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Towards Greater Certainty

Ethan D Leder at United BioSource Corporation discusses the evolving role of evidence in the post-approval biopharmaceutical market

Until recently, the healthcare community operated within a spectrum of ‘acceptable’ uncertainty when it came to using a new prescription medicine. Efficacy and safety data collected from the limited populations that comprise randomised controlled trials were considered the gold standard for understanding the benefits and risks associated with new biopharmaceuticals. For guidance on the appropriate and safe use of new products, doctors, patients and insurers relied on product labels developed by manufacturers in close collaboration with regulatory authorities.

Traditional clinical development programmes continue to provide data that establishes the fundamental safety and efficacy of biopharmaceuticals. Product labels remain the primary mechanism for guiding their use. However, rising demand for continuous data collection, actionable analysis and technology-driven, ‘high touch’ programmes to support the actual use of medicines after regulatory approval has been a catalyst for an emerging breed of service providers focused on helping biopharmaceutical manufacturers address safety issues, characterise product effectiveness and demonstrate value in real-world settings.


As regulators, providers and payers demand more evidence of product performance in the real world, this new generation of scientific and medical affairs organisations are realigning the capabilities of traditional CROs and drawing in specialised expertise from a fragmented array of smaller scientific organisations that have blossomed in recent years. The result is a new breed of service provider with a roster of scientific experts focused on post-approval evidence, nimble and scalable research operations, and reliable technologies that are well-suited to post-marketing scientific needs. Beyond their unique mix of experts and services, these organisations are building innovative partnerships with speciality and retail pharmacies that extend their reach in realworld healthcare and help drive smarter medicine that combines data collection, analysis, technology and targeted or controlled distribution of medicines. The goal is to collect more information, provide analyses and deploy innovative technology that provides actionable information to ensure patient safety, maximise product effectiveness and deliver the best overall value.

Uncertainty is a significant and ever-present reality for the global biopharmaceutical industry and those who prescribe, consume, pay for and regulate its medicines. The industry invests billions to develop and commercialise new biopharmaceuticals; regulators deploy rigorous processes to evaluate these products; and payers seek new methods to measure effectiveness and quantify value. Despite all this work, it is still difficult to predict exactly how a new medicine will perform in the real world until doctors begin prescribing and patients begin taking these drugs.

Historically, data on real-world use of biopharmaceuticals were collected and reported informally, and with varying degrees of rigour, between healthcare professionals, often through programmes sponsored by industry or academia. For manufacturers, the focus during postapproval was on compliance with safetyrelated regulatory obligations, adhering to internal adverse event reporting policies, securing new indications and enhanced product labelling, and advancing the commercial position of the product by disseminating information on its approved indications. Most products were not commercialised with customised risk management programmes and there were few formal processes for evaluating and reporting product effectiveness and value.


For the most part, traditional CROs assisted manufacturers by focusing on executing research projects from product discovery through to approval. These projects typically focused on the design and implementation of early-phase safety studies in animal and human subjects, as well as randomised, controlled clinical trials to secure regulatory approval. Post-approval research was aimed at securing additional indications, addressing specific questions on efficacy or safety, or highlighting product characteristics that could provide commercial opportunity and a competitive advantage. Today, those functions remain important, but manufacturers are increasingly seeking research partners with specialised, ‘high touch’ scientific and analytical capabilities to help with the ongoing management and analysis of products after regulatory approval, as medicines are being prescribed and used by real people outside the highly controlled environment of a traditional clinical trial.

While the need for scientific evidence to drive product commercialisation is not ‘new’ for most industry insiders, demand for this information by regulators, payers and healthcare professionals during post-approval is now continuous and significantly more intense. Events during the last eight years have forced the industry to shift its peri- and post-approval focus from promotion to satisfying new demands for rigorous scientific evidence of safety, effectiveness and value.


Focus on product effectiveness and value in the post-approval phase intensified during the 1990s when mushrooming costs led many healthcare payers – particularly the largest payers managed by governments – to probe the cost-effectiveness and overall value of the most expensive brand name therapies. Generally speaking, Europe led the way, focusing stakeholders on costeffectiveness and overall product value. In the UK, the National Institute for Health and Clinical Excellence (NICE) was established to evaluate medicines and deliver analyses of costs and benefits that would have a direct impact on reimbursement for medicines. In 2004, Germany established the Institute for Quality and Efficiency in Health Care (IQWiG) to perform these evaluations. In the US, rising cost pressure on the state’s Medicaid programmes, combined with the creation of Medicare’s prescription drug benefit, sharpened the focus to cost containment and comparative effectiveness research. The private sector also took aggressive steps to contain the rising costs of healthcare insurance and prescription medicines. Manufacturers faced mounting pressure to provide a constant stream of credible evidence justifying the use of their products.

