Specialty Pharmacy Pipeline Session Highlights Upcoming Approvals

During a session at AMCP Nexus 2017, Amiee Tharaldson, PharmD, senior clinical consultant of emerging therapeutics at Express Scripts, outline the upcoming specialty pharmacy pipeline drugs.

Dr Tharaldson explained that specialty drugs are specifically drugs designed to treat complex conditions, with administration often requiring dosing adjustments, intensive clinical monitoring, patient training, and specialized handling. 

She outlined some trends in the specialty drug market, and highlighted the fact that competition is increasing as approvals accelerated significantly in 2017, relative to 2016. 

“We are seeing more medications approved in certain therapy classes, ranging from orphan conditions, cancer conditions, and even more common specialty therapy classes,” Dr Tharaldson said during her presentation. “This allows for more cost-savings opportunities.” 

Trends in Biosimilars

Dr Tharaldson also highlighted that increased competition amongst biosimilars will also create cost-savings opportunities, with a potential for $46.2 billion in savings through 2021, due to patent expirations. 

However, according to the presentation, some of the biosimilars already approved in 2016 and 2017 will not impact the market for years due to patent protections. For example, Erelzi (etanercept‐szzs)—a biosimilar to Amgen’s Enbrel for inflammatory conditions—could potentially not hit the market until 2029, despite being approved on August 30, 2016. 

Dr Tharaldson did mention that there are two biosimilar products that will receive approval in the remainder of 2017. The first is trastuzumab (Mylan), a biosimilar to Genentech’s Herceptin for the treatment of breast cancer, which could be approved on December 3, 2017. The second is Grastofil (filgrastim; Apotex/Intas), a biosimilar to Amgen’s Neupogen for the treatment of Neutropenia, which is expected to be approved before 2018. Dr Tharaldson noted that these two drugs are not anticipated to be held up by patent issues, with Grastofil expected to launch in 2017 and trastuzumab in 2019.

She also highlighted the seven biosimilar products that are pending approval in 2018. Most notably, Retacrit (epoetin alfa; Pfizer) a biosimilar of Amgen’s Epogen for treatment of anemia, which is expected to launch in quarter four of 2018.

Dr Tharaldson outlined some of the key points and challenges associated the impact of biosimilars on the market.

“These are really acting like competing brands, the price discounts that we are seeing right now is about 15% to 30%, and there is a lot of legal hurdles that need to be overcome before these products can be launched, “ she said. Dr Tharaldson also pointed to the confusing new naming guidelines and the need for more interchangeability approvals as other barriers to market impact. 

2017 Specialty Drugs

Dr Tharalson explained that specialty drugs make up 37% of all drug spending and within that space. Within the specialty drug pipeline, 17% of drugs are for oncology, 35% are for orphan drug conditions, and the remaining 48% are for other specialty conditions. 

“The trend for specialty is much higher than for traditional medications,” she said. “Specialty trend is going to outpace traditional for the foreseeable future an by 2020 it is expected that half of spend for the pharmacy benefit is going to be on specialty drugs—but it is important to remember that less than 2% of patients actually take specialty medications.”

The most spending is concentrated on drugs for inflammatory conditions, which make up $118 billion in spending annually—followed by oncology drugs, which account for $61 billion in annual spending. 

Among the most impactful approvals in 2017, Dr Tharaldson highlighted Dupixent (dupilumab; Sanofi/Regeneron) for atopic dermatitis, which has a potential patient population of 400,000 in the United States. Additionally, she noted that it could get an expanded indication that could impact spending even more.

“This is an important drug to keep an eye on,” she said. “It’s about $37,000 per year, but it can be used long term. It is [also] expected to get an expanded condition for severe uncontrolled asthma next year, and that population is about 1 million patients—so, this definitely going to expand in its use.”

From the data presented by Dr Tharaldson, there is a potential cost of $37 billion associated with the expanded indication for Dupixent. 

Dr Tharaldson also highlighted the approval of the first CAR-T gene therapy in the United States, Kymriah (tisagenlecleucel; Novartis) for the treatment of childhood leukemia. The drug is currently priced at $475,000 and is expected to receive expanded indications. 

Specialty Pipeline

In the remainder of 2017 through 2018, Dr Tharaldson said that she expects approvals to keep pace with the accelerated rate we’ve seen so far in 2017. She noted that oncology medications and orphan drugs will take up most of the drug spending for 2018. 

Of the new oncology medications coming to the market, there is a push toward oral therapies, with 40% of oncology drugs in the pipeline administered orally. She also highlighted another gene therapy that will be hitting the market soon, axicabtagene (Gilead) for the treatment of non‐Hodgkin lymphoma. 

Other conditions like HIV and NASH are expected to see a lot of approvals in the coming years; however, Dr Tharaldson stated that most manufacturers are abandoning their HCV drug development programs, as competition in this drug space has reduced the profitability and market share.

Other miscellaneous approvals that could have a significant impact include a gene therapy for
blindness— voretigene neparvovec (Spark Therapeutics) that could cost between $500,000 and $1 million, and a new DMD drug from PTC Therapeutics that could be more effective than Exondys 51.
 —David Costill