November 20, 2019
The Honorable Seema Verma
Administrator, Centers for Medicare and Medicaid Services
Department of Health and Human Services
7500 Security Boulevard
Baltimore, MD 21244
Submitted electronically to ProgramIntegrityRFI@cms.hhs.gov
RE: Request for Information on the Future of Program Integrity
Dear Administrator Verma:
The National Pharmaceutical Council (NPC) appreciates the opportunity to submit comments in response to the Request for Information on the Future of Program Integrity. NPC supports the agency’s continued efforts to improve program integrity across the Medicare program.
NPC is a health policy research organization dedicated to the advancement of good evidence and science and to fostering an environment in the United States that supports medical innovation. NPC is supported by the major U.S. research-based biopharmaceutical companies. We focus on research development, information dissemination, education and communication of the critical issues of evidence, innovation and the value of medicines for patients. Our research helps inform important health care policy debates and supports the achievement of the best patient outcomes in the most efficient way possible.
We concur with the agency’s desire to ensure program integrity and “pay it right,” with a focus on reducing provider burden and putting the patient first. Our comments focus on the request for information regarding the potential to improve the prior authorization process — and we specifically focus on step therapy protocols, which frequently include prior authorization as part of the process. Step therapy is often used to ensure clinically appropriate care and while also managing costs. However, there is currently no comprehensive set of criteria or guide for step therapy. Research conducted by NPC demonstrates wide and varied use of step therapy in commercial health plans. Additional research by NPC shows that while stakeholders disagree on when to use step therapy, there is broad agreement on how to develop, implement, communicate, and evaluate step therapy policies and protocols. As CMS looks at next-generation strategies, tools and technologies, NPC encourages CMS to keep these findings in mind as the agency considers the use of prior authorization within the Medicare benefit.
Step Therapy Protocols Vary Widely and have Important Implications for Patient Health
Findings on the use of step therapy in commercial health plans can provide insight into the utilization and design of these protocols should they become part of Medicare Fee for Service (FFS). An NPC analysis of the data available in the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) database showed wide variation in the frequency with which plans apply step therapy in specialty drug coverage decisions. Utilization of step therapy ranged from 2 percent to 49 percent across the plans included in the SPEC database, which obtains information from publicly available coverage decisions issued by 17 of the largest 20 commercial health plans for their commercial lines of business. Even within the plans that utilize step therapy, the set-up of the protocols varies greatly, with some protocols being more onerous and burdensome than others. In the SPEC database analysis, of the just over 1,200 coverage decisions that included step therapy, 63 percent required beneficiaries to step through a single therapy while 37 percent required multiple therapies. Fifteen percent of the total coverage decisions included three or more steps; some protocols required failure on up to five treatments before patients could access a specified therapy. [1,2] Depending on the clinical conditions utilized in a step therapy program, a beneficiary could face irreversible harm or a delay in treatment use. The more restrictive a step therapy program is, the more problematic and burdensome it can be for an elderly patient or a patient with a disability.
Medicare covers both the elderly and patients with disabilities, meaning beneficiaries tend to be older, in poorer health, have more comorbidities, and take multiple medications. Logistical burdens could arise with step therapy protocols, such as additional lab tests and more doctor visits. These challenges can be more burdensome in the aged and disabled population of the Medicare program, especially those patients with severe conditions, limited mobility, or few transportation options. For example, beneficiaries with metastatic cancer facing limited survival time may see a delay in therapy as they face the administrative and logistical hurdles of a step therapy protocol. Additionally, beneficiaries may struggle to navigate the Medicare system or the appeals process. Again, delays in treatment could occur, further exposing these patients to harm.
Stakeholders Do Not Agree on When Step Therapy Should be Applied as a Utilization Management Tool
To better understand the optimal development and implementation of step therapy protocols, NPC employed a four-step modified-Delphi process to build consensus across a multi-stakeholder group of experts from the patient, provider, plan, pharmacy, policy and ethics communities. This process included a pre-meeting round of voting, individual telephone interviews with each participant to understand the context of and motivation behind each rating, and a group discussion followed by a second round of voting. Criteria were identified across five themes: clinical criteria as the foundation for protocol development; implementation of protocols; transparency and communication of processes; navigation of the appeals process; and evaluation of health and administrative impact. Roundtable participants were asked to do the following:
- Rate the overall appropriateness of step therapy;
- Rate the appropriateness of specific criteria and
- Identify individual criterion as a standard or a best practice.
Roundtable participants did not agree on the overall appropriateness of step therapy, with only 50 percent saying it was appropriate while just under 19 percent characterized step therapy as inappropriate. The remaining approximately 31 percent said step therapy was neither inappropriate nor appropriate. Some roundtable participants emphasized the role of step therapy to ensure clinically appropriate medication use; others voiced concerns that step therapy prioritizes cost management rather than patient health. However, participants emphasized that because any step therapy protocol should be developed and implemented in an evidence-based, patient-centered, and transparent manner.
