Specialty drug utilization is increasing, as are the associated challenges around adherence, cost, and delivery of optimal value. Specialty drugs are reported to represent 39% of the overall pharmacy benefit spend, with projections to increase to 48% by 2020.1,2 The prices for specialty drugs had a reported average increase of 9.6% from 2014 to 2015, and are expected to continuously rise, with the largest growth expected within oncology.3,4 The specialty medication pipeline is also expanding, comprising 60% of all new drug approvals in 2017,5 and grew an additional 25% in 20186.

This growing landscape offers patients with complex and chronic conditions more treatment options; however, increased utilization is often associated with certain challenges. Health care stakeholders face increasing pressure to address the rising costs of these specialty medications while continuing to provide affordable and accessible care.

A 2017 Express Scripts report documented that 118 specialty drugs entered the market over the preceding 5 years, with predictions outlining another 25 expected annually until 2022.7 This indicates that health care stakeholders—payers in particular—must prepare strategies to address the expanding number and costs of specialty medications that this large influx of new drug developments might preface.

Payers are joining forces with pharmacy benefit managers (PBMs) to enhance their ability to combat the increase in drug prices.8 As more specialty medications enter the market, payers and PBMs are being more selective in their formulary management, and negotiating better pricing structures. Manufacturers want to be included on the larger plans’ formularies; therefore, price negotiations often lead to offering payers and PBMs a larger discount or rebate.9 Within formulary management, dose optimization and utilization management are 2 proactive strategies that payers and PBMs might use to address the increasing costs.10

For the purposes of one program—implemented at Diplomat Specialty Pharmacy in Flint, Michigan—dose optimization is defined as consolidating a patient's regimen into fewer units without a change in their dosing frequency. Dose optimization strategies offer a potentially valid, clinically based intervention in which payers can realize a direct drug cost savings, and indirect medical cost avoidance. Dose optimization programs have been evaluated with once-daily, oral maintenance medications using various methods that produced varied results.10 These studies were conducted in medication classes such as gastroesophageal reflux disease (GERD), anxiety and depression, and hypercholesterolemia.11-12 While the current literature describes dose optimization in the nonspecialty space, there is limited literature on dose optimization strategies used for specialty products.

A specialty pharmacy developing a dose optimization program could evaluate the implications and viability for specialty products, since they work closely with payers and providers. A successful dose optimization program within a specialty pharmacy could contribute a significant cost savings for payers, further mitigating the rising costs of specialty medications. Therefore, the goal of this pilot program was to evaluate the scenarios and opportunities for dose optimization within a selected group of oncolytics.

Dose-Optimization Program
This Diplomat program was designed as a proof of concept to illustrate the implications and barriers associated with dose optimization in a specialty pharmacy setting. This setting offers access to many specialty medications, and uses a high-touch model of therapy management for patients with complex conditions, such as cancer, multiple sclerosis, psoriasis, human immunodeficiency virus (HIV), and others. With more than 1100 products currently in development, oncology is one of the fastest-growing specialty segment on the market.13 This growth is what led the specialty pharmacy to focus on dose optimization opportunities within this population.

Diplomat’s oncology program delivers comprehensive care management to help patients address complex aspects of their treatment and condition. The crossfunctional oncology team is composed of specialized clinicians, and nonclinicians leveraging evidence-based care for treatment optimization, improved care coordination, and therapeutic cost management. The steps the team followed are displayed in the Figure below.

Figure – Method Steps for the Program


The retrospective prescription evaluation of oral oncology medications identified 58 initial opportunities for dose optimization. Financial contingencies varied for each stakeholder, and did not always align among the patient, the payer or PBM, and the pharmacy, which hindered dose optimization recommendations. During the evaluation phase, it was imperative to assess the impact on these stakeholders, including patient, payer, and pharmacy, to ensure a win for all. As shown in Table below, several circumstances would have resulted in a therapy delay that could potentially have adversely affected patient outcomes and adherence.

