In January 2021, the United States (US) Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) published Advancing Health through Innovation: New Drug Therapy Approvals 2020. This report provides a summary of a number of approvals and highlights the novel therapies approved in 2020, continuing the generally upward trend in approval volume seen over the past decade, despite the impact of coronavirus disease 2019 (COVID-19). Compared to 2018 and 2019, in which CDER approved 59 and 48 new drugs, respectively, 53 novel agents were approved in 2020. This number does not include new and expanded uses of already approved drugs, new formulations, new dosage forms, vaccines, blood products, cellular or gene therapy, or biosimilar approvals. Once again, the number of approvals exceeded the average of 41 novel approvals per year in the past 10 years. Figure 1 outlines approvals over the past 10 years.

Despite the ongoing pandemic the FDA continued their strategic initiatives to expedite the safe review of treatments in 2020. With the unprecedented challenges incurred in 2020, the FDA acknowledged that maintaining their commitment to bringing forth innovative therapies was difficult. Remarkably, the numbers reported by the FDA do not include the several emergency use authorizations (EUAs) issued by the FDA for COVID-19.

Last year, all 53 novel drug approvals again met their Prescription Drug User Fee Act (PDUFA) goal dates, cementing this as a priority for the Agency. In 2020, 40% were considered first-in-class, and 58% were approved for rare diseases (Orphan Drugs), the latter of which increased from 44% in 2019. Priority Review was granted to 57% of novel drugs, 23% received Accelerated Approval, 42% were designated as Breakthrough Therapy (up from 27% in 2019), and 32% garnered Fast Track designation. Overall, 68% of all drug approvals in 2020 used expedited development and review methods. In addition, 92% were approved in the first review cycle, and 75% were approved in the US prior to receiving approval in other countries. A breakdown of the types of drugs approved in 2020 is illustrated in Figure 2, with agents within the oncology spectrum representing over one-third of 2020’s novel approvals.

The notable 2020 approvals encompassed new advances for the treatment of infectious diseases, including a new medication class for the treatment of human immunodeficiency virus-1 (HIV-1). Garnering perhaps the most attention, the FDA also approved the first medication for hospitalized patients with COVID-19. Unique infectious diseases in the US also received attention, with a new drug for malaria, two new options for the Ebola virus, and a new treatment for Chagas disease approved in 2020. In the neurology arena, there were multiple approvals of agents for more common conditions, such as migraine or Parkinson’s disease. Moreover, there were significant advances for rare neurological conditions, including the first oral agent for spinal muscular atrophy and new treatments for rare seizure disorders. In addition, two immunological agents were approved for the treatment of neuromyelitis optica spectrum disorder. Additional treatment options emerged for several autoimmune conditions in 2020 as well. Not surprisingly, numerous advances were made within the oncology umbrella, including both novel approvals and new or expanded indications for several existing agents. With over half of the novel approvals being classified as Orphan Drugs, in 2020, the FDA has fortified their dedication to providing innovative and often targeted treatment options for all individuals.

In January 2020, the United States (US) Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) published Advancing Health through Innovation: New Drug Therapy Approvals 2019. This report provides a summary of a number of approvals and highlights the “game-changers” approved in 2019, solidifying the upward trend in approval volume initially seen in 2017. Compared to 2017 and 2018, in which CDER approved 46 and 59 new drugs, respectively, 48 novel agents were approved in 2019. This number does not include new and expanded uses of already approved drugs, new formulations, new dosage forms, vaccines, blood products, cellular or gene therapy, or the 10 biosimilar approvals. This far exceeds the average of 38 novel approvals per year in the past 10 years. Figure 1 outlines approvals over the past 10 years.

In 2019, the FDA continued its strategic initiatives, with additional approvals of biosimilars and generics (both first-time agents and those off-patent with limited competition), as well as a focus on the expedited approval process, particularly for rare diseases and agents supported by positive patient-reported outcomes. Notably, the FDA hosted a public meeting in April 2019 to receive input on how to tackle barriers associated with the development of treatments for rare diseases, and held another meeting to celebrate “Rare Disease Day” in February 2020. Moreover, this trend is likely to continue, with the FDA releasing several guidances on gene therapy. The new pathway for insulin approval under biologic license applications planned for March 2020 and the launch of internet-based repositories or apps to improve access to information for both providers and patients (e.g., CURE ID in 2019, antiretrovirals in 2020) also highlight strategic initiatives of the FDA.

