By Steve CaseyEarly-stage companies commonly focus solely on investors and regulatory stakeholders and overlook other stakeholders who are critical in optimizing the later development-commercialization pathway. Addressing these other stakeholders maximizes the “downstream” value of any product. Such stakeholders are gatekeepers critical to life-cycle areas such as market access, reimbursement, and medical education. Even investors in early-stage companies are now considering the impact the “downstream” stakeholders have on their ability to secure financial exits. Today’s newly minted healthcare-product companies are frequently being asked to demonstrate both regulatory viability and commercial potential at the earliest stages. Long gone are the days when a product would “sell itself” and companies could count on guaranteed reimbursement and rapt attention from healthcare providers. Rather, today’s stakeholders are focused on specific therapies with defined risks and demonstrated healthcare benefit. As a result of these changes, a broader definition of product success must be established during early development to generate a rational basis and plan of action for both product approval and commercial success.
Engineer the Product Value It has long been understood that an evaluation of commercial potential during the early stages of clinical development in parallel with a well-developed regulatory strategy can greatly increase the value of a product. The integration of market forecasting, health economics, reimbursement and anticipated medical practice input leads to a development strategy that engineers more value and is more cohesive than a traditional staged approach. While commonplace in large pharmaceutical companies, this integration can be difficult for smaller companies where resources are limited, information is incomplete, and, frankly, product commercialization is viewed as the job of whatever company decides to license the product. While many legitimate limitations exist in the ability to describe a product’s potential completely at an early-development stage, future success will be determined by how well companies are able to structure integrated development programs to meet the needs of all stakeholders. How to Get There from Here Fortunately, it doesn’t require armies of healthcare professionals, scientists, and MBAs to develop an integrated clinical, regulatory, and commercial development plan. If a company takes a structured approach to establishing the program goals, much of the information is common to all perspectives. It has been said that the value of a product is driven by its product label; this is true if the commercial aspects of a healthcare product’s business are limited to communicating only what is in the label. To help guide development programs the FDA has provided a good starting point for drug developers with the Target Product Profile (TPP). The FDA TPP template follows the major categories of a package insert, allowing developers to forecast the desired target product attributes and track progress against this profile. The TPP tool is well understood and should always be used by companies developing new therapies. However, the TPP is focused primarily on regulatory approval and is not sufficient to describe fully the commercial attributes of a successful therapeutic. In parallel to the development of the TPP, companies should also consider creating a value hypothesis. The Value Hypothesis A value hypothesis considers the desired global commercial attributes for the therapeutic. As with the TPP, for early-stage programs the value hypothesis will be more general and aspirational in nature, but the real value of the hypothesis for an early-stage development program is in its creation. The development of the hypothesis, the identification of unknowns, and the specific questions that are generated as a result of the analysis, will all guide where early-stage candidates may warrant additional commercialization diligence. While all items will be important eventually, prioritizing where a company should focus limited resources in the value hypothesis is an important decision. A Value Hypothesis The importance of intellectual property (IP) protection in the biopharma industry dictates that IP strategy should be the top priority. Although many companies have competent legal staff handling their IP strategy, many early-stage programs stop with IP and provide limited focus to the other important elements that define commercial value of an early-stage therapeutic (ie. clinical differentiation, pricing/reimbursement, and demand forecasting). An exhaustive analysis of these commercial components is not practical or necessary at the early stages of product development; however, any company seeking to invest $10M or more in getting to a Phase 1 clinical study should have a clear understanding of the commercial assumptions for their product. But a value-hypothesis analysis is extremely valuable and better informs clinical decisions. Starting with a value hypothesis, therapeutic developers will improve clarity of the development/commercialization pathway, enhance confidence in the clinical program, and create a solid understanding to address stakeholder needs. This integration of regulatory and commercial factors will be critically important for product success in the new healthcare-market dynamics. The senior staff at Omni Healthcare Communications have extensive expertise in creating value hypotheses for developmental agents during their experiences while working at pharma companies and as proactive agency partners. Omni welcomes the opportunity to bring an unparalleled depth and breadth of experience to maximize the benefit and value that your product can achieve.
