Breakthrough Therapies, Opportunities for Savings in Near-term Specialty Pipeline


As more competition makes its way into the specialty drug market, there will be opportunities for cost savings.

health costs

As more competition makes its way into the specialty drug market, there will be opportunities for cost savings for orphan conditions, cancer types, and even common specialty conditions, explained Aimee Tharaldson, PharmD, senior clinical consultant for emerging therapeutics at Express Scripts, who presented on the specialty pharmaceutical pipeline during her regular session at AMCP Nexus 2019.

There is a $34 billion market opportunity for first-time generics for specialty medications over the next 4 years as several specialty drugs go generic, she explained. There is expected to be generics for the multiple sclerosis drug Tecfidera, which has annual sales of about $3.5 million, beginning in September 2020. At the same time, the market will see first-time generics to Truvada, the pre-exposure prophylaxis for HIV, which has annual sales of about $3 billion.

Based on Express Scripts’ data, 45% of per-member per-year (PMPY) spend in the pharmacy benefit is for specialty medications. The specialty trend is higher than the traditional trend (+9.4% vs –5.8%), and by 2020 about half of all spend in the pharmacy benefit will be for pharmacy medications, Tharaldson said. And yet, less than 2% of patients take specialty drugs.

The leading specialty therapy classes are inflammatory conditions, which cost $174 PMPY and include Humira, Stelara, and Enbrel. The second most expensive specialty therapy class is cancer, which costs $80 PMPY; followed by multiple sclerosis (MS), which costs $56 PMPY; and HIV, costing $49 PMPY.

Related: AMCP NEXUS 2019 Street Team: Susan Trieu of MedImpact Healthcare Systems, Inc.


There were 17 novel cancer drug approvals in 2018, but only eight in 2019, which represented a big drop. Each year, there are 1.7 million new cases of cancer diagnosed and since 1991, the death rate has dropped 25%.

There are more than 100 types of cancer: breast cancer is the most common in women and prostate cancer is the most common in men. There are only 6 cancer drugs in the pipeline pending approval:

  • Tazemetostat from Epizyme is an enhancement of zeste homolog 2 inhibitor to treat metastatic or advanced epithelioid sarcoma. It has an expected approval date of January 23, 2020.

  • Avapritinib from Blueprint Medicines is a breakthrough therapy. The oral kinase inhibitor treats PDGFRA exon 18 mutation in gastrointestinal stromal tumors. It could also get a second indication for fourth-line use after other options.

  • Zanubrutinib from BeiGene is also a breakthrough therapy. The Bruton tyrosine kinase inhibitor would treat relapsed or refractory mantle cell lymphoma in patients who progressed on other therapies.

  • Enfortumab vedotin from Asetllas and Seattle Genetics is another breakthrough therapy. It is an antibody-drug conjugate targeting Nectin-4 to be used as a third-line treatment in advanced urothelial cancer. According to Tharaldson, the early data had a 44% overall response rate, although 12% of patients discontinued therapy due to adverse events.

  • Trastuzumab deruxtecan from AstraZeneca and Daiichi Sankyo is the fourth cancer breakthrough therapy pending approval for 2020. It is an antibody-drug conjugate targeting HER2 in patients with HER2-positive metastatic breast cancer who had been treated with at least two previous anti-HER2 therapies. It is also being looked at for other HER2 cancers, like gastric, lung, and colon cancers.

  • Isatuximab from Sanofi Genzyme is an anti-CD38 monoclonal antibody delivered as an intravenous (IV) infusion once weekly for 4 weeks and then every other week to patients with relapsed or refractory multiple myeloma (MM).

In addition to the therapies pending approval in 2020, Tharaldson also highlighted 12 therapies in the near-term pipeline. These therapies include another eight breakthrough therapies, two of which are chimeric antigen receptor (CAR) T-cell therapies: idecabtagene vicleucel from bluebird bio to treat multiple myeloma and lisocabtagene maraleucel from Celgene to treat diffuse large b-cell lymphoma (DLBCL).

Additionally, five of the near-term therapies are orphan drugs to treat non–small lung cancer, MM, small cell lung cancer, DLBCL, and cholangiocarcinoma.


In 2019, there were three new MS drugs approved. Two were approved in the first quarter of the year: siponimod (Mayzent) and cladribine (Maveclad). Both were approved for relapsing forms of MS, but they have different mechanisms of action. Siponimod is a sphingosine 1-phosphate (S1P) receptor modulator and cladribine is a purine antimetabolite. The third was approved the morning of Tharaldson’s talk. Diroximel fumarate (Vumerity) is also approved to treat relapsing MS, and it is an immunomodulatory drug.

Additional drugs in the pipeline, all to treat relapsing MS, include:

  • Ozanimod from Celgene, which has an expected approval data of March 25, 2020. It also targets S1P.

  • Monomethyl fumarate (Bafiertam) from Banner Life Sciences is another immunomodulatory drug. It has an expected approval date of June 20, 2020.

  • Ofatumumab from Novartis is an anti-CD20 monoclonal antibody with an expected approval date in 2020. While the other recently approved treatments and the treatments in the pipeline are oral therapies take once or twice daily, this would be a subcutaneous injection given once monthly.

  • Ponesimod from Janssen is another S1P receptor modulator with approval expected sometime in 2020.

Gene Therapies

There are more than 600 cell and gene therapies in the pipeline. These therapies modify a person’s genes to treat or cure disease either by replacing disease-causing genes, inactivating disease-causing genes, or introducing new or modified genes. Express Scripts expects that gene therapies market will reach more than $16 billion per year by 2024.

Currently there are only six approved: four immunotherapies (two of which are CAR T-cell therapies) and two gene replacement therapies. Another nine could be approved in the next year, including four cancer immunotherapies, three gene replacements for rare conditions, and two gene replacements for hemophilia.

There are two gene therapies in the pipeline for Duchenne muscular dystrophy, both with expected approval dates in 2021. One is from Pfizer and the other from Sarepta Therapeutics. Both would be one-time IV infusions. Pfizer’s is a mini-dystrophin gene therapy and Sarepta’s is a micro-dystrophin gene therapy.

There are seven hemophilia gene therapies in the pipeline: two have expected approval dates in 2020, while the rest have 2021 or later expected approval dates. Five of those therapies are for hemophilia A and the other two are for hemophilia B. All of the gene therapies are IV infusions. The two expected in 2020 are both gene replacement therapies.

Four of the gene therapies in the pipeline are for cancer: aglatimagene besadenovec for prostate cancer, nadofaragene firadenovec for bladder cancer, idecabtagene vicleucel for multiple myeloma, and lisocabtagene maraleucel DLBCL. They are all immunotherapies.

Finally, three of the therapies are for rare conditions: beta beglogene darolentivec, a gene replacement for beta thalassemia; eladocagene exuparvovec, a gene replacement for aromatic L-Amino Acid Decarboxylase deficiency; and elivaldogene tavalentivec, a gene replacement for cerebral adrenoleukodystrophy.

Laura Joszt is associate editorial director for Managed Healthcare Executive®.

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