Evidence-Based Assessments to Evaluate Medication Value

Eileen Koutnik-Fotopoulos

Cincinnati—Medication assessments directly impact the way a drug is covered, and medications that have demonstrated high potential clinical value have betters odds of being covered, according to Sean Karbowicz, PharmD, manager, clinical pharmacy, RegenceRx Pharmacy Benefit Management.

During a Contemporary Issues session at the AMCP meeting, Dr. Karbowicz discussed the topic during his presentation titled Evidence-Based Value Assessment of New Medications: 2012 Update. New medications and accompanying technologies require sophisticated and specialized assessments. Medications brought to market with high-quality evidence are more favorably covered. Potential gaps in clinical evidence could impact medication coverage. From a payer’s perspective, significant gaps persist in evidence that describe clinical value, said Dr. Karbowicz.

Using the Institute for Clinical and Economic Review (ICER) matrix, he described the synthesis of clinical evidence that exists for specialty drugs, potential blockbusters, and reformulations of known compounds. The ICER matrix looks at degree of certainty (high, moderate, and low) and net health benefit when assessing medications.

Dr. Karbowicz highlighted several specialty drugs with the applied ICER matrix. For example, the evidence assessment for Inlyta® (axitinib) for the treatment of renal cell carcinoma, which has other alternatives available, was “promising but inconclusive.” He said the drug’s efficacy benefit was unclear and its comparative benefit unknown. As for safety, the drug exhibited no apparent safety advantage. The most costly treatment option is $14,000 per month.

The evidence assessment for Adcetris (brentuximab vedotin) for the treatment of lymphoma was deemed insufficient. Cost of the drug is $13,600 per infusion (maximum 16 infusions). He said it is uncertain if the potential benefit of the drug “to shrink tumor size outweighs the risk of serious adverse events.” For the treatment of lung cancer, the evidence assessment for Xalkori® (crizotinib) was also insufficient. It is uncertain what benefit crizotinib provides, and safety of the drug is evolving with visual disturbances of particular concern. Besides its unknown clinical value, crizotinib is “more costly than platinum-based chemotherapy, which has known clinical benefit and a track record of safety in the metastatic non–small-cell lung cancer setting,” according to Dr. Karbowicz.

He also described the synthesis of clinical evidence that exists for other specialty drugs including Jakafi® (ruxolitinib) for myelofibrosis, Zelboraf® (vemurafenib) for metastatic melanoma, Perjeta (pertuzumab) for metastatic breast cancer, and Kalydeco (ivacaftor) for cystic fibrosis.

The presentation continued with potential blockbusters for antiplatelet and anticoagulation therapies. Effient® (prasugrel) and Brilinta® (ticagrelor), both indicated for the prevention of thrombotic events following acute coronary syndrome, were given a certainty degree of moderate and low, respectively. As an alternative for clopidogrel intolerance, the evidence assessment for prasugrel was “promising but inconclusive.” The evidence assessment for ticagrelor was insufficient and “interpretation of safety data in the United States is limited by clinical trial flaws,” leading to the uncertainty of the drug’s value in North America, according to Dr. Karbowicz.

The effectiveness of anticoagulation therapy for atrial fibrillation was also presented. Pradaxa® (dabigatran) was given a degree of moderate certainty, while Xarelto® (rivaroxaban) was rated low certainty. Both drugs were assessed for use in hip/knee arthroplasty with the degree of certainty the opposite, compared with their use in atrial fibrillation. Dabigatran was rated low certainty and rivaroxaban was rated moderate certainty.

“Dabigatran has greatest potential value in patients with atrial fibrillation when warfarin monitoring is problematic or who are on medications that have clinically significant interactions with warfarin,” said Dr. Karbowicz. “Rivaroxaban has potential value in hip/knee arthroplasty as a convenient oral alternative to more costly injectable therapy.”

The potential value of reformulations of known compounds was also part of the presentation. These drugs included Onfi® (clobazam) for seizures, Intermezzo™ (zolpidem sublingual) for insomnia, and QNASL® (beclomethasone dipropionate) for allergic rhinitis.

Accurately assessing the evidence for new medications using study population and sample design, efficacy and safety data, cost, outcome measures, progression-free survival data, overall data, and equivalent comparators will help payers determine a medication’s benefit-to-risk ratio.