness days and months.
Therapeutic Area Focus Oncology
Oncology deals with cancer and related treatments. This comprises various types of malignancies, including both solid tumors and hematological cancers.
Disease Burden of Oncology
According to the WHO, cancer is a leading cause of death worldwide, accounting for nearly one in six or 10 million deaths in 2020. The most common types of cancer are breast, lung, colorectal and prostate cancer, which make up more than 40% of all cancers diagnosed worldwide. Estimates are that there will be 28 million new cases of cancer each year by 2040.
Oncology Research and Development
Given that health need, oncology is the most researched therapeutic area. It has the highest number of clinical trials of any therapeutic area. As of 2024, approximately. 29% of all trials actively recruiting patients in the ClincalTrails.gov database are cancer-related.
The Intelligencia AI platform currently* captures over 2000 Phase III trials and almost 10,600 trials in Phase I and II for both solid and liquid cancers.
Mechanisms of Drug Action for Oncology
Cancer drugs have various distinct mechanisms of action: cytotoxic agents directly kill rapidly dividing cells by interfering with DNA replication, damaging DNA or disrupting cell division; targeted therapies target specific molecules involved in cancer cell growth and survival; hormone therapies block or lower the levels of hormones that stimulate cancer growth, immunotherapies enhance the immune system’s ability to recognize and attack cancer cells, angiogenesis inhibitors prevent the formation of new blood vessels, and epigenetic modulators alter the expression of genes involved in cancer progression.
Oncology Outcomes
Advances in early detection, screening, and treatment have improved cancer survival rates over the last 30+ years. The overall cancer death rate continued to decline from 2019 to 2020 by 1.5%, contributing to a 33% overall reduction since 1991. This progress reflects advances in treatment, which are particularly evident in the decline in mortality for leukemia, melanoma, and kidney cancer despite stable/increasing incidence and accelerated declines for lung cancer.
Economic Burden of Oncology
The economic burden of cancer is substantial. In 2020, the total annual economic cost of cancer globally was estimated at approximately $1.16 trillion, accounting for healthcare costs, lost productivity and informal care. A study by the European Society for Medical Oncology estimates that the global economic cost of cancers from 2020 to 2050 will be $25.2 trillion.
Therapeutic Area Focus Immunology/Inflammation (I&I)
The therapeutic area of Immunology/Inflammation deals with the study and treatment of diseases that involve the immune system and inflammatory processes, including autoimmune diseases, allergies, chronic inflammatory diseases, immunodeficiency disorders and transplant immunology.
Disease Burden of Immunology and Inflammation
Immunological and inflammatory diseases comprise a common but clinically diverse group of conditions with different disease burdens. Autoimmune diseases such as rheumatoid arthritis, lupus and multiple sclerosis affect approximately 10% of the global population and are among the leading causes of morbidity and mortality.
Allergic diseases, such as asthma, have increased over time, are currently affecting approximately 10% to 30% of people worldwide and are a major cause of illness and disability.
Chronic inflammatory diseases such as inflammatory bowel disease and chronic obstructive pulmonary disease affect around 16 million people in the U.S. alone. These diseases result in frequent hospitalizations and long-term treatments.
Immunodeficiency disorders comprise relatively rare primary immunodeficiency diseases and secondary immunodeficiencies (e.g., HIV/AIDS), which continue to affect millions worldwide and lead to significant morbidity and mortality rates.
Transplant patients face chronic immunosuppression, increasing their risk of infections and certain cancers.
I&I Research and Development
The rising prevalence of immunological and inflammatory diseases has led to increased research and development activities. Biologics are the main drivers based on the success of monoclonal antibody treatments.
The Intelligencia AI platform currently captures more than 3,500* trials in all phases of development, of which more than 350 are biologics.
Mechanisms of Drug Action for I&I
The main drug mechanisms of action rely on modulating the immune response and reducing inflammation. The primary mechanisms include the use of anti-inflammatory agents that inhibit the production or activity of pro-inflammatory molecules, immunosuppressants that suppress or reduce the strength of the body’s immune response, disease-modifying antirheumatic drugs that slow disease progression by modulating the immune system, monoclonal antibodies that target specific antigens on immune cells or cytokines, and cytokine inhibitors that block the activity of specific cytokines involved in the inflammatory response.
