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.

A major trend in the pharmacy space continues to be increasing specialty drug spend, which is expected to continue with the introduction of new specialty agents for oncology, autoimmune disorders and rare diseases. In this year’s Medical Pharmacy Trend Report Employer Group Supplement, we found that 88 percent of employers reported a medical benefit spend of less than $10 million, and a year-over-year drug trend between 1-20 percent. For the few employer groups with spend above $10 million, it was due to a higher number of lives, and may be assumed that the employee mix for these groups may have included those with more costly health expenditures.

The Employer Group Supplement assists employer groups and third-party administrators in determining specialty drug trends and strategies to solve complex challenges impacting the medical benefit drug landscape. Our goal is to expand the information shared with employer groups to create a more dynamic picture of specialty drug management and help employers make more effective healthcare decisions. Building an effective medical benefit drug management strategy requires an in-depth knowledge of and expertise in this complex area, but it’s essential to help employers rein in costs and improve the quality of care for members. It is our hope that the survey data presented in this report helps employer groups begin to think about and investigate escalating medical pharmacy costs.

Download the full report or listen to our webinar to learn more.

Medicine continues to evolve quickly, with new treatments and trials coming to market every month. As a doctor in practice, staying up-to-date on the newest innovations and school of thought can be overwhelming, particularly for rare diseases such as Duchenne muscular dystrophy or spinal muscular atrophy. What’s the role of a pharmacy benefit manager in cases like these?

As part of its value-based approach to pharmacy benefit management (PBM), Magellan Rx Management sees itself as an idea connector and conversation starter. Recently, a discussion forum connected doctors with two key opinion leaders to discuss the complexities and effective management strategies for Duchenne muscular dystrophy (DMD) and Spinal muscular atrophy (SMA).

• Duchenne muscular dystrophy is a genetic disorder characterized by progressive muscle degeneration and weakness. It is one of nine types of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Symptom onset is in early childhood, usually between ages three to five. The disease primarily affects boys, but in rare cases it can affect girls.* According to the UCLA Duchenne Muscular Dystrophy Research Center, DMD affects one in every 3,500 male births.

• Spinal muscular atrophy is a genetic disease affecting the part of the nervous system that controls voluntary muscle movement. SMA involves the loss of nerve cells called motor neuronsin the spinal cord and is classified as a motor neuron disease. The age at which SMA symptoms begin roughly correlates with the degree to which motor function is affected: The earlier the age of onset, the greater the impact on motor function.* For SMA, the disease affects one in every 6,000-10,000 births, according to the Orphanet Journal of Rare Diseases.

The physician KOLs provided their expertise and insight on a wide range of topics including review of the clinical evidence, appropriate patient selection, and a discussion of the treatment landscape.  Additionally, effective clinical polices, practical dosing considerations, and emerging therapeutics were explored in this robust webinar. Importantly, doctors who took part in this discussion were given the opportunity to ask the KOLs questions about DMD and SMA – providing an opportunity for open dialogue and discussion about these complex issues.

*Information supplied by www.mda.org