Pipeline Session: Specialty Pharmaceuticals in Development

The dynamic landscape of healthcare and managed care pharmacy will be deeply impacted by new and emerging specialty medications. The ever-spiraling costs of specialty medications have led many experts to question whether these treatments translate into true improvements in health outcomes or patients’ quality of life. However, there are a number of promising agents in the specialty drug pipeline that will play a role in therapy treatments, as well as impact the managed care market.

A specialty drug is one that:

• Requires frequent dosing adjustments and intensive clinical monitoring
• Has intensive patient training or compliance assistance
• Has limited distribution
• Has specialized handling and administration
• Costs more than $500 a month.

While <1% of patients use specialty drugs, they represents one-fourth of drug spending.

Aimee Tharaldson, PharmD, senior clinical consultant, Express Scripts Holding Company, says there are 3 key specialty market trends for pharmaceuticals: increased competition, rare/orphan drug conditions, and more oral options. Dr Tharaldson suggests there will be an $11.3 billion specialty generic opportunity through 2017 as 38 specialty products’ patents reach expiration, and a $31.8 billion biosimilar opportunity as 51 biologic products reach patent expirations through 2020. Currently, there has also been much incentive for extensive orphan drug development. With 25 million people diagnosed with orphan diseases, the growth rate for orphan drugs is expected to be >25%, while non-orphan drugs are projected at only 20%. The specialty pipeline for orphan drugs shows that they account for 42% of the drugs in development—surpassing the 18% of non-orphan cancer specialty drugs, 16% of inflammatory condition specialty drugs, and far more than any other specialty drugs used to treat hepatitis C, multiple sclerosis (MS), or other orphan diseases.

Dr Tharaldson provided some recent 2013 select orphan drug approvals, which included Ravicti (glycerol phenylbutyrate—Hyperion; oral solution; alternative to Buphenyl) for urea cycle disorders, and Procysbi (cysteamine bitartrate&mdash;Raptor; oral; alternative to Cystagon) for nephropathic cystinosis. Select 2013 approvals in cancer included Kadcyla (trastuzumab emtansine&mdash;Genentech; IV infusion; antibody‐drug conjugate) which is indicated for use in HER2+ advanced/metastatic breast cancer, and Tafinlar/Mekinist (dabrafenib/trametinib — GlaxoSmithKline; oral; BRAF/MEK kinase inhibitor) which is indicated for BRAF V600+ advanced/metastatic melanoma.

In the specialty pipeline, Dr Tharaldson said there are many therapy class drugs that continue to mature. For example, current MS treatment options include immunomodulators like Avonex (interferon beta‐1a), monoclonal antibodies like Tysabri (natalizumab), and oral therapies like Aubagio (teriflunomide). Pipeline trends suggest MS immunomodulators will improve and go generic (ie, Copaxone; glatiramer acetate—Teva), there will be further monoclonal antibody drug development (ie, Lemtrada; alemtuzumab&mdash;Genzyme/Bayer), and there will be increased competition among oral drugs.

Other emerging specialty pipeline therapy class treatments covered by Dr Tharaldson included those for inflammatory conditions, cancer, HIV, hepatitis C, cystic fibrosis, pulmonary arterial hypertension, and bleeding disorders. Some of those pharmaceuticals include blockbuster drugs like Ibrutinib (Pharmacyclics) for leukemia and MCL.