The biopharmaceutical pipeline contains thousands of innovative new treatments that have the potential to address unmet medical needs, save lives and improve patients’ health. A new report by the Analysis Group, “Innovation in the Biopharmaceutical Pipeline,” examines the drug development pipeline and shines a light on the innovations researchers are currently pursuing
Many of the new and life-saving medicines in the biopharmaceutical pipeline provide the hope for a potential future treatment option for patients who have not found success with existing therapies. With many of these medicines and scientific approaches targeting rare disease, cancers, cardiovascular disease, diabetes, Alzheimer’s disease, ALS, and HIV, advances in the pipeline can offer patients a renewed sense of hope for the chance to live fuller, longer lives.
Key findings from the report, include:
- Nearly 70% of medicines in clinical development are potentially first-in-class medicines, meaning they represent a unique pharmacological class distinct from other marketed products and would offer a new option for patients who may currently have no existing treatment options.
- 1,135 projects in clinical development received orphan drug designation by the U.S. Food and Drug Administration (FDA), which is critically important for patients with rare disease, given less than 10% of rare diseases have an approved medicine.
- A range of innovative scientific approaches are being pursued, including cell and gene therapies, DNA and RNA therapeutics and monoclonal antibodies.
Across the clinical development pipeline, first-in-class medicines account for 69% of projects. This is a tremendous milestone as first-in-class medicines use a different mechanism of action than any other approved medicine and can expand treatment options for patients by potentially providing greater efficacy, improved delivery and/or fewer side effects.
According to the National Institutes of Health (NIH), there are roughly 7,000 rare diseases, which when considered together, affect approximately 30 million people in the U.S. Roughly 95% of these diseases have no approved medicines and more than 85% are serious or life threatening. Orphan drugs are medicines intended to treat these rare diseases and there are over 1,100 projects in clinical development across several disease states, including cardiovascular disease, cancer and neurology, that received orphan drug designation by the FDA.
Alongside first-in-class medicines and orphan drugs, new scientific discoveries and approaches are leading the way toward newer, better treatments. According to the report, scientific advancements have led to several innovative strategies that researchers are using to fight disease including 545 cell therapy projects, 281 gene therapy projects, 348 CAR-T cell therapy projects, 265 DNA or RNA therapeutic projects and 133 Oncolytic Virus projects, therapies that would “retrain” virus to attack and kill cancer cells.
The benefits of the strong biopharmaceutical pipeline extend beyond patients, and positively impact the United States’ health care system, and society as a whole. New innovations and medical advancements contribute to reduced hospitalizations, surgeries, health care costs and the support of more than 4 million jobs across sectors. Despite the challenging research and development (R&D) process, biopharmaceutical companies continue to stay committed to bringing new life-saving medicines to market. Companies are also taking the lessons learned from COVID and applying them in the appropriate ways across the drug development pipeline to bring patients the treatment options they need.
Over the last decade alone, biopharmaceutical companies have invested over half a trillion dollars in R&D and more than 600 new medicines have been approved by the FDA. The high proportion of projects that have the potential to be first-in-class reflects growing scientific opportunities and demonstrates this commitment to innovation.
Today’s pipeline of new medicines holds incredible promise for patients, and it is important we ensure that policy and regulatory frameworks evolve to match the rapid pace of biopharmaceutical innovation so patients and their families can realize the promise of the pipeline. Policy frameworks must include strong intellectual property protections and a market-based delivery system that promotes access, all while continuing to foster innovation. Recent hyper-partisan drug pricing plans are a detriment to patients and the future of medical research, by ignoring the inherent challenges of bringing a new drug to market and undervaluing innovation. Government price setting will decimate the competitive ecosystem in the United States that has brought hope to so many Americans in the form of new medical advances. No matter what they call it, this plan will result in the same outcome: negative consequences for the patients with the most need.
To read the full report and find out more on the biopharmaceutical pipeline, click here.
Abigail Lore
Abigail Lore, MPA, is Director of Policy and Research at PhRMA. At PhRMA she is responsible for developing educational materials and research studies on a range of issues impacting innovative biopharmaceutical companies including FDA policy issues, the R&D process, and emerging technologies such as digital health tools and cell and gene therapies. Before joining PhRMA in 2019, she worked at a patient advocacy organization as a project manager. Abigail came to Washington, DC via New Hampshire to attend American University, and fell in love with the city including the running and biking trails, endless restaurants to try and the DC sports teams. Abigail has seen the first hand benefits of biopharmaceutical research and is passionate about advocating for a strong STEM workforce as well as the patients they work to help.
Abigail Lore
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New report details the promise of the biopharmaceutical pipeline
Research and Development
New report details the promise of the biopharmaceutical pipeline
New report details the promise of the biopharmaceutical pipeline