Redefining the value of medicines with patients at the centre
Today’s cancer advocates are smart, savvy and changing the treatment paradigm in significant ways. Broad accessibility of information has enabled us to make meaningful contributions on a host of topics, from clinical research to adherence programmes to disease awareness campaigns. They have moved from describing how they feel to what they know. Today, they gather and interpret data persuasively, never shy about putting patients’ interests first, even when their views conflict with those of industry or other members of the cancer community. Some patient advocacy groups oversee registries, develop patient-reported outcomes (PROs) and ensure relevant clinical endpoints. Others advise regulators on drug approvals and pharmacovigilance. They have earned their place at the table in these conversations.
While patients and their advocates have raised their voices regarding the value of innovative treatments, including their cost, they are rarely included in key decisions by payers about coverage and reimbursement. We call on payers to be more inclusive of the patient perspective.
At a time marked not only by significant scientific and medical progress but also spiraling health care costs, ‘value’ has become the dominant word in healthcare. Yet, more often than not, patients aren’t included in defining what it means or in deciding what’s worth paying for.
Professional societies and healthcare systems have sought to quantify the value of innovation in treatment guidelines and value frameworks. In 2015, the prestigious Institute of Medicine identified “patient-centeredness” as one of six critical elements of quality cancer care. Most other frameworks from professional associations are even less focused on patients’ views of value.
Putting patients in the middle of discussions about value is challenging, to be sure. Every patient has a different view. Who decides for patients? How representative are patients and advocates’ views? What is the balance between what patients want and what society can afford? How can the needs of one patient be balanced with the needs of our larger society? The questions are endless, yet the effort to explore those questions is worthwhile. In 2011, for example, ruxolitinib was approved by the US Food & Drug Administration (FDA) for treatment of myelofibrosis based in large part on data from a novel PRO instrument recording symptoms. In Canada, a patient group was able to interrogate a registry to support a change in access guidelines for two drugs used in kidney cancer. Doctors and patients can now discuss what is important to the patient and determine which drug to use based on patient preference.
Ongoing conversation with patient advocates about value is essential for every part of the cancer community, including payers. For decades, Takeda has been committed to putting patients first, built on three centuries of our company’s singular focus on trust, integrity, reputation and business. We always knew what is good for patients and their families would be good for our business. That mindset allows us to take bold steps to incorporate patients’ views and preferences at every level.
At Takeda, we routinely consult patients and their advocates. For example, in developing a novel compound to treat myelodysplastic syndrome (MDS), a fatal blood cancer, we included patient and advocate input into PRO endpoints in pivotal trials to improve the accuracy and sensitivity of measures of symptoms and quality of life. A novel partnership with the MDS Foundation has produced more clinically relevant insights and targeted measures leading to both better discussions with regulators and HTA officials and to enhanced submissions.
Takeda also engages with the entire cancer community in an ongoing dialogue on value. The company has been an active participant in roundtable discussions hosted by organisations, such as the Network for Excellence in Health Innovation (NEHI). NEHI’s convenings and publications on challenging topics like value-based contracting are richer because all stakeholders, including advocates, are actively involved in planning, debate and model programmes.
Over the past few years, payers have co-opted the value conversation. Under the guise of value, they have limited formularies, pushed more treatments into Tier 3 and specialty drug categories and even, in some cases, distributed cash bonuses to doctors who treat their patients with drugs from a specified, limited list provided by the payer. These decisions are made with little or no patient input, and often without the knowledge or consent of the patient. Payers have the opportunity, even responsibility, to understand and act on patients’ views and expectations.
The challenges do not rest with payers alone, of course. Value-based pricing faces challenges due to regulatory constraints. Tension exists between the FDA and the Department of Justice (DOJ) over patient involvement. DOJ is concerned with the appearance of pre-approval marketing while FDA is pushing for incorporating patients’ views early in the drug development process. In Europe, the European Medicines Agency’s (EMA) conflict of interest policy often precludes meaningful exchange between patients and industry. On another front, pharma must change both its practices and culture to ensure that patients’ preferences are a part of company calculations of value.
Expanding the conversation and creating a consistent definition about value is essential. The pharmaceutical industry will continue to advance innovative molecules from its laboratories to the bedside while comparative effectiveness research and health technology assessments will continue to determine how to allocate limited financial resources new treatments, often without patient consultation. Prompt action is needed if patients are to benefit from advances that can enhance and even save lives.
Specifically, we call on:
- All stakeholders to welcome patient advocates to the table and make a genuine effort to understand the patient perspective on value. A suggested start would be for payers to publicly disclose their policies and procedures on listening to and acting on the patient voice
- Regulatory bodies to continue including patients early in the process and to streamline policies that prevent the full participation of patients. The existence of policies and procedures that facilitate, rather than regard patients’ views, is crucial
- Pharmaceutical, biotech and device companies to commit to a culture of engaging patients to understand better their definition of value. Programmes that focus on integration of patients into value determination are essential
- Patients to continue contributing to the dialogue through government, industry and association meetings and policy-making channels
- Professional societies to reassess value frameworks by incorporating patient preferences
Advancing this call to action advances our shared goal: an honest, transparent conversation about value demonstration and resource allocation that reflects patients’ needs and preferences. That’s good for everyone.