Thomas Reinke

Specialty pharmacy got its name, in part, because unique medications were approved for use in special situations — for rare diseases or for use only after failure of traditional therapy.

In some ways, health plans have created an impression that specialty biologics and high-cost medications are out of reach by limiting access to them through formulary design and utilization management policies. Unfortunately, such policies designed to ensure appropriate use and control costs may have damaged the image of health plans in the eyes of patients and physicians.

Effect of scientific discovery

The special status and limited role of specialty medications is changing. They are being used much earlier in the course of treatment for certain diseases. Drugs that were approved as second-line therapy are now receiving approval for new indications as first-line agents, and some drugs are receiving FDA approval for multiple conditions. Variations in the prescribing patterns and therapy regimens of individual physicians also add to the expanded use of specialty products.

For health plans, the implication is that they need to remain or become more diligent in managing these products. Some health plans, such as UnitedHealthcare, are stepping up efforts to keep pace with the expanded use of specialty drugs.

“I am seeing expanding use of biologics in certain disease states,” says Atheer Kaddis, PharmD, senior vice president at Diplomat Specialty Pharmacy. “In cancer, which is difficult to treat and where therapy varies greatly, we are seeing that drugs originally used as second-line therapy are being used much sooner. For example, this is happening with Afinitor and Tarceva, which were originally recommended as second-line therapies. We now see them used as first-line therapies.”

Atheer Kaddis, PharmD

Health plans need to better understand the course of treatment at the individual level, says Atheer Kaddis, PharmD, senior vice president at Diplomat Specialty Pharmacy.

In some cases, like the cancer drug Tarceva, the movement from second- to first-line therapy is the result of scientific and technologic advances. Tarceva is a tyrosine kinase inhibitor approved in 2004 for second-line treatment of patients with locally advanced or metastatic non-small-cell lung cancer (NSCLC) after failure of at least one chemotherapy regimen.

In it is original 2004 trials, epidermal growth factor receptor (EGFR) mutations correlated with response to Tarceva, but the exact details of this association were not known. Tarceva lengthened survival in the EGFR-positive subgroup and the subgroup whose EGFR status was unmeasured, but it did not appear to affect survival in the EGFR-negative subgroup.

The results and confidence levels for the various groups were wide and overlapped so that a survival benefit in the negative subgroup could not be excluded. Therefore, the drug was approved without any restriction for EGFR status.

Between 2005 and 2010, Tarceva received additional approvals for pancreatic cancer and second-line maintenance treatment for NSCLC that had not progressed after first-line chemotherapy.

Last May, Tarceva was approved for first-line treatment of patients with metastatic NSCLC with one of two EGFR mutations. This approval erased the second-line requirement in the 2004 approval. In the two covered mutations, Tarceva showed progression-free survival of 10.4 months compared with 5.4 months for patients who received chemotherapy.

Tarceva’s latest approval was made possible by advances in molecular diagnostics and the FDA’s process for the timely development and approval of companion diagnostic tests. The FDA’s first-line designation requires the use of a genetic test that was approved by the FDA specifically to identify the two mutations. Often in oncology, says Kaddis, “The advances in specialty agents cause them to be used first-line. In many cases, they have demonstrated benefits over the previous first-line therapies.”

Zytiga is another example of a specialty medication that trumped earlier drugs. It is an oral oncolytic approved in 2011 for treatment of patients with metastatic castration-resistant prostate cancer (mCRPC) after failure on the first-line agent, docetaxel.

Last December, following the release of results from a second clinical trial, Zytiga received approval as first-line therapy for mCRPC. In this study, Zytiga significantly extended overall survival and progression-free survival.

“This approval demonstrates the benefit of further evaluating a drug in an earlier disease setting and provides patients and health care providers the option of using Zytiga earlier in the course of treatment,” said Richard Pazdur, MD, in the FDA’s announcement.

Early use

Specialty products are being used more for other diseases as well. “In the case of inflammatory diseases and TNF inhibitors, there is increasing use as first-line therapy for Crohn’s disease, psoriasis, and ulcerative colitis,” says Kaddis. “The idea of stepping through successive traditional agents and corticosteroids has gone by the wayside. Some individuals diagnosed with mild Crohn’s now receive biologic therapy earlier.”

The trend in inflammatory diseases is to achieve remission as soon as possible. That requires rapid assessment of the effect of initial therapy and escalation of therapy if progress is not made. In Crohn’s disease, guidelines say that corticosteroids are first-line treatment and may be followed by biologics such as Humira and Cimzia if progress is not evident.

The treatment of hepatitis C has been turned on its head by two new specialty products. “Prior to mid-2011 the recommended treatment was a combination of a pegylated interferon and ribavirin. Then came the protease inhibitors, and they immediately changed the recommended therapy,” says Kaddis. “Now a patient with hepatitis infection is put on triple therapy including pegylated interferon, ribavirin, and a protease inhibitor, either Victrelis or Incivek.”

Therapy guidelines for hepatitis C recommended triple therapy almost immediately after the release of the protease inhibitors. The Express Scripts 2013 Drug Trend Report says that in 2012, the utilization of specialty medications for hepatitis C increased by 29% — driven almost entirely by Victrelis and Incivek.

Keeping pace

Health plans face a challenge in keeping up with early use of specialty drugs that may be becoming the standard of care.

“One might think that health plans have the advantage of monitoring claims so that they can see changing treatment patterns in the use of specialty drugs, such as early treatment, but that is not necessarily the case,” says Brian Solow, MD, chief medical officer at OptumRx, UnitedHealthcare’s in-house PBM.

Brian Solow, MD

“I don’t think tracking claims is the way to go,” says Brian Solow, MD, CMO of OptumRx. He doesn’t want to merely respond after the fact.

While health plans and PBMs are working to capture complete, accurate, and timely claims information, there are many holes in the process that are beyond the control of health plans, starting with pharmacies submitting claims.

“One of the biggest challenges that managed care organizations have is understanding the course of treatment at the level of the individual patient,” says Kaddis.

“In many cases, it’s difficult to coordinate and track use of conventional drugs under the pharmacy benefit and biologics under the medical benefit. That means it’s difficult to track evolving patterns in the course of therapy for diseases with specialty drugs.”

The data-analysis approach may not be the answer. “I don’t think tracking claims is the way to go,” says Solow. “We want to act as an adjunct to physicians’ care, not monitor and respond to it after the fact. Physicians often believe that when the standard of practice changes, health plans are not keeping up. We need to be timely, monitor the literature, monitor the national organizations, and keep in touch with our consultants to keep ahead of the changes.”

He adds that it is important to stay focused on real evidence for specialty products, but this approach is more proactive than relying entirely on clinical guidelines or major published studies.

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