A Robust Specialty Pipeline for Chronic and Orphan Diseases

Tampa—The specialty pharmacy market is going to see increased competition and a number of orphan drug and breakthrough therapy approvals in the near future, according to Aimee Tharaldson, PharmD, senior clinic consultant, emerging therapeutics department, Express Scripts, who spoke during a session at the AMCP meeting. The generic and biosimilar markets may also break out in the specialty realm, with $13.1 billion in specialty generic opportunities as 39 drugs go off patent through 2018 and $31.8 billion in biosimilar opportunities with 51 biologic patents expiring through 2020.

While Dr. Tharaldson expects there will be 5 more draft documents submitted for biosimilars by the end of 2014, she does not foresee biosimilar approvals for another 3 years. Orphan drugs, however, are proving to be a lucrative area for specialty pharmacy. Currently, 36% of the specialty pharmaceuticals in the pipeline are for orphan drugs, and the average annual cost of orphan drugs approved in 2013 was $185,000, said Dr. Tharaldson.

Breakthrough therapies, including new drugs and drug combinations to treat life-threatening diseases, will continue to come to market, as the FDA has received 133 requests for breakthrough therapies so far, 40 of which were granted by the FDA, and 3 have been approved to date: (1) sofosbuvir, which took 8 months to be approved; (2) ibrutinib, which took 4.5 months to be approved; and (3) obinutuzumab, which took 4.5 months to gain approval.

Currently in the specialty market, in terms of pharmacy drug spend, 28% is on specialty medications and 72% is on traditional medications. Dr. Tharaldson estimated that by the end of this decade, 50% of pharmacy drug spend will be for specialty drugs. So far this year, at the time of the presentation, the following specialty pharmaceuticals had been approved by the FDA: tasimelteon, elosulfase, droxidopa, metreleptin, and apremilast.

Dr. Tharaldson went into detail about the specialty pharmacy pipeline for the following disease states:

            • Inflammatory conditions

            • Multiple sclerosis (MS)

            • Cancer

            • Hepatitis C

            • HIV

            • Cystic fibrosis (CF)

            • Idiopathic pulmonary fibrosis

            • Other orphan diseases

For inflammatory conditions, Dr. Tharaldson noted that secukinumab, a subcutaneous medication indicated for psoriasis and expected to come to market in October 2014, is “a drug to keep an eye on.” And while apremilast was just recently approved for psoriatic arthritis, she expects that by September 2014, the drug will also be approved for psoriasis.

For MS, Dr. Tharaldson noted that a copaxone generic is expected in May. While there are a few oral medications for MS in trials now, it is still going to be some time before we see these come to market, with Dr. Tharaldson targeting those for 2016 or beyond.

Currently, one-third of the specialty drugs in the pipeline are for oncology. Dr. Tharaldson indicated that targeted therapies and immunotherapies may begin to play a bigger role, with some experts projecting that within the next 10 years, half of cancer patients will be treated with immunotherapy.

As recent drug approvals for hepatitis C have sparked controversy and conversation, Dr. Tharaldson noted that the standard of care for this disease state is rapidly evolving, and she expects the guidelines to continue to be updated frequently as more drugs come to market. With the controversy of the sofosbuvir price tag, Dr. Tharaldson said reports suggest the drug will reap $5 billion to $9 billion in revenue in 2014. There are a number of drugs in the pipeline for proteasome inhibitors, NS5a inhibitors, polymerase inhibitors, and next interferon-free regimens.

For HIV, we can expect to see a new booster, more single tablet regimens, and injectable therapies, although Dr. Tharaldson noted that it will be several years before injectables are approved for this disease state, potentially in 2017 and beyond. She also said that generic HIV medications would have no real impact on this market, so she does not expect to see many in the future.

For CF, there are medications in the pipeline that will treat the underlying disease, increase mucous clearance, and fight lung infection, in which 30% of CF patients face infections of the lungs. Dr. Tharaldson highlighted that the lumacaftor/ivacaftor combination that is expected to reach the market in early 2015 could be used in up to half of CF patients in the coming years.

Lastly, for idiopathic pulmonary fibrosis, there are currently no approved drugs to treat this class; however, there are drugs in the pipeline that are estimated to reach the market in 2015. Dr. Tharaldson expects to see multiple drugs with different mechanisms of action for this disease state, similar to the pulmonary arterial hypertension therapy options.

Other orphan drugs for the following disease states are also in the pipeline: hemophilia A, hemophilia B, gaucher disease, hypoparathyroidism, and hereditary angioedema.