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. Thanks in large part to innovation from America’s biopharmaceutical research companies, the five-year survival rate for all cancers has hit an all-time high of 70%. Improved survival in many forms of cancer has been attributed to treatment advances driven by targeted therapies immunotherapies.
Small and Large Molecule Medicines: Why We Need Both to Fight Cancer
Small molecule medicines, which represent the majority of cancer medicines today, typically come in the form of a tablet or capsule, are taken by mouth and contain an active therapeutic compound that can be made in a chemistry lab. Due to their size, small molecules can more easily reach therapeutic targets inside of cells, crossing the blood-brain barrier. They are also often available in oral dosage forms which offer greater flexibility and convenience 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. Since cancer starts due to genetic changes inside cells, targeted small molecule medicines provide an essential tool in combating cancer where it originates.
Biologics, also referred to as large molecule medicines, 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 or other molecules that interact with 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.
40 Years of Cancer Immunotherapy Research
Immunotherapy is a form of cancer treatment that harnesses the natural strength of a body’s immune system to prevent, control and — in some cases — even eliminate cancer. It has revolutionized the field of oncology and shifted the paradigm of cancer care, delivering significant improvements in survival and side effects for numerous patients across a wide range of cancers.
40 year immunotherapy preview
Fact Sheet
Revolutionizing Cancer Care with Immunotherapy
Revolutionizing Cancer Care with Immunotherapy
CAR-T Therapy Revolutionizing Cancer Treatment
Immunotherapy Through the Years
The 1980s
1980s – Foundational Research Begins
Early 1980s
- Scientists begin to research new ways to use T-cells and monoclonal antibodies (mAbs) 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.
Scientist injecting liquid into vile
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).
Nurse showing something to patient on tablet
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.
Woman looking at prescription pill bottle
The 2010s
2010s – Novel Immunotherapies Reach Patients
2010
- The FDA approves first therapeutic cancer vaccine, sipuleucel-T, for prostate cancer.
2011
- The 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
- The FDA approves two more immune checkpoint inhibitors, pembrolizumab and nivolumab, both of which target the PD-1 pathway.
2015
- The 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
- The 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
- The 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
- The 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.
Group of 3 people working together in meeting room
The 2020s
2020s
- 2022: The FDA approves the first gene therapy to treat cancer, indicated for the treatment of a severe type of bladder cancer.
- 2024: The FDA approves the first cell therapy to treat a solid tumor, indicated for the treatment of a type of metastatic melanoma.
- 2026: The FDA approves the first inhibitor of the RAS GTPase family, nearly doubling the overall survival rate for patients with advanced pancreatic cancer compared to those who received standard chemotherapy.
The industry’s innovation, along with the FDA’s regulatory guidance and review, continues to expand the arsenal against cancer. Cutting-edge research and development adds new treatment options for patients year after year.
Liquid sample on tray
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.
- Radioligand therapies work by pairing a tumor-targeting molecule with a radioactive payload, allowing radiation to be delivered precisely where it is needed.
The Role of Post-approval Research and Development
For many diseases, some of the most successful developments happen after a medicine is already approved. Post-approval research and development (R&D) is particularly important in cancer as much of the progress seen over the past decade has been due to additional investments in clinical research to seek approval for new uses of medicines in different types of cancer or patient populations.
The critical role of post-approval R&D in cancer is in part due to the nature of cancer itself, the practical manner in which it is researched, and how scientific evidence accumulates over time. This process often reveals a better understanding of the disease pathways shared by different cancer types to inform potential new uses of medicines in different forms of cancer. As a result of these shared pathways, oncology medicines are increasingly developed to treat multiple cancer types. Likewise, researchers typically begin cancer R&D in narrowly defined patient populations with advanced stages of cancer or that have exhausted other treatment options. Then, once a medicine has been shown to be safe and effective in these populations, researchers often seek to introduce the medicine in earlier stages of the disease where a new treatment is more likely to significantly modify the disease course and lead to better outcomes.
Among oncology medicines approved from 2000-2021, more than half of all indications approved and approximately two-thirds of industry-funded clinical trials occurred post-approval. Additionally, 61% of cancer treatment approvals that fight solid tumors before they spread were developed after the treatment’s initial approval. Post-approval research and development delivers real results for patients, and it’s important we protect the policies that support innovation and reject those that threaten it.
