The Value of Cancer Treatment Today

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. More than 1,300 medicines and vaccines for various cancers are currently in development, either in clinical trials or awaiting review by the FDA. 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.

America’s Health Care Ecosystem Encourages Biopharmaceutical Efforts to Fight Cancer

The new era of medicine transforming 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 is 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. The progress that we have seen in cancer in recent years and our ability to catalyze and accelerate future progress will be impeded without public policies that reward and incentivize innovation.

Setbacks and Stepping Stones

Over the past 20 years, the FDA has approved 115 medicines across the cancers, but there have been a total of 1,366 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 1,300 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.

The eras in progress

Female technician working with equipment in a biopharmaceutical laboratory

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.
Older male patient with a concerned expression speaking with a health care provider

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).
Closeup of a woman's hands holding a medication bottle in one hand and a mobile device in another

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.
People working a glass-walled conference room

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.
Closeup of a pipette dropping liquid into a petri dish

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 February 28, 2022