Novel treatments and vaccines approved for U.S. patients in 2023 reach five-year high

2023 marked a five-year high for the number of new treatments and vaccines developed by the biopharmaceutical industry and approved by the U.S. Food and Drug Administration (FDA). According to new information from the FDA’s Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER), 71 novel treatments and vaccines were approved last year for people facing various cancers, certain rare diseases and more. Each new treatment represents new hope and progress for patients and their loved ones.

Some of the key milestones patients and biopharmaceutical researchers celebrated in 2023 include: 

• Significant progress for people with cancer. Biopharmaceutical companies have made tremendous progress in the fight against more than 200 different types of cancer. The FDA approved 13 new cancer medicines, including for blood, breast, colorectal and lung cancers.
  
  
• New treatments for rare and hard-to-treat diseases. In a historic year for patients suffering from sickle cell disease, two cell-based gene therapies were approved by the FDA in 2023. Additionally, this past year saw new treatments, including gene therapies, for hemophilia A and Duchenne’s muscular dystrophy. First-time treatments for progressive and debilitating genetic conditions commonly affecting children, such as Fredreich’s ataxia, Rett syndrome and many other were also approved.
  
  
• Advances for those suffering from neurological and degenerative disorders. Nine new treatments targeting various neurological diseases were approved, including one for Amyotrophic Lateral Sclerosis (ALS) and the first treatment shown to slow the progression of Alzheimer’s disease.
  
  
• Meeting unmet medical need in mental health. The first oral medication specifically for the treatment of postpartum depression was approved, providing another treatment option for mothers suffering from this debilitating condition.
  
  
• Treatments and vaccines against deadly infectious diseases. Critical options for vulnerable at-risk populations against a wide range of infectious illnesses became available this past year. These include the first-ever vaccines and treatments to protect newborns and older adults from the risk of respiratory syncytial virus (RSV), vaccines to protect teens against meningococcal disease and those at risk of exposure to a mosquito borne illness.  

2023 was a banner year for patients and biopharmaceutical innovation. With more than 8,000 medicines currently in the development pipeline, our industry will continue to push to develop new treatments and preventative medicines to improve health and save lives. This work is supported in part by the Prescription Drug User Fee Act (PDUFA VII), which plays a critical role in ensuring the FDA’s ability to review human drug applications. PDUFA also helps support the regulatory predictability that biopharmaceutical companies need to invest in the research and development of new medicines.

However, the PDUFA program is only one piece of the larger policy and regulatory ecosystem needed to promote and protect biopharmaceutical innovation for patients. Unfortunately, today’s policy environment — including the Inflation Reduction Act — does not support biopharmaceutical innovation and risks creating uncertainty that could jeopardize the development of the new, life-changing therapies patients are counting on. With so many patients still lacking viable treatment options, it is imperative that U.S. policymakers also prioritize policies that help ensure the innovative drug development process continues and patients can access the medicines they need in 2024 and beyond.