A Varied Group of Stakeholders Agreed Upon Several Criteria to Develop, Implement, Communicate and Evaluate Step Therapy Protocols
While the participants of NPC’s roundtable did not reach a consensus on the appropriateness of step therapy, they did agree on criteria related to the development, implementation, communication, and evaluation of these protocols when used. Of 21 criteria considered, the majority (14) were rated as a standard that should be adopted for use in step therapy. Five were identified as best practices; two criteria were categorized as neither a standard nor a best practice. The checklist is available upon request. We highlight a subset of these criteria related to the RFI questions below.
Can clinical decision support tools play a role? If yes, how?
Clinical decision support tools can play a role in prior authorization and step therapy. As outlined in the roundtable, stakeholders agreed that clinical evidence should be considered before costs when developing step therapy policies. Second, these policies should be based on all available clinical evidence regarding therapeutic need, efficacy, safety and effectiveness, use established clinical practice guidelines or compendia, and be updated regularly to reflect current evidence. In addition to these inputs, the roundtable agreed that step therapy policies should include the beneficiary or member perspective. Finally, the risk of failure should include traditional markers of success such as surrogate endpoints and biomarkers, outcomes, lack of adverse events, but also include quality of life and provider judgment based on markers that may only be evident during the provider-patient interaction. Clinical decision-support tools can and should incorporate these inputs. However, clinical evidence found in guidelines may lead to step therapy protocols that assume one-size-fits-all patients, often not the case for vulnerable patient populations such as Medicare beneficiaries. When clinical decision-support tools suggest multiple treatments may be optimal based on different patient preferences, the use of step therapy may be less optimal.
How can prior authorization be applied while maintaining timely and complete access to medically reasonable and necessary covered services for beneficiaries?
As outlined at the roundtable, transparency and communication of any step therapy requirements is critical to maintaining timely access to medically reasonable and necessary services. For example, requirements regarding the step therapy protocol should be easily accessible via a public link on the benefits administrator’s website (e.g., CMS website). Panelists agreed on the need to share status within a step therapy protocol electronically (e.g., Mymedicare.gov). Efforts to facilitate the electronic exchange of information to reduce burden on providers, create more standardized submission forms for exception or prior authorization requests, and provide a clear opportunity for the patient or the health care provider to request an exception are needed.
How can provider and supplier burden be minimized?
Panelists at the NPC roundtable noted that step therapy should be implemented to ensure that provider — and most importantly —patient burdens are minimized. For example, patients should face no more steps than clinically reasonable, and the required time to try an initial treatment should ensure that the patient does not face, nor is at risk for harm. As noted, harm can extend beyond clinical measures such as biomarkers, lab tests, or clinical outcomes if a treatment impacts a patient’s quality of life or ability to perform daily activities. Many non-clinical markers may only be evidence during the provider-patient interaction and overlooked if there is over-reliance on clinical measures. For these reasons, greater treatment flexibility is needed when treatment choice should account for differences in disease severity, unpredictable treatment response, or patient experience. In these situations, step therapy and prior authorizations — approaches to limit treatment options — are not appropriate.
Once authorization is granted, approval should stand as long as the patient remains a beneficiary. Tests documenting lack of treatment success within the Medicare FFS program, or under a prior health benefit plan, should be acceptable to the FFS program and diminish interruptions in care. Finally, participants recommended utilizing electronic processes for communication to allow patients and health care providers to view the step edit protocol and exception status. As outlined in the RFI, MyHealthEData and other portals may facilitate such access. Finally, as explained at the roundtable, the electronic process for communication should also include the pharmacist providing point of care service.
Are there other issues with respect to prior authorization and step therapy that CMS should consider?
Finally, CMS should consider evaluating the processes and outcomes resulting from any expansion of such policies by measuring factors such as the total annual step therapy requests, approvals, denials, % of appeals accepted and denied, % of appeals sent for external review. Without such evaluation, the administrative costs (staff and resources for both CMS and the providers complying with step therapy) may outweigh the potential impact of getting the right care at the right cost and optimizing patient health.
As our health care system moves towards paying for value over volume, it is vital to ensure that the use of step therapy and prior authorization do not adversely impact patient outcomes. Positive health outcomes are the ultimate goal of this transition, and it is important to ensure that it does not come at the expense of administrative burdens or a patient’s health.
The roundtable conducted by NPC created actionable standards and criteria that can be incorporated by health plans, including Medicare FFS, should they choose to employ step therapy. We hope to continue this discussion with CMS and other stakeholders as we work towards better patient outcomes and would be happy to meet to expand upon our comments and share our research.
Robert W. Dubois, MD, PhD
Chief Science Officer
National Pharmaceutical Council
Taruja Karmarkar, PhD
Health Policy Fellow
National Pharmaceutical Council
1 Chambers, J., Kim, D., Pope, E., Graff, J., Wilkinson, C., and Neumann, P. (2018). Specialty Drug Coverage Varies Across Commercial Health Plans in the US. Health Affairs, 37(7), 1041-1047.
2 Chambers, J., Panzer, A., and Neumann, P. (2018). Variation in the Use of Step Therapy Protocols Across US Health Plans. Health Affairs Blog. Available at: https://www.healthaffairs.org/do/10.1377/hblog20180912.391231/full/.