2022 marked another year of significant progress by biopharmaceutical research companies conducting lifesaving research and development for innovative medicines with a total of 45 new medicine approvals by the U.S. Food and Drug Administration (FDA). According to a new report released by the FDA’s Center for Drug Evaluation and Research (CDER), 37 novel medicines were approved by CDER last year. The Center for Biologics Evaluation and Research (CBER) also approved 8 new medicines in 2022 — including several gene therapies treating two rare blood disorders and a devastating neurodegenerative disease impacting young boys. Collectively, these newly-approved novel medicines offer the potential to expand treatment options for patients suffering from many debilitating and hard-to-treat diseases, resulting in improved survival, better health outcomes, enhanced quality of life — and in many cases, they provide a first-time treatment option for patients.
These benefits are underscored in CDER’s New Drug Therapy Approvals 2022 report, which found 54% (20) of new medicines approved by CDER were first-in-class, meaning they employed new mechanisms of action different than those of existing therapies. Novel drug approvals last year included the first medicine to delay the onset of type 1 diabetes in patients eight years and older, a new treatment for amyotrophic lateral sclerosis (ALS), an antiviral for treatment-resistant HIV that can be delivered as infrequently as once every six months and 12 new therapies for a range of different cancers. Importantly, 68% (25) of the novel medicines approved by CDER were approved in the U.S. before any other country.
It is important to note that while the number of medicines approved by FDA each year can ebb and flow, the pipeline of innovative medicines is robust with more than 8,000 medicines in development around the world offering the potential to provide new treatments and cures for patients. Since 2000, nearly 900 medicines have been approved by the FDA with drug, biologic and vaccine products playing a central role in keeping patients healthy and reducing the need for costly medical care.
The recently enacted Prescription Drug User Fee Act (PDUFA) VII will further support biopharmaceutical innovation and science-based regulatory review of new medicines. First enacted in 1992, the PDUFA program plays a critical role in strengthening the FDA’s ability to review human drug applications. The PDUFA program also provides biopharmaceutical companies with greater regulatory predictability, which fosters industry investment in research and development. In part because of PDUFA, the U.S. leads the world in the introduction of innovative new medicines, and the FDA’s human drug review program is the global gold standard for regulatory review and approval.
Key findings from the CDER New Drug Therapy Approvals 2022 report are:
54% of Novel Medicines Approved by CDER were for Rare Diseases
Although rare diseases are those impacting fewer than 200,000 Americans, collectively they affect about 30 million — or roughly one in 10 Americans. But just less than 10% of known rare diseases have available treatments. Developing medicines to treat these diseases is particularly challenging due to the complexity of the diseases themselves and a limited understanding of the underlying causes and the clinical course of many of these illnesses. America’s biopharmaceutical researchers are leveraging new technologies and the growing understanding of the genetic basis for many rare diseases to develop groundbreaking therapies to meet this unmet medical need. In 2022, 54% (20) of CDER’s new medicines approvals were for rare diseases. These medicines include novel treatments for a life-threatening skin disease, a form of melanoma that develops in the eye, and a first-time treatment for seizures in patients 2 years and older with a brain dysfunction disorder driven by certain gene mutations. Despite significant odds where setbacks are more likely than success, biopharmaceutical researchers have made incredible progress over the past decade fighting rare diseases. In fact, today, there are more than 700 medicines in development to treat many of these devastating illnesses.
The Accelerated Approval Pathway Continues to Bring Earlier Treatment Options for Patients
In 2022, the FDA approved six novel medicines via the accelerated approval pathway. The accelerated approval pathway, created in 1992 at the urging of patient advocates, has been a lifeline for millions of patients. Primarily utilized for cancers, rare diseases and HIV/AIDS, the pathway allows patients earlier access to potentially lifesaving medicines — often when no other treatment is available and when patients are facing difficult odds — while still maintaining FDA’s high standards for safety and effectiveness. During the most recent year, approvals under the pathway provided critical treatments for patients with many rare forms of cancer. For example, a medicine for adults with follicular lymphoma whose cancer has relapsed or did not respond to previous treatment, and a medicine for adults with relapsed or refractory multiple myeloma who have received at least four treatment regimens, among others.
Post-Approval Research and Development Brought Additional Treatments to Patients
After initial FDA approval, scientists and physicians may continue to generate information on a new medicine. This work, referred to as post-approval research and development, increases treatment options for patients by demonstrating, for example, that an existing medicine can also treat a different disease or stage of disease, or that a medicine is safe and effective for use in children. Though these medicines are not accounted for in the 2022 CDER report’s tally for novel drug approvals, the report specifically highlights new uses of approved medicines as critical treatment advances, particularly for cancer patients, children and those impacted by rare diseases. In fact, many of these post-approval research advances brought first-time treatments for patients. For example:
- A new treatment for “HER2 low” breast cancer, which historically has had limited treatment options.
- A first-time treatment for chronic weight management in patients six years and older with obesity due to a rare genetic disorder known as Bardet-Biedl syndrome.
- A first-time treatment for pediatric patients with juvenile myelomonocytic leukemia, a rare blood cancer primarily impacting young children.
- Two treatments approved for use in combination for use in pediatric patients whose tumors express a BRAF mutation.
America’s biopharmaceutical research companies’ core mission is to research and develop treatments and cures improving the lives of patients around the world. PhRMA’s member companies continue to apply expertise and resources in support of this mission, including investing more than $1 trillion in research and development of new medicines since 2000. The novel therapies approved in 2022 highlight the innovative biopharmaceutical industry’s continued commitment to fighting the root causes of disease and meeting serious unmet patient needs.
Unfortunately, the price setting provisions in the Inflation Reduction Act (IRA) undercut industry R&D efforts, diminishing hope for the development and availability of future treatments and cures. In particular, the law discourages the development of small molecule medicines and critical innovation that takes place after a medicine is first approved. Companies are already making difficult decisions in the wake of the new law. Rather than advancing policies that may have a chilling effect on the biopharmaceutical industry’s ability to develop new treatments and cures, policymakers should work together to ensure that the promise of the biopharmaceutical research pipeline is able to be realized for patients, including mitigating the unintended consequences of the IRA on future biopharmaceutical innovation.