The Value of Cancer Treatment Today

The Fight Against Cancer

Innovative biopharmaceutical research companies are committed to fighting cancer. Biopharmaceutical companies have made tremendous progress in the fight against the more than 200 diseases we call cancer. Continued research has expanded our knowledge of how cancer develops and how to target medicines for specific cancer types — resulting in more effective therapies for patients. Biopharmaceutical companies are developing 1,600 medicines and vaccines for cancer, all of which are in clinical trials or awaiting review by the U.S. Food and Drug Administration (FDA). Thanks to innovation from America’s biopharmaceutical research companies, the cancer death rate has fallen 33% from its peak in 1991, in large part due to innovations from targeted therapies along with immunotherapy. There are now a total of six CAR-T therapies approved by the FDA to treat a wide range of cancers — including pediatric cancer. The first of these did not receive FDA approval until 2017.

The Biopharmaceutical Pipeline

After decades of research to advance cancer progress, an average of 68% of medicines in the oncology pipeline today are likely to be first-in-class, meaning they use a new and unique mechanism for treating a disease. The pipeline is also ripe with innovative therapeutic approaches, like mRNA, with the potential to transform a wide range of cancers — many which have already seen approvals in recent years. For example:

• Immunotherapies, which include monoclonal antibodies and CAR-T, is an approach that works by unleashing the immune system to target and kill cancer cells.
• Gene editing involves manipulation of DNA at particular locations in order to treat a specific cancer.
• Oncolytic viral therapies work by zeroing in on cancer cells, to replicate and cause them to rupture.
• Antibody Drug Conjugates target specific cancer cells with cytotoxic agents without harming normal cells.

The Importance of the Biopharmaceutical Ecosystem

Our ability to treat, and in some cases even cure some of the most challenging diseases facing our country today like cancer, is built on the robust U.S. biomedical R&D ecosystem. This ecosystem has been sustained by a policy framework designed to support and advance America’s leadership in the innovation of new medicines, including strong intellectual property protections, a well-functioning, science-based regulatory system and coverage and payment policies that support and encourage medical innovation. Nearly 60% of oncology medicines approved a decade ago received additional indications for other types of cancer, but legislation enacted in 2022 — The Inflation Reduction Act — puts this kind of medical innovation in jeopardy by setting the price of medicines well before many of these critical advancements can be realized. This disincentives the investment necessary to conduct post-approval R&D and get approval for new uses for lifesaving treatments. The progress that we have seen in cancer in recent years and our ability to catalyze and accelerate future progress will be further impeded without public policies that reward and incentivize innovation.

Small and Large Molecules Medicines: Why We Need Both to Fight Cancer

Small molecule medicines, which represent the majority of cancer medicines, typically come in the form of a tablet or capsule, are taken by mouth and contain a single chemically synthesized active ingredient. Due to their size, small molecules can more easily reach therapeutic targets inside of cells, cross the blood-brain barrier, and are often available in oral dosage forms which offer greater flexibility and convenience in their administration and ultimately reduce barriers to treatment adherence and factors that can drive health disparities. For cancer specifically, targeted small molecule therapies can act upon specific proteins or genetic material inside cancer cells, causing cancer cells to die. Given cancer begins with genetic changes occurring inside cells, targeted small molecule medicines provide an essential tool in combating the cause of cancer.

Biologics, also referred to as large molecule medicines, in contrast are made by or from living cells, are structurally complex and are generally administered in a doctor’s office or hospital setting via injection or infusion. Due to their larger size, biologics are generally unable to enter cells, but rather are designed to reach therapeutic targets on the surface of cells.

Together, these two types of medicines offer patients and health care providers a wide choice of treatment options that are needed and are indispensable in the treatment arsenal against cancer.

Setbacks and Stepping Stones

Since 1998, the FDA has approved 111 medicines across the cancers, but there have been a total of 1315 unsuccessful investigational drugs. So-called “failures” are an inherent part of the process because treating human disease is one of the most complex undertakings on the planet. But these projects are not wasted efforts as their findings inform future study and direct research efforts toward new approaches to addressing the cancer causes, growth and progression.

Cancer continues to be a major challenge, but biopharmaceutical companies have over 1,600 potential cancer medicines in development and are dedicated to transforming cancer from a devastating diagnosis to a chronic, manageable condition. This goal drives researchers past the setbacks to discover how to apply the knowledge gained to inform the development of innovative medicines that bring hope to patients and their families and ultimately win the battle against cancer.

