Can We Work Together to Ensure Patient Access to Treatments and Encourage Innovation?


Is it possible for all health care stakeholders to work together to develop solutions for containing costs that ensure patient access to care and appropriate treatments and encourage future innovation? As stakeholders meet in Washington, DC, on November 20 at the HHS Pharmaceutical Forum, we hope that they’ll consider a few of the ideas below, many of which we have discussed in greater detail in peer-reviewed research. This is not a comprehensive list, but rather a starting point for a constructive conversation.

Considering Risk-Sharing Agreements.

Under risk-sharing agreements (RSAs), health care payers and biopharmaceutical manufacturers agree to link coverage and reimbursement levels to a drug’s effectiveness and/or how frequently it is utilized. According to a recent study, there are benefits to these arrangements. Manufacturers can use RSAs to differentiate and demonstrate the effectiveness of their product versus their competitors, which can assist payers in making formulary decisions. Payers can utilize RSAs to gain experience with a product, reducing uncertainty regarding clinical value, performance and financial impact. Yet existing barriers, such as the effort and expense involved in patient monitoring, make it difficult to design and implement RSAs in the current U.S. health care environment.

Moving From “How Much” to “How Well” We Spend.

One approach toward reducing health costs currently being tested in the Medicare program and by some employers is value-based insurance design (V-BID). V-BID shifts the focus from “how much” to “how well” we spend health care dollars. V-BID is driven by the concept of clinical nuance, which recognizes that medical services differ in the benefit they provide, as well as the benefit each individual patient experiences from a particular service. By incentivizing consumers to utilize the treatments that are most beneficial to them, this could shift spending to higher value care.

Recognizing Individual Patient Differences Via Copays.

Each patient may respond differently to a particular treatment, meaning that a biopharmaceutical listed on the first tier of a formulary may not work well for everyone. The individual who does not respond well to that first biopharmaceutical might need to try one or more treatment options until finding one that works best, which might be on a higher, or more expensive tier of the formulary. Should that person be required to pay more for a medicine—whether it’s brand or generic—simply because his or her biological response is different?

Recognizing the Full Value of Treatments.

There is no one “value assessment framework” that can accurately determine all aspects of the value of a treatment for an individual patient, society and continued innovation. With the Institute for Clinical and Economic Review Value Assessment Framework, for example, there is a lack of model transparency, challenges with how the budget impact is calculated, and the need to realize the effects of some treatments over a longer time horizon, among other concerns. With the American Society of Clinical Oncology’s (ASCO) Conceptual Framework to Assess the Value of Cancer Treatment Options, it will be important to broaden and sharpen many of the framework’s components and ensure that it is not misused to limit access to life-extending cancer treatment regimens. It also is important to be mindful of the flaws in current assessments and ensure that they are not misused.

Understanding and Addressing the Gaps in Quality Measurement.

Quality measures play a critical role in the implementation of accountable care organizations (ACOs), as physician payments will be based on how they measure up against quality metrics. It’s important to understand how these delivery and payment structures save money, as well as whether they result in improved patient health.

A first step is to examine where gaps exist in quality measurements for prevalent and costly health conditions such as breast cancer, HIV, diabetes and stroke, and to evaluate whether we are incenting the most appropriate and effective treatments for patients with these illnesses. We need to continue strengthening our understanding of how new payment models may be impacting individual patient care and health outcomes, as well as how biopharmaceuticals fit into these models.

Collaborating on Data.

Thanks to innovation in the technology sector, we are now starting to see the benefits of a revolution in health care data and, with it, the potential to make health care payment and delivery even more effective and cost-efficient. Through electronic health records, claims data and even wearable devices, we are able to gather insightful, real-world evidence about how health care is being delivered, the quality of that care, and how biopharmaceutical treatments are performing in patients in everyday settings. This is even an area in which segments of the biopharmaceutical and insurance sectors have been able to collaborate to conduct real-world studies designed to determine how to most effectively and economically treat disease. How payers will utilize this evidence in making coverage decisions is still a work in progress, however, as new methods are being developed to ensure the quality of the data and how the research study is conducted, as well as how the evidence is evaluated.

Similarly, broader access to research-quality data taxpayer-funded databases could help researchers tackle a host of health care questions that cannot be addressed through other information sources. Although the Centers for Medicare and Medicaid Services recently allowed biopharmaceutical companies to access certain federal databases, access to state all-payer claims databases remains limited.

Enabling More Open Conversations Between Payers and the Biopharmaceutical Industry.

Section 114 of the Food and Drug Administration Modernization Act (FDAMA) of 1997 established a special mechanism for health care economic information that biopharmaceutical companies can share with payers. According to research on FDAMA 114 funded by NPC, “the language of Section 114 is sufficiently vague … leaving legal, regulatory, health economics, and other teams within drug companies to interpret the statute and gauge the company’s risk tolerance. The seemingly limited use of Section 114 to date may reflect companies’ uncertainty about its scope… In practice, it is hard to know exactly what the law allows given the imprecision of the statute and the lack of guidance about its scope.”

The uncertainty around Section 114 can present a barrier to the exchange of important information that payers need to make formulary decisions.

Now it’s time to roll up our sleeves and begin the conversation.

Interested in learning more about these and other important health policy issues? Stay up to date on these topics by visiting NPC's website, subscribing to our email alerts, and following us on Twitter at @npcnow. Check out NPC’s blog, Evidently Today, to view the previous posts in this series.

[Note: Previous posts in this series focused on considering all health costs; strengthening an innovative environment; and balancing quality improvement with patient access as we shift to value-based care.]