As published in Clinical Leader
Pharmaceutical companies face many challenges: developing life-changing products that meet the needs of patients, physicians, and payers; adhering to regulatory standards; and managing health technology and payer scrutiny, all while trying to satisfy investors. As drug prices seem to be continually rising, many health plans are shifting more of the cost-sharing burden to patients. As a result, the patient is becoming more of a “consumer” in the traditional sense.
This shift demands that the pharmaceutical industry begins to use the types of consumer research that are a mainstay of other industries where true value of a product is defined by those who will ultimately use it. For instance, just an aisle away from retail pharmacists, packaged goods and health and beauty companies are gaining a competitive edge by seeking consumer input throughout development, from product conception through market entry and beyond.
Despite growing efforts to engage patients in research and regulatory activities, the pharmaceutical industry has yet to maximize patient involvement in the drug development process. As healthcare systems continue to evolve and establish patients as the primary stakeholder in their own healthcare decision-making, the pharmaceutical industry will need to find innovative ways to demonstrate the value of their products relative to the outcomes experienced by the ultimate consumers of healthcare: patients.
The patient perspective on value is especially important now, as patients are responsible for more and more of the costs of their care. Not surprisingly, a recent Kaiser Family Foundation poll found that two-thirds (67%) of Americans, regardless of political affiliation, feel that lowering out-of-pocket healthcare costs should be a top priority for President Donald Trump and Congress. But despite the drive toward value-based healthcare reimbursement and patients’ ever-increasing financial stake in their own healthcare treatment, many traditional value assessment tools fail to consider value from the patient’s perspective.
Developing treatments that can truly help improve patients’ lives should be rooted in a firm understanding of the daily challenges patients face, their needs, and the trade-offs they are willing to make to gain relief. To create valuable treatments, patient needs must be aligned in all aspects of the healthcare system, including research priorities, product development, trial design, regulatory approval, access, reimbursement, and treatment decisions. This realignment has started to take place on the payer side with the use of health technology assessments (HTAs), which stipulate that patients’ preferences or perspectives be integrated in value demonstration. Such HTAs are in use in several European countries, and similar value frameworks are beginning to emerge in the United States, bolstering hopes that these nascent efforts to increase patient involvement in various aspects of healthcare will develop and expand in scope.
Despite this paradigm shift toward incorporating patient perspectives, pharmaceutical companies have yet to maximally engage with patients to learn what they value before developing a product. Most pharmaceutical companies have advocacy and market research groups that have networks and skills in patient outreach.
These departments, however, tend to focus more on the commercial aspects of launch and post-launch activities, such as disease awareness and education. Individuals working in these areas often have different objectives for patient engagement than their colleagues in clinical development. Because they may also lack the scientific or technical skill set necessary to collect patient input in a meaningful way that will result in actionable insights for their clinical development programs, many decisions in clinical development are made without patient input.
Presently, there are limited data that describe the scope and overall benefit of existing patient-centered drug development activities. Nevertheless, the story of the inhaled insulin product, Exubera® (human [rDNA origin]), illustrates how a better understanding of patient needs can inform the development of a more valuable product and prevent costly missteps. Pfizer’s Exubera inhalation powder received U.S Food and Drug Administration (FDA) approval in January 2006 as a mealtime insulin.
The goal of this product was to offer the convenience of an alternative, less invasive route of administration over injected insulin. Unfortunately, the delivery device was found to be too cumbersome and patients did not find it appealing enough to stop using injectable insulin. Challenges to gaining the interest of patients and providers led Pfizer to voluntarily withdraw Exubera from the market; the decision cost the company $2.8 billion.
Outside the pharmaceutical industry, however, patient engagement is increasing. Patients have been invited by regulators such as the FDA, the European Medicines Agency, and the National Institute for Health and Care Excellence to provide perspectives and advice during decision-making. In the Prescription Drug User Fee Act V, spanning fiscal years 2013 to 2017, the FDA launched the Patient-Focused Drug Development initiative. This initiative was designed to gain the patient perspective in certain disease areas and more effectively inform the FDA’s evaluation of the risks and benefits of new therapies. The British Medical Journal now includes patients in its peer-review process and encourages authors to coproduce articles with patients. Researchers submitting grants to the Patient-Centered Outcomes Research Institute, a nongovernmental organization, are also encouraged to work with patients.
Participant-led research using patient-powered research networks and social media is challenging our traditional views of what “a participant” is truly capable of doing. Medical conferences take steps to self-accredit as “#Patients Included™” by involving patients in planning, as presenters, and in the audience. Medical journals can accredit themselves by inviting patients to join their editorial boards, submit content, and act as peer reviewers. Such patient-centered activities differ from mere tokenism because patients themselves are making decisions with real consequences, instead of merely being asked for opinions that can arbitrarily be used or ignored by decision makers.
These efforts to increase patient involvement in various facets of healthcare highlight a growing need for the pharmaceutical industry to shift its approach to proactively engage patients in the drug development process. If pharmaceutical companies remain unengaged with patients, they risk not only falling behind what FDA learns as a result of the Patient-Focused Drug Development initiative, but also misunderstanding their customers’ concerns and ultimately breaking their social contract with patients.
Given the time and money required to develop a new drug, any misstep is likely to have significant financial ramifications. Engaging patients in the drug development process is not yet commonplace for most pharmaceutical companies, but the companies that are first to find meaningful ways to include patients in this process stand to benefit both in the approval process and in market access.
(To read the complete article as it appears in Clinical Leader, please click here.)