Immunotherapy Through the Years

Female technician working with equipment in a biopharmaceutical laboratory

The 1980s

1980s – Foundational Research Begins

Early 1980s

Scientists begin to research new ways to use T-cells and monoclonal antibodies to treat cancer. During the same period, the role of the T-cell receptor (TCR), a type of immune cell that recognizes and binds to foreign substances, is determined.

Mid 1980s

The first immune checkpoint molecule, cytotoxic T-lymphocyte antigen number 4 (CTLA-4), is discovered.

Late 1980s

First human testing and use of genetically engineered T-cells that can recognize and kill cancer cells.

The 1990s

1990s – Foundational Research Continues, Early Therapeutic Successes and Setbacks

Early 1990s

First tumor-specific antigen, the melanoma antigen gene (MAGE), discovered by melanoma researchers in Belgium in 1991, opens up new ways to use tumor antigens to stimulate the immune system to better fight cancer cells. A second immune checkpoint protein, programmed cell death-1 (PD-1), is discovered by researchers at Kyoto University in Japan (1992).

Mid 1990s

The concept of modifying Chimeric antigen T-cells (CAR T-cells) is introduced but fails in initial 1990s clinical studies due to technical intricacies and knowledge gaps.

Late 1990s

The first mAbs for cancer—rituximab for non-Hodgkin’s lymphoma (1997) and trastuzumab for HER2 positive breast cancer (1998)—are approved by the FDA, and the first evidence that gene-expression profiling can distinguish between cancer types is published (1999).

The 2000s

2000s – New Targets Identified and Medicines Developed

2000

Clinical trials launched to test the first immune checkpoint inhibitor drug containing a mAb targeted against CTLA-4 (ipilimumab for melanoma).

2001

Two separate in-vivo studies show that certain tumor cells are destroyed by natural killer (NK) cells—a type of white blood cell that has small particles with enzymes that can kill tumor cells or cells infected with a virus—establishing a new mechanism for how NK cells recognize tumor cells and laying the groundwork for them to become key components of multipronged therapeutic strategies for cancer.

2004

More mAb treatments (including cetuximab and avastin for metastatic colorectal cancer) are approved by the FDA.

2008

First PD-1 targeted immune checkpoint inhibitor enters Phase I trials.

The 2010s

2010s – Novel Immunotherapies Reach Patients

2010

FDA approves first therapeutic cancer vaccine, sipuleucel-T, for prostate cancer.

2011

FDA approves first checkpoint inhibitor targeting the CTLA-4 protein, ipilimumab, for metastatic melanoma. It is the first drug of any kind ever shown to extend survival in metastatic melanoma.

2014

FDA approves two more immune checkpoint inhibitors, pembrolizumab and nivolumab, both of which target the PD-1 pathway.

2015

FDA approves first oncolytic virus therapy, a new class of immunotherapies. Talimogene laherparepvec is approved for metastatic melanoma. It is a genetically engineered virus that has been tweaked to preferentially kill cancer cells.

2016

FDA approves fourth checkpoint inhibitor, atezolizumab, for bladder cancer. It targets the PD-1 pathway and is later approved for use in a total of six different cancers.

2017

FDA approves first CAR T-cell therapy, tisagenlecleucel, to treat adults with certain types of large B-cell lymphoma. FDA approves the fifth and sixth checkpoint inhibitors, which target the PD-1 pathway: avelumab, for Merkel cell carcinoma, and durvalumab, for bladder cancer, both of which are later approved for use in several additional cancers.

2018

FDA approves second CAR T-cell therapy, axicabtagene ciloleucel, for the treatment of adult patients with several types of large B-cell lymphoma, and a seventh checkpoint inhibitor, which targets the PD-1 pathway, cemiplimab, for cutaneous squamous cell carcinoma.

2020s – Treatment Options Expand, Research Flourishes

FDA approves the third CAR T-cell therapy, brexucabtagene autoleucel, for the treatment of patients with mantle cell lymphoma.

The 2020s

The Future

2021

FDA approves two more CAR T-cell therapies, lisocabtagene maraleucel, for large B-cell lymphoma, and idecabtagene vicleucel, for multiple myeloma.
FDA approves the eighth checkpoint inhibitor, dostarlimab, targeting PD-1, for patients with certain types of endometrial cancer.

2022

FDA approves the sixth CAR T-cell therapy, ciltacabtagene autoleucel, for multiple myeloma.

Featured Resources

Biopharmaceutical innovation and new drug discovery delivers far-reaching benefits to patients, the U.S. health care system and our state and national economies. New cancer medicines discovered and developed by America’s biopharmaceutical research companies are helping patients lead longer, more productive lives, controlling health care costs and stimulating the economy through high-quality jobs and a healthier workforce.

Updated as of December 2023.