ANALYTICAL


Patient Focus: The Heart of Biopharmaceutical R&D


J. Michael Maher, MS, MBA Executive Director, U.S. Regulatory Policy at Pfizer, Inc.


Submitted: 01/13/2015 Accepted for Publication: 02/24/2015


Maher JM. Patient focus: the heart of biopharmaceutical R&D. Pharm Outsourcing. 2015;16(2):22-25.


Successful companies are customer-focused, and in the research- based pharmaceutical industry, patients are the key customer. The biopharmaceutical industry is unique in many ways, however, with attributes that complicate the straightforward consumer-provider relationship enjoyed by many other businesses. To begin with, this industry is highly regulated. New biomedical innovations are not in and of themselves marketable products. National regulators must first agree that adequate clinical and preclinical data are in evidence to demonstrate a favorable benefit-to-risk profile. Absent regulatory approval, a company’s investigational compound is not a medicine. Once a drug is approved, product promotional messages—whether to patients or other stakeholders—must be consistent with the scope and content of the regulator-approved label.


Beyond this, pharmaceutical companies do not generally supply product directly to customers. The sophistication of most medicines and the narrow circumstance of use typically require prescribing and dispensing through healthcare intermediaries: physicians, nurses, and pharmacists. Direct patient access to medicine is limited to the relatively small group of over-the-counter therapies.


In addition to these complexities, the essential value of most medicines warrants that they are paid for—at least in part—by governments or third-party insurers. Patients are the prime customers, but the access, use, and purchase of innovative medicines involves other stakeholders— namely regulators, healthcare providers, and insurers.


Patient Participation in Industry


Drug Development Patient engagement is important to all healthcare system stakeholders, none more so than drug developers. At the most fundamental level, innovator drug companies generate 2 products: the intellectual property that defines the basic biochemical inventions and the approved product labeling that summarizes years of clinical and pre-clinical development.


Patients are involved in many aspects of company R&D with their most visible role being that of essential but generally anonymous clinical trial participants. The contribution of patients in industry drug development has always been more extensive than that, however. Even during the early stages of research, the project operating proposals that set company discovery priorities need patient data to characterize the unmet medical need for conceptual products and to define desired treatment attributes. During the discovery research stage, information may be gathered to characterize patient concerns regarding disease symptoms, aspects of care, function, and potential functional assessment methods.


Later in the R&D process at the pre-clinical phase, when investigational drug candidates have been identified, patient information may be


collected from registries, from global patient communities, and from advocacy groups in order to assess patient willingness to participate in clinical trials or to partner in the development of functional measures and patient-reported outcome tools (PROs).


The clinical development phase, where the therapeutic characteristics of an investigational compound are explored and the data package to support product regulatory approval is assembled, by definition is focused on and depends on patients. However, beyond clinical trial design and operation, there are other patient-focused activities that take place during clinical development. For example, activities may include working with patients to conduct usability tests on devices intended for use in clinical trials such as diaries, seeking patient participation in advisory boards to inform aspirational product commercial profiles, using semi-structured qualitative interviews to understand disease symptoms and illness experience from the patient perspective, and partnering with patient groups to develop and qualify patient-reported outcome assessment tools.


Patient engagement during R&D has always been extensive and multifaceted. Before the recent launch of the U.S. Food and Drug Administration (FDA) Patient Focused Drug Development initiative,1 however, elements of company patient-centered drug development were often less visible, less coordinated, and, consequently, some- times underdeveloped.


FDA Patient Focused Drug Development


The FDA Patient Focused Drug Development (PFDD) initiative includes several important components described in the 2012 Prescription Drug User Fee Act (PDUFA V).2


As an element of PDUFA, these FDA PFDD


activities are supported in part by industry user fees. This new and successful FDA patient engagement program continues to integrate patient priorities further into FDA benefit-risk decision-making. A hallmark feature of the program is a series of FDA-hosted disease area stakeholder meetings. To date, FDA has held 11 of 20 meetings which are planned over the 5-year PDUFA interval. During these meetings, FDA listens to patients to receive their insights on disease conditions and the availability of acceptable treatment options. Gathering patient input to assess medical need is not in and of itself unique, having long been part of company discovery prioritization and development planning, but doing this through a common, inclusive, and public process is novel.


To assist in this process and to help to incorporate patient information into drug reviews, a second, PDUFA-supported regulatory tool, known as the Structured Benefit-Risk Framework,3


was developed and is being


incorporated into product regulatory review procedures. The current version of the Benefit-Risk Framework is outlined in Figure 1.


Pharmaceutical Outsourcing | 22 | March/April 2015


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