Last year, all 48 novel drug approvals met their Prescription Drug User Fee Act (PDUFA) goal dates, cementing this as a priority for the Agency. In 2019, 42% were considered first-in-class, and 44% were approved for rare diseases (Orphan Drugs). Priority Review was granted to 58% of novel drugs, 19% received Accelerated Approval (more than doubling the rate of 7% last year), 27% were designated as Breakthrough Therapy, and 35% garnered Fast Track designation. Overall, 60% of all drug approvals in 2019 used expedited development and review methods. In addition, 90% were approved in the first review cycle, and 69% were approved in the US prior to receiving approval in other countries. A breakdown of the types of drugs approved in 2019 is illustrated in Figure 2, with approvals once again dominated by drugs in the expansive oncology spectrum.

Some of the notable 2019 approvals included new advances for the treatment of cystic fibrosis (CF), with the first triple combination therapy and expanded indications of existing CF therapy available to younger patients, and the first new medications in several years for sickle cell disease. Additionally, several approvals targeted rare conditions, such as erythropoietic protoporphyria, neuromyelitis optica spectrum disorder, tenosynovial giant cell tumor, Duchenne muscular dystrophy, and systemic sclerosis-associated interstitial lung disease. Beyond treatments for rare disease and cancer, the FDA also approved agents for more common disorders, including multiple sclerosis, novel treatments for depression, migraine and cluster headaches, epilepsy, several autoimmune conditions, age expansions for diabetes therapy, and a drug-sparing regimen and new prophylaxis option for human immunodeficiency virus-1 (HIV-1). Although down slightly from 2018, the large number of approvals from 2019 represent a continued trend by the FDA to expedite and increase the quantity of annual approvals.

In January, the Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) published Advancing Health through Innovation: 2018 New Drug Therapy Approvals. This report provides a benchmark for approvals and highlights the game-changers approved in 2018. Compared to 2016 and 2017, in which CDER approved 22 and 46 new drugs, respectively, in 2018 a total of 59 novel agents were approved. This number does not even include new and expanded uses of already approved drugs, new formulations, new dosage forms, or the seven biosimilar approvals. This marks the largest number of novel approvals in over two decades and far exceeds the average of 33 novel approvals per year in the past 10 years, surpassing the record setting approvals in 2017. Figure 1 outlines approvals and filings over the past 10 years.

Several potential explanations may account for this uptake in approvals in the past couple years. The FDA’s leadership, with its continued strategic initiatives, may be partly responsible for the increase. In early 2017, the FDA was criticized following the lower than average approvals seen in 2016. Public interest in opioid abuse and dependence treatment, access to drugs for rare diseases, drug shortages, and competition potentially affecting pricing have also put the FDA at the forefront of the public’s mind, further challenging the FDA to demonstrate action in the public’s interest. Finally, additional defined methodology for evaluating data in rare diseases, such as real-world and patient reported outcomes, may also have played a role.

Last year, all 59 novel drug approvals met their Prescription Drug User Fee Act (PDUFA) goal dates. In 2018, 32% were considered first-in-class and 58% were approved for rare diseases (Orphan Drugs). Priority Review was granted to 73% of new drugs, 7% received Accelerated Approval, 24% were designated as Breakthrough Therapy, and 41% garnered Fast Track designation. Furthermore, 95% were approved in the first review cycle, and 71% were approved in the US prior to approval in other countries. A breakdown of the types of drugs approved in 2018 is illustrated in Figure 2, with drugs in the expansive oncology spectrum once again dominating the approvals.

Some of the notable 2018 approvals included the first non-opioid drug approved to reduce opioid withdrawal symptoms, a new antiretroviral for multidrug resistant human immunodeficiency virus-1, a new class of drugs for migraine (calcitonin gene-related peptide receptor antagonists), the first FDA-approved drug derived from marijuana, the first treatment approved for multiple sclerosis in children, expanded options for cystic fibrosis, and the first antibiotic approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs.

Among the several Orphan Drugs approvals were drugs for Fabry disease, phenylketonuria, X-linked hypophosphatemia, Lennox-Gastaut syndrome, Dravet syndrome, hemophagocytic lymphohistiocytosis, adenosine deaminase deficiency, and Lambert-Eaton myasthenic syndrome.

Although 2019 approvals have begun more slowly, given the substantial number of approvals in 2018, it appears that the large number of approvals from 2017 was not an anomaly but, perhaps, the continuation of a trend.