Preview of the 2018 Pipeline Drugs Expected to Impact Formularies

With new approvals accelerating in 2017 under new, competition-focused leadership at the FDA, novel options for the treatment of many chronic and critical illnesses have expanded greatly. A total of 43 new drugs have been approved so far in 2017, and the announcement of new initiatives to accelerate approval of gene therapies and other critical illness treatments based on early clinical trial data indicates that next year’s approvals will continue at a similar speed.

As approvals continue to gain momentum in 2018, Formulary Frontlines will continue to cover the latest breaking formulary updates for a wide range of disease states impacting specialty pharmacy spending. Here is a look at a few of the conditions expert clinicians and formulary decision makers will be highlighting in the coming year.

Atopic Dermatitis

Formulary Frontlines will cover recent approvals for treatment of eczema or atopic dermatitis. According to a recent pipeline update session at AMCP Nexus 2017, atopic dermatitis represents a potential patient population of around 400,000 in the United States.

In March 2017, the FDA approved Dupixent (dupilumab; Sanofi/Regeneron), a subcutaneous treatment for moderate to severe atopic dermatitis. Dupixent could have a significant impact on spending, considering its $37,000 price tag and large patient population, which will have to stay on the drug long term in order for continued disease relief. However, according to clinical trials, only about 25% of patients on Dupixent meet the responder criteria.

Additionally, recent reports indicate that uptake of Dupixent is high among dermatologists, suggesting that the drug will likely have a significant spending impact in 2018.

Severe Migraines

Migraines effect a potential patient population of around 39 million Americans, and are 3 times more common among women than men. The condition is characterized by a severe throbbing headache that lasts between 4 hours and 72 hours.

Three drugs with promising clinical trial data are slated for approval in 2018 to treat severe migraines. Erenumab (Amgen/Novartis), a subcutaneous medication taken monthly, is expected to receive FDA approval on May 17, 2018. Recent data in the New England Journal of Medicine showed that the drug reduced the length of migraine attacks by half and significantly reduce the frequency of attacks.

Two similar drugs, fremanezumab (Teva) and galcanezumab (Eli Lilly), are also expected to be approved later in 2018. Analysts and migraine experts expect these drugs to be priced between $8,000 and $20,000 per patient per year when they hit the market next year.

Psoriasis

Psoriasis is a chronic, t-lymphocyte mediated inflammatory disease that effects approximately 2% to 3% of the US population. It is commonly characterized by silvery white erythemous plaques that vary in presentation.

In 2017, the FDA approved Siliq (brodalumab; Valeant) for the treatment of moderate to severe plaque psoriasis. The drug is an interleukin (IL)‐17 inhibitor that is injected every 2 weeks. Siliq carries a list price of $3500 per month, suggesting a significant impact on pharmacy spending. Likewise, the FDA approved Tremfya (guselkumab; Janssen) in the summer of 2017. Tremfya has an average list price of around $13,000; however, the drug is only administered every 8 weeks.

The 2018 pipeline contains two drugs for the treatment of psoriasis, including tildrakizumab (Sun Pharmaceuticals) and an expanded  indication of Cimzia (certolizumab; UCB). Tildrakizumab is an interleukin‐23 inhibitor that is administered every 12 weeks. Recent clinical trials showed that tildrakizumab was significantly effective compared to placebo and Enbrel (etanercept; Amgen).

Adult Attention Deficit/ Hyperactivity Disorder (ADHD)

According to the NIH, adult ADHD affects 8.1% of individuals in the United States with 41% of these cases considered severe. ADHD is characterized by difficulty staying focused and paying attention, difficulty controlling behavior, and hyperactivity.

In June 2017, the FDA approved Mydayis (mixed salts of a single-entity amphetamine product; Shire), a new, once-daily option for adolescent and adult ADHD. In 16 clinical trials, Mydayis improved attention scores with effects demonstrated within 2 to 4 hours of administration and lasting up to 16 hours.

In 2018, KemPharm Inc is expected to submit its experimental ADHD drug KP415 for FDA approval. KP415 uses KemPharm’s platform technology known as ligand activated therapy. The drug is an extended-release d-methylphenidate. Additionally, a controlled-release version of mazindol (NLS Pharma) is currently in late-stage testing and is expected to hit the market soon.

Looking Beyond

Innovative therapies for conditions in novel treatment areas are expected to impact spending and formularies beyond 2018. In 2019, elafibranor (Genfit) is expected to be the first drug to hit the market to treat nonalcoholic steatohepatitis (NASH). As a disease with limited treatment options, NASH management currently includes reducing weight, eating a healthy diet, exercising, avoiding alcohol, and treatment of concurrent conditions. However, there are currently more than 10 drugs to treat NASH in the specialty pharmacy pipeline. When combined with the potential patient population of between 6 and 16 million, these pipeline drugs suggest that NASH could significantly impact pharmacy spending in the years to come.

Additionally, gene therapies for cancer and other rare diseases will pack a spending punch that is expected to significantly alter the way drugs are paid for in the United States. Two gene therapies have been approved in 2017, both with huge price tags, and the approval of voretigene neparvovec (Spark Theraputics) to treat biallelic RPE65‐mediated inherited retinal blindness is expected in Q1 of 2018. Furthermore, the potential for expanded indications for a range of cancer types make gene therapy approvals worth keeping an eye on. 

—David Costill

For articles by First Report Managed Care, click here

To view the First Report Managed Care print issue, click here