MedAdNews spoke to Bill Dreitlein, Senior Director, Pipeline & Drug Surveillance at OptumRx, about the state of the industry’s prescription drug pipeline and what enticing prospects will soon emerge.
MedAdNews: Please discuss some of the notable drugs that are currently being reviewed by the FDA and what is their anticipated impact on the market?
Bill Dreitlein: In our Q1 2020 Pipeline Report, we look at three drugs that will have a significant impact on the health care system that are approved or are expected to be approved by the FDA in the months ahead. We identified Palforzia (AR101), bempedoic acid and obeticholic acid because of the unique way they work and the potential high costs they will likely bring to the health care system.
First-of-its-Kind Treatment for Peanut Allergy: On Friday, January 31, the FDA approved AR101, the first oral immunotherapy to reduce allergic reactions from exposure to peanuts. Because of the large patient population – an estimated 6 to 15 million children experience allergic symptoms from peanuts – and minimal existing treatment options, there is a $4.5 billion global market for drug manufacturers to treat peanut allergies.
A New High-Cost Class of Drugs for High Cholesterol: The second drug, bempedoic acid, if approved, will be the first in a new class of drugs for the treatment of high cholesterol, which affects more than 37 percent of Americans. Bempedoic acid is notable because it presents an alternative form of treatment for patients who may have failed prior treatments. It can also be taken orally which may be more convenient than injectable PCSK9 inhibitors.
A Rare Disease Drug Targeting More Common Condition (Fatty Liver Disease): Obeticholic acid, while already on the market for the treatment of rare disease biliary cirrhosis, is nearing approval for the treatment of nonalcoholic steatohepatitis, or NASH, a liver condition affecting 16 million American adults with no available treatment. Given the large and growing target patient population in conjunction with the high unmet treatment need, it is projected that pricing for obeticholic acid for NASH will be between $10,000 to $18,000 per patient per year.
MedAdNews: Will 2020 be a record year for FDA drug approvals?
Bill Dreitlein: Looking ahead to 2020, 64 drugs have been filed with the FDA (majority of which are novel drugs) and are expected to be approved this year. Comparatively, 48 novel drugs were approved by the FDA in 2019 and 59 were approved in 2018.
MedAdNews: What are some of the R&D trends in the oncology space?
Bill Dreitlein: A continuation from past years, oncology is a top therapy class indicating that pharmaceutical manufacturers continue to invest heavily in this space. One notable trend within oncology is that roughly 70% of anticipated approvals in 2020 will be high cost orphan drugs, with new drug developments primarily focused on very narrow populations with rare subsets of cancer defined by a biomarker or genetic mutation. In these populations, trial sizes are often small and use surrogate endpoints, making it challenging to assess the overall value of the drug. Although the impact of such drugs has historically been small, the continued growth in the number of orphan indications and products could lead to substantial growth in the overall cost of care.
MedAdNews: What are some of the exciting potential new treatments in the field of neurology?
Bill Dreitlein: Treatments for neurological conditions are second on the list of top drug categories outlined in our report, and the pipeline of products treat a wide array of conditions within that class. This includes new therapies for conditions such as migraine, Parkinson’s disease, and epilepsy, to novel therapies that treat rare or ultra-rare conditions such as spinal muscular atrophy and neuromyelitis optica.
One drug in this category to watch is risdiplam, an investigational oral liquid medicine used to treat spinal muscular atrophy (SMA). With an anticipated approval date of May 2020, risdiplam will be the first oral treatment for patients affected by SMA, offering an alternative treatment option that is administered at home.
MedAdNews: Anything else you would like to add?
Bill Dreitlein: Specialty pharmacy care management will play a critical role in ensuring these drugs are being used appropriately, reducing waste, improving safety, and improving health outcomes for these patients. Health plans and clients should work with their pharmacy benefit managers to monitor the drug pipeline and assess where these drugs fit within the market.