I&I Outcomes
The outcomes of immunological and inflammatory diseases have generally improved over the past decades, driven by advancements in medical research, improved therapeutic strategies and early diagnosis.
Some diseases, e.g., rheumatoid arthritis and multiple sclerosis, have seen enhanced survival rates; quality of life has improved for many patients due to novel treatment modalities (e.g., biologics). Immunotherapies (e.g., checkpoint inhibitors and CAR-T cell therapy), hold promise for treating autoimmune and inflammatory diseases in addition to cancer. Focus on long-term management of chronic diseases and integrated care approaches have led to better control of chronic diseases and better outcomes.
Economic Burden of I&I
Many of the immunological and inflammatory diseases are chronic and, therefore, have a substantial economic burden due to direct medical costs and lost productivity.
Therapeutic Area Focus Central Nervous System (CNS) Diseases
The therapeutic area of CNS diseases comprises a group of neurological disorders that affect the structure or function of the brain or spinal cord. These disorders may have various causes, e.g., infection, injury, blood clots, age-related degeneration, cancer, autoimmune dysfunction, and birth defects. The symptoms vary widely, as do the treatments.
Disease Burden of CNS
Globally the burden of CNS diseases is large and growing due to a variety of factors, including an aging population. A study finds that collectively, CNS diseases ranked as the leading cause of disability-adjusted life-years (DALY) in 2021, affecting 3.4 billion people worldwide, which is approximately 43% of the global population.
CNS Research and Development
Despite decades of research and development, there are currently no drugs that can prevent or cure many of the CNS diseases and only limited options for symptomatic relief are available. Research into CNS diseases is hampered, e.g., by the fact that animal models for CNS diseases are insufficient. Novel techniques, e.g., genomic profiling, brain imaging, novel trial designs, AI and disease biomarkers, promise to support a better understanding of these diseases and their underlying causes.
The overall CNS pipeline shows significant growth after a prolonged period of limited interest by pharmaceutical companies in the therapeutic area due to one of the lowest success rates of all TAs and long approval times. Increased funding by the NIH, the significant unmet medical needs, and other factors have led to a renewed interest in this therapeutic area.
It now accounts for 14% of the overall industry R&D pipeline, second only to oncology by the number of assets in development.
The Intelligencia AI database currently contains more than more than 2,400* trials in all phases related to CNS diseases.
Mechanism of Drug Action for CNS
Common mechanisms of action for CNS disease drugs include neurotransmitter modulation, i.e., modulation of how various drugs influence the activity, release, reuptake, or degradation of neurotransmitters. This includes the modulation of gamma-aminobutyric acid, the glutamate system, dopamine, serotonin, the cholinergic system and ion channels.
CNS Outcomes
New treatments have succeeded in improving the outcomes of certain CNS diseases. For example, biologics have improved outcomes for diseases like migraine; precision medicine has led to better-targeted therapies and improved patient outcomes in diseases such as epilepsy and various other neurodegenerative disorders.
Economic Burden of CNS
The unmet need and the burden of illness in CNS continue to be high, and the COVID-19 pandemic has aggravated the situation. For 2019, the WHO estimated the long-term impact as 256 million disability-adjusted life years (DALYs) globally, an increase of 16% since 2010. This translates into significant direct and indirect costs, with one 2017 study calculating the economic burden to be approximately $800 billion annually in the United States alone.
Download Our Therapeutic Area Insights Brief
Download all In Focus: Therapeutic Area Insights issues below, and check back to see next month’s findings.
- May: Lupus
- June: Scleroderma
- July: Juvenile Arthritis
- August: World Breast Cancer Research Day
- September: Ovarian Cancer
Are you curious about data-driven insights on these and other disease areas? Let us know here.
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*Data as of July 30, 2024, and reflects FDA-track interventional clinical trials that meet our data capture criteria.