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by Michael Caso Biopharmaceutical companies pride themselves on conducting comprehensive research activities. These could be clinical programs to determine the viability of a new chemical entity to satisfy an unmet medical need, market access programs, or more recently, patient-centric needs of targeted populations. Why then have these same companies virtually ignored the medical, social and cultural needs of the fastest growing segment of the U.S. population? Ethnic and racial minorities currently comprise over 30 percent of the U.S. population and will grow to 50 percent by 2050. In fact, in many geographical areas, minority populations represent an “emergent majority” — a collective majority of minorities. Additionally, this population can have unique responses to medications and are differentially impacted by a number of common disease states for which they are diagnosed later and have poorer control or survival rates than majority populations. It would appear that there exists a nexus of needs and opportunities that should appeal to the biopharmaceutical industry, where a focus on a medical need can result in a healthier population and a healthier bottom line. This is the first of a three-part posting that will provide supportive data that identifies the need, demonstrates targeting opportunities, and provides specific strategies and tactics designed to positively impact complementary health disparity reduction and business goals. By Kevin D. Pawley“Market Access is a dynamic process for building an ever-growing, ever-changing value story that validates a product’s price/position in healthcare practice”—at least, that’s what I claimed in my last article. Trying to see into the future, I’ve been reading, searching, studying, and talking with people to define the term and predict where this area of the healthcare industry, Market Access, is heading. The irony is that Market Access is evolving so rapidly that I settled on a definition that was equally changeable…my definition is more vague than I wanted it to be: it includes three terms—dynamic, ever-growing, ever-changing—that guarantee constant change. But I believe in the foundation of my definition: “Market Access is a process for building a value story.” Logically, the next question is, “What are the building blocks of that value story…how do we construct the story?” I have found answers as simple as “In the US, just build your value story with the Academy of Managed Care Pharmacy’s (AMCP) dossier template.” Or as complex as “Building a Market Access value story is finding the nexus of disease, product, economics, models and markets.” The answer must lie somewhere in between those two: using a “template” and “finding a nexus.” I’ve arrived at an answer to the question—but because of the ever-changing nature of Market Access, I am open to everyone’s input to correct my answer or expand on it, but please don’t just dismiss it! Clearly, Market Access positioning and the value story to support a product in the US begins with the AMCP Format for Formulary Submissions (now in version 4.0)…but I offer the following “high altitude” view in answer to the question, “What are the building blocks of that value story?” The Four “E”s of Market Access Epidemiology: The value of any product begins with a comprehensive understanding of the disease: pathophysiology and presentation; risk factors; diagnostic pathways; scope and impact in the target population; practical impact on healthcare systems; and current clinical options. Epidemiology considerations should include real-world questions of “concomitant conditions” that may come into play…who are the subpopulations commonly found in this group—additional diseases or conditions (renal, hepatic, cardiovascular, etc.) that impact the patient, treatment decisions, or healthcare system capabilities. Ultimately, the epidemiology information needed for inclusion in the value story may be modest compared with the clinical and economic data, but it creates a framework for all the other evidence: who has the disease? How does it affect their lives, their jobs, the people around them? How does it affect the healthcare system they belong to? Efficacy: We can presume that any product for which a value story is being built must have demonstrated its efficacy. But efficacy is a broad topic of consideration: clinical efficacy and effectiveness, safety and tolerability, comparative effectiveness versus standard treatment (where appropriate), and new levels/aspects of efficacy compared to currently available treatments. Importantly, the value of efficacy may be relative—does the product offer a unique aspect to its efficacy? Best-in-class efficacy in a high-risk specific subpopulation? Can its efficacy (ie, responders) be predicted based on genetic or clinical markers? The AMCP Format for Formulary Submissions discusses heterogeneity of treatment effect—"Individual patient variability, variability within populations studied, and variability between clinical studies”—that may be a special consideration in determining the value of a product…perhaps filling a specific unmet need in seemingly refractory patients. Economics: The economic impact of any product is also an ever-changing issue. When a product first enters the market, its economic status is based on modeling, which itself is based on subjective assumptions and modifiable variables. In an economic model, change a price or an incidence of disease or predicted clinical events and the economic picture will change greatly. The solution has been to create several coordinated models—clinical events, life expectancy, and quality-adjusted life-years (QALYs)—to create reliably interpretable scenarios. Over time, the assumptions included in the models give way to or are refined by real-world evidence, moving the data away from subjectivity and closer to reality. Ultimately, the economic value of a product will depend on patient, provider, healthcare-system, disease-state, and treatment variables. Everything Else: For want of a better term, this last category encompasses all of the other potential factors that feed into a “value” proposition. Everything and anything may impact the value story depending on the real-world experience of having, diagnosing, treating, and covering a disease.
Which brings me to my summation: the Four “E”s of Market Access can be summed up with a single E: Evidence. In days gone by, Sales & Marketing of a pharmaceutical product was a matter of “features and benefits.” Today, creating a space for a pharmaceutical product—Market Access—is a matter of “features and evidence.” To tell the ever-changing value story that creates a product’s Market Access, the new world requires that Epidemiology, Efficacy, Economics, and Everything Else be supported by Evidence that proves the case. By Kevin D. Pawley I’ve enjoyed a long career in medical communications, a business that has changed as constantly and quickly as the pharma market that it supports. Among all the constant change has been the rise, expansion, and confusion of a thing called “Market Access.” A long time ago, Market Access was only a matter of “negotiating a price with payers.” As payers began to manage their costs more aggressively—based on utilization data that piled into their databases—the challenges of offering the “right price” grew more complex. Outcomes became an important addition to clinical efficacy; economic impact became an important addition as healthcare costs climbed; now, real-world evidence has provided a powerful perspective as the volume of utilization data has grown to astronomical size and modeling of these data gives it refined meaning. Today, definitions of the term Market Access are as numerous as the people you ask…but everyone will use the word “value” in the definition, maybe not even mentioning the word “price.” You’ll notice, however, that those same people will show frustration with their definitions, because in the end, they’ll have to justify and validate a price…based on an ever-changing value story. Even the Academy of Managed Care Pharmacy (AMCP)—which established the industry standards for evaluating products for formulary placement, coverage, and reimbursement decisions in the US—says that “communications between manufacturers and healthcare decision makers should evolve over the product lifecycle as new evidence becomes available.” While launch-timed dossiers may rely to a greater extent on modeled projections based on clinical trial evidence and reasonable assumptions related to market dynamics and product uptake, new evidence describing the actual use and effect of the product in a real-world setting should be developed to inform formulary management across the product lifecycle. Ongoing generation of real-world evidence serves the important purpose of further defining and validating claims related to product value. (The AMCP Format for Formulary Submissions, Version 4.0; “Foreword”) The resulting definition of Market Access, then, is a dynamic process for building an ever-growing, ever-changing value story that validates a product’s price/position in healthcare practice. “An ever-growing and ever-changing value story” creates a continual challenge to keep the story up-to-date, to educate internal personnel continually, and to communicate with the market about complex real-world evidence…the scope and meaning of Market Access continues to evolve. |
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