Current approaches to assessing the value of new and innovative medicines are narrow. Genentech is committed to working with a broad set of stakeholders on defining the full value medicines bring to patients, the healthcare system and society.

Across the U.S. healthcare ecosystem, different stakeholders have unique and sometimes overlapping priorities and perspectives on what constitutes value, from the effect of a medicine on a patient’s quality of life and a caregiver’s burden, to cost-effectiveness and equitable access to care.¹ This often leads to a fragmented view of the value of specific medicines for patients, the healthcare system and society, leaving us without a broad-based consensus on the definition of value and how to evaluate it for specific medicines.

The concept of “value” in medicines is, broadly, a way of considering the aggregate benefits of a treatment and the degree to which those benefits substantiate the costs to patients, payers and society. “Value” should encompass multiple factors from the perspectives of a broad spectrum of stakeholders across the healthcare system.

“Value is the amount of good or benefit derived from the use of a medicine by an individual stakeholder,” says Jennifer Whiteley, executive director, Evidence for Access, Genentech. “There is an opportunity to move beyond the traditional ‘cost per quality-adjusted life-year’ and communicate the value of medicines using a more holistic set of attributes that are meaningful to all stakeholders – from patients, families, and caregivers, to payers, providers, health systems and the broader society.”

What if we could come to a consensus on a more holistic approach to what value is and how to assess it? By looking more closely at the full range of benefits medicines offer, we could change the way we evaluate medicines throughout their lifecycle. By agreeing on definitions and approaches to assess value, we can help ensure benefits are incorporated into decisions about drug development, pricing, payer coverage and regulatory actions. Quantifying and increasing the visibility of these benefits will improve our ability to compare similar treatments and ensure we get the right treatments to the right patients at an appropriate cost.

CURRENT VALUE ASSESSMENTS FALL SHORT

Within the U.S., society rarely considers the broader benefits of treatments – such as lessening the impact of a disease on family caregivers – in value assessment and access discussions.² While we have seen important progress in convening stakeholders to discuss key issues in advancing the science of value assessment in health care, most discussions of value are based too heavily on narrow, generic measures – such as cost per quality-adjusted life year – that fail to represent the perspectives of stakeholders beyond the health care payer.³

Consider spinal muscular atrophy (SMA), a rare genetic disease that results in the loss of motor neurons, leading to debilitating and sometimes fatal muscle weakness. It can affect a person’s ability to walk, swallow or even breathe. The disease affects roughly one in 10,000 babies and is the leading genetic cause of infant mortality.⁴

Despite recent advancements, the majority of people in the U.S. with SMA remain untreated and there has been a need for continued therapeutic innovation. Over the past several years, new disease-modifying therapies have become available that have fundamentally modified the natural course of the disease, enabling patients to have improved outcomes. They have the potential to help keep infants off ventilators, hit key motor milestones such as sitting, and enable later-onset patients to maintain certain motor skills.

The traditional approach to assessing value often underestimates the broad impact of new and innovative treatments and does not account for the incremental effects, like caretaking activities, and overall costs to society. The value of such therapies will be viewed differently by patients and their families and caregivers, payers, the healthcare system and society overall, and quantifying the value from each of these perspectives holds its own challenges.

MOVING TOWARD A HOLISTIC DEFINITION

Genentech delivers innovative and groundbreaking medicines in areas of high unmet need. We take the responsibility of pricing those medicines very seriously, carefully considering the benefits they bring to patients, the healthcare system and society, and ensuring that patients have sustainable and equitable access while maintaining our ability to invest substantially in future innovations. We are committed to responsible pricing actions and meaningful transparency and we are mindful of our obligation to define value in a way that helps communicate the full impact of our medicines to a broad set of stakeholders. And while we do believe our medicines deliver clinical, economic and humanistic value, we recognize it can be difficult for stakeholders to fully understand a treatment’s total impact – especially if their definition isn’t aligned with ours.

We believe a consistent approach to defining, measuring and communicating the value of a medicine is urgently needed. In the past year, we’ve undertaken an effort to standardize definitions across our company and our portfolio. As we think more comprehensively about how our medicines affect a broad range of external stakeholders, we’re identifying areas where we need to provide and communicate metrics that can drive a shared understanding of value.

BUILDING AN IDEAL FRAMEWORK

We are striving to assess the value of our medicines using a more holistic set of attributes that are meaningful to all relevant parties. Over the lifecycle of a medicine, we are committed to investing in research to generate continuous evidence to support the full valuation of our treatments. However, it will take time to accumulate the evidence we need to routinely demonstrate holistic value.

Genentech is actively engaged in conversations with payers, policymakers and patient organizations on how we go about defining relevant and more widely accepted measures for value. We are committed to putting forth recommendations and ideas to help build an ideal framework that takes into account all stakeholders.

We believe advancing value assessment methodologies, portfolio-level perspectives and generating clinical and health economic evidence will be key components of a successful framework. We are working to define value on multiple fronts and advancing the ways that value can be assessed, including:

• Advancing research methods to enable us to better quantify the full value our medicines bring to all stakeholders
• Determining how to use these research methods to guide our actions – from drug discovery to pricing
• Generating information on how our medicines affect patients, families and society more broadly, including impacts on health equity
• Seeking to deliver value by demonstrating improvement in patient outcomes and maintaining or improving health-related quality of life
• Working to maximize economic gains to society – by pricing our medicines responsibly, engaging with policymakers and collaborating with public health and advocacy organizations

ALIGNMENT FOR OUR INDUSTRY AND PARTNERS

Making advances in defining and assessing the value of medicines throughout their lifecycle will require many interested parties coming together. No single biopharmaceutical company can do this alone, and our industry will need to work together and with a broad set of stakeholders who have a vested interest. We invite collaboration and a broader discussion of what benefits our medicines bring to society and how we can measure these benefits in more precise and relevant ways. In places where we disagree, it will be important to understand why and find ways to achieve better alignment as we navigate this complex territory. And as we find places where we agree, we can take the next step of initiating a much better-informed conversation about policies that speak to a shared, holistic, transparent definition of value – and how to assess it – that benefits all.