Table – Scenarios Impeding Dose Optimization
Scenario Hindrance Program Patients Impacted
Additional Prior Authorization Requirement Therapy delay 6
Insurance Override Requirement Therapy delay 4
Patient Therapy Change Opportunity no longer applicable 8
Patient Recently Optimized Opportunity no longer applicable 3
Financial Assistance Changes Access barrier 1
Pharmacy Business Implications Unsustainable financial impact 27

Specialty pharmacy is uniquely positioned to ensure proper patient care while optimizing medication adherence and outcomes. If we anticipated that the dose optimization program would negatively impact patients, as shown in Table 1, we excluded those patients from the dose optimization program. Specifically, we excluded:
  • Patients who had a change in therapy to a different medication during the evaluation time period
  • Patients whose providers initiated a dose-optimized prescription without any intervention from the pharmacy
  • Patients who would face increased financial responsibility, such as a copay, or a change in financial assistance
    • One patient would have been required to resubmit a grant application for financial assistance if a dose-optimized prescription were pursued. The outcome of the application would be unknown until the prescription was processed and could potentially compromise the patient’s current financial assistance.

Overall, we successfully optimized dosing for 9 of 58 patients.

What We Found
  • For payers and PBMs, this optimization produced a cost savings of approximately $135,000 that was not payer- or medication-specific.
  • We successfully reached all providers of qualifying prescriptions via phone, and received responses within 5 days of initial contact. Of the 9 potential prescriptions, we received 6 and successfully filled 5. The sixth prescription received a payer mandate rejection that necessitated it be transferred to a different pharmacy.
    • For the 3 patients for whom we did not receive a dose-optimized prescription, the provider indicated either that the patient was not interested in dose optimization at this time or that the patient had swallowing issues and preferred the smaller formulation.

What It Means
This proof-of-concept study of dose optimization in a specialty pharmacy setting evaluated various scenarios and identified significant barriers. Importantly, this program illustrated how stakeholders might influence one another, creating competing financial and service contingencies.

Often, the payer and PBM are part of the same organization, adding an additional layer of influence to medication access and prescription reimbursement claims. This situation was true for 1 particular patient that was eligible for dose optimization. Although they were included in this program, they encountered a dispensing pharmacy barrier when the dose-optimized prescription was processed: the claim was rejected, and the pharmacy was mandated to transfer the prescription to a payer/PBM stakeholder. The exact reason behind this subtle channel management is unknown; however, it was noted that the dose-optimized prescription was a profitable prescription while the previous prescription was not.

This scenario is an example of financial contingencies from 1 stakeholder impacting the standard clinical program implementation of another through financial incentives. In this scenario, the patient ultimately experienced a disruption in care, and an inconsistent patient experience, given that the patient had been well-managed by the specialty pharmacy for approximately 23 months.

This transferring of care forces patients to establish relationships with new specialty pharmacy staff, and creates duplicative work within the industry. In addition, the patient might experience a lapse in therapy during this process, leading to a potential loss in efficacy and worsening of their condition. It is imperative that key stakeholders understand their impact on each other and work together to ensure that their contingencies are aligned through transparency, and beneficial relationship building. This helps avoid a disruption in care and any of the clinical implications of such events.

These situations highlight the complex and interdependent financial and service contingencies that influence the ability to optimize doses. For the payer, dose optimization programs prove to produce a positive impact, as seen in a direct savings on the cost of product. For the patient, there might be a decrease in copay or coinsurance, depending on how their benefits are structured. Additionally, reducing the patient’s pill burden can potentially increase medication adherence and safety.10 Properly ordering dose-optimized quantities can help minimize medication error risk by reducing the quantity of medication that the patient has on hand. This makes the patient less likely to be confused, and unknowingly take an incorrect dose.

Advocating for patient safety can help minimize these risks, which can be characterized as a soft savings benefit seen downstream from an initial action such as dose optimization.

Conclusions
A specialty pharmacy plays a critical role in providing patients with access to their medications, and therapy management. This proof-of-concept dose optimization program, implemented in a specialty pharmacy, showcased various scenarios that result from attempts to dose optimize that were unfavorable under specific circumstances, and that might otherwise have gone unnoticed without deeper review. Although this program was conducted in a small population of patients receiving oral oncology products, the scenarios observed describe some of the barriers that other organizations might face with dose-optimization programs. With an awareness of each stakeholder’s contingencies and influences, future health care policies can address these issues on a national level to increase the success rates of cost-containment strategies.
 
Song Lee, PharmD; Stephanie Lapointe, PharmD, CSPO, FMPA; Linda Lampi, PharmD, CSP, CPHQ; Steven M. Schwartz, PhD, and Florencio Calderon were affiliated with Diplomat Pharmacy Inc. in Flint, Michigan during project implementation. Their writing was assisted by Kanika Kapoor, PharmD.


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