Medicare Drug Price Talks Could Drive More Patient-Reported Data

Medicare’s drug price negotiations could advance broader use of the patient experience into drug development if there’s more clarity on what to submit and the level of impact it will have.

The patient experience is one of several factors the Centers for Medicare & Medicaid Services will consider as the agency prepares to negotiate drug prices for the first time, according to initial guidance published last month to lay out how the negotiation process will work.

Specifically, patient experience will contribute to the evaluation of a drug’s clinical benefit compared with therapeutic alternatives. The agency also said it will consider patient-reported outcomes as part of its analysis.

That determination of clinical benefit ultimately adjusts the starting point for drug-price negotiations. If patient data demonstrates a greater clinical benefit of a therapy, it could potentially increase how much Medicare is willing to pay for that drug.

“The fact that they’ve even captured the need to bring in patient experience and analogously patient experience data is a step in the right direction,” Esther Krofah, executive vice president of health at the Milken Institute, said. “We need to understand clinical benefit not only through the approval of the drug, but clinical benefit overall in that patient’s experience with that approved drug.”

Systematic Submission

At the same time, she cautioned that the agency still needs to offer more details, such as how to submit data, how to structure the data so they’re meaningful to the agency, and the types of elements and content the CMS is looking to provide into its overall data package.

“CMS has not engaged in a very systematic way with patient organizations in the past. They need to build all of these processes,” Krofah said.

There are still a number of questions to answer, such as: What’s the intake office? How do you reach out? How is it staffed? What’s the call line? What’s the follow up? How should the data be submitted? Will it be different by the type of organization? Is there advance notice?

“All of this is important from a resourcing perspective from the patient organizations who then have to do that initial upfront investment that can then feed into this entire process—if the desire is for CMS to do this in a systematic way,” Krofah said.

Patient Experience Data Increasing

Incorporation of the patient experience now happens more often, Karin Hoelzer, director of policy and regulatory affairs for the National Organization for Rare Disorders (NORD), said.

The Food and Drug Administration has “very robust engagement of the patient perspective,” but “you can never have enough engagement,” Hoelzer said. “As CMS is developing the negotiation process from scratch, we hope that CMS will incorporate the patient experience to the same extent and the same level. And we do hope that that will trigger additional research into capturing the patient’s perspective.”

Looking at indication-based value and indication-based perspective is an appropriate step for CMS, Heidi Ross, NORD’s vice president of policy and regulatory affairs, said.

As rare indications are likely to come up for negotiation down the line, rare disease patients’ unique perspective need to be part of the conversation, Ross said.

“We really hope that the value that these therapies bring to the rare community will be rewarded within this process,” Ross said. “Their experience should be valued within that negotiation.”

Patient-reported outcomes refer to a report of the patient’s health condition that comes directly from the patient, without a clinician interpreting those results. They can range from a description of symptoms to side effects in a clinical trial to how a disease affects their well-being.

Including the patient voice into every phase of drug development “will make the process faster, it will align to the needs of patients, and then ultimately, it will help us get to better health-care outcomes,” Krofah said. She is also the executive director of Faster Cures, a Milken Institute center focused on building a patient-centered health system.

The FDA drug center has worked on patient-focused drug development for years, holding public meetings for more than a decade issuing a series of guidances.

Meanwhile, the CMS also has done work in this space, including the 2021 release of meaningful quality measures that prioritized outcome and patient-reported measures.

Stakeholders could use more insight from both agencies on how they’ll use the data, Henrietta Awo Osei-Anto, a director at FasterCures, said. “That’s really what’s needed next.”

Some data points are more established than others, and Osei-Anto expects those to carry more weight. “But who knows how much that patient experience, as they’ve indicated, is going to play into that decision making?”

Krofah added, “The interesting thing is, actually, the clearer CMS is about what data they want on patient experience, the more the industry itself will shift and move.”

Peter J. Neumann, director of the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center, said drug companies already incorporate the patient voice.

“The fact that they list patient approaches is important,” he said. “Will this create a stronger incentive because of that line in the guidance? I don’t know.”

CMS Response

The initial guidance is one of many tools the CMS will use to ensure interested parties’ voices are heard on implementation of the new drug law, an agency spokesperson said. The revised guidance scheduled to come out this summer will explain the rationale for any changes.

“CMS intends to broadly evaluate the body of clinical evidence including data received from the public and manufacturers and data identified through a CMS-led literature review,” the spokesperson said.

The agency may also rely on Medicare claims, pharmaceutical drug data sets, and any other information relevant to the selected drug and its therapeutic alternatives.

Drug manufacturers and members of the public “may optionally submit information on selected drugs and their therapeutic alternatives.”