Looking Ahead
America’s innovative biopharmaceutical companies remain deeply committed to fighting cancer, driven by decades of progress that have transformed once‑fatal diagnoses into treatable conditions. Continued research is expanding our understanding of how cancer develops and enabling more precise, targeted therapies—contributing to a 34 percent decline in cancer mortality since its peak in 1991. With more than 1,600 cancer medicines and vaccines in development, the industry is steadfast in its mission to bring forward the next generation of treatments and improve survival and quality of life for every patient facing the disease.
Related Resources
America's cancer research progress is real and worth protecting
National Cancer Research Month is a reminder that smart policies and sustained investment have made America the global leader in cancer research. Maintaining this important leadership is not guaranteed.
Why it matters: The numbers behind America's cancer fight tell a story of accelerating progress, the kind that only happens in an ecosystem of smart policies, long-term investment and consistent scientific commitment.
- The five-year survival rate for all cancers has hit an all-time high of 70% for patients diagnosed between 2015 and 2021, according to the American Cancer Society.
- Cancer mortality has fallen 34% since peaking in 1991, saving an estimated 4.8 million lives.
- Most oncology R&D happens after initial approval, highlighting the importance of continued investment in this research to advance treatments for American patients. For cancer medicines approved between 2000 and 2021, more than half of new uses approved and two-thirds of industry-funded trials occurred post-approval.
- Patients in the United States have access to 88% of new cancer medicines versus just 36% in other high-income countries, who wait 3 years longer on average.
That progress didn't happen by accident. It reflects decades of R&D investment, a strong research ecosystem and policies that reward scientific risk-taking.
The big picture: Some of the most transformative progress is happening at the frontier of cell and gene therapies, treatments that don't just manage disease but target its root causes.
- Nine cell and gene therapies are now approved for cancer in the U.S., with more than 438 more in active development.
- CAR T-cell therapy—first approved in 2017—represent an effective treatment option in cancer care, with patient outcomes data indicating these drugs can functionally cure advanced cancers in certain cases.
Between the lines: PhRMA member companies are backing this progress with billions in U.S. manufacturing and R&D investment, bringing next-generation cancer treatments to American patients. America's leadership in cancer innovation isn't a guarantee. It's the result of smart policy choices that must be maintained and strengthened.
Government price controls and short-sighted reimbursement policies limit access to new medicines and chill the investment needed to fund the next generation of breakthroughs including post-approval innovations. At a moment when oncology science is advancing faster than ever, the wrong policy environment could stall progress in the pipeline.
The bottom line: When America leads in medical innovation, American cancer patients benefit first. Protecting the policies that make that possible isn't abstract, it's what keeps cancer survival rates growing higher.
Learn more at PhRMA.org/Cancer.
Drew Voytal
Future of Medicine: Cell & Gene Therapies
Cell and gene therapies address the root causes of disease, offering the potential to change or halt its course rather than simply managing symptoms. These breakthroughs, along with hundreds of promising treatments in development, signal a new frontier for treating cancers, rare genetic conditions, neurological disorders and other debilitating diseases. Continuing to foster U.S. innovation of cell and gene therapies is critical to ensuring American patients have access to these life-changing medicines.
Research and Development,Cancer,Cell and Gene Therapy,Future of Medicine
Report
Emerging Value in Oncology
Research and development of cancer medicines after their initial FDA approval can help expand treatment populations, find new ways of treating a cancer or help patients earlier in their cancer battle. Unfortunately, provisions in the Inflation Reduction Act put this progress in jeopardy by selecting medicines for price setting before many of these critical advancements can be fully realized.
Emerging Value in Oncology image
Cancer
Report
The Inflation Reduction Act Discourages Vital R&D in Cancer Medicines
New research from the Partnership for Health Analytics and Research (PHAR) demonstrates how price setting provisions in the IRA could have an acute impact on cancer medicines.
Read a Summary on Cancer Impact
The Inflation Reduction Act Discourages Vital R&D in Cancer Medicines image
DOWNLOAD THE FACT SHEET ON CANCER IMPACT
Research and Development
Fact Sheet
Chart Pack Cancer Medicines Value in Context
Chart Pack: Cancer Medicines Value in Context
To download the full report, click here.
This chart pack provides facts and figures about the role innovative treatments play in the fight against cancer.
Data and information found in this publication were drawn from a wide range of sources, including government agency reports, peer-reviewed journals, and the Pharmaceutical Research and Manufacturers of America’s (PhRMA’s) own research and analysis. PhRMA hopes this publication provides useful context for discussions about the value of cancer medicine.
Chart Pack: Cancer Medicines Value in Context image
Advances in Treatment
Report