As the focus on cost and value escalated, the industry faced new pressures on product safety. The withdrawal of Vioxx in 2004, followed by scrutiny of other blockbuster medicines, immediately increased demand for rigorous systems for managing risk and ensuring safety when a new biopharmaceutical is used by large numbers of patients. In Europe, risk management plans (RMPs) became a standard requirement for products approved by the European Medicines Agency (EMA). In the US, public focus on drug safety culminated with passage of the Food and Drug Administration (FDA) Amendments Act of 2007 that brought new requirements for manufacturers to provide risk evaluation and mitigation strategies (REMS) for some products. For products identified as high risk, regulators imposed special safety controls, including elements to assure safe use (ETASU) for some medicines.

In the span of several short years, safety, effectiveness and value – once buzzwords in the industry – became fully embedded as industry standards where manufacturers must focus rigorous scientific evidence to support products in the post-approval market. Healthcare stakeholders – particularly regulators and payers – became increasingly uncomfortable with uncertainty associated with the safety, effectiveness and value of commercialised medicines used by doctors and patients in real-world situations. Manufacturers have responded to this discomfort by concentrating unprecedented levels of resources on peri- and postapproval programmes aimed at proactively identifying and addressing safety issues, guiding effective product use and demonstrating value.


Scientific and medical affairs organisations that have emerged during this transformative period focus on helping biopharmaceutical manufacturers ‘leave less to chance’when it comes to managing new medicines after regulatory approval. UBC, for example, was established based on the assumption that the biopharmaceutical industry would shift its focus to the scientific evidence generation during the post-approval period of the product life cycle. It was, and is still, believed that manufacturers would need more ‘high touch’ programmes that directly engage healthcare professionals and patients to collect, analyse and disseminate scientific evidence on safety, effectiveness and value. Most of the assumptions that form the rationale for building UBC are now reality. While many clients continue to request the stand-alone research, most progressive organisations are building comprehensive evidence generation programmes geared toward managing product use and eliminating much of the scientific uncertainty that has caused friction in the modern biopharmaceutical market.

More of the programmes we deliver embody this evolution of our industry. We have partnered with one manufacturer to design and implement an electronic system that will provide safety monitoring in the US for primary care products that are usually dispensed in retail pharmacies. This ‘hub’ system has been designed to fulfil the prescription management and data collection obligations included in the FDA-mandated REMS, while building a robust database of post-approval information that can be leveraged by the manufacturer to address the ongoing safety, effectiveness and value of the product, throughout its life cycle. For clients with speciality care products, there has been a sharp increase in demand for the ‘hub’ approach, because these programmes are ideally suited for products that require careful safety monitoring, controlled distribution and administration, and often have unique reimbursement challenges.

Medco Health Solutions, one of the world’s largest and most advanced pharmacy operations, recently acquired UBC. In many ways, this acquisition exemplifies the changes that are transforming the demand for scientific evidence in the post-approval market. While UBC operates independently from Medco’s core pharmacy benefit management business, a number of opportunities to provide additional value for clients can be identified. A carefully managed collaboration with Medco’s Accredo, as one of the largest speciality pharmacies in the US, is a valuable feature for a hub programme to support a speciality biopharmaceutical.

Others in our industry share this thinking and have taken action to address market demand for evidence to support the safe and effective use of biopharmaceuticals. In the mid-2000s, WellPoint acquired HealthCore, Inc, a specialised company focused on outcomes research and other scientific services critical during post-approval. Given the significant opportunity to address evidence needs during this critical phase of the product life cycle, traditional CROs such as PPD and Quintiles will almost certainly follow suit by building scientific and operational capabilities focused in the post-approval market.


As manufacturers seek innovative approaches to meet rising demand for evidence, our industry will continue to evolve. As the market adapts to evolving information needs by regulators, providers and payers, organisations that orient scientific expertise, technology and operations toward addressing evidence needs in the post-approval market will be well positioned for success in the modern biopharmaceutical industry.

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Ethan D Leder is founder and Chief Executive Officer of United BioSource Corporation (UBC), a global medical and scientific affairs organisation that partners with life science companies to develop and commercialise their products. Ethan has over 20 years of experience managing organisations in the healthcare industry. Prior to UBC, he was founder and CEO of US Bioservices, a leading provider of distribution services for specialty pharmaceuticals and biologics. In addition, Ethan was a co-founder, President and Chief Investment Officer of Healthcare Financial Partners (HCF; now part of GE Capital), a leading healthcare financial services company. Ethan received his undergraduate degree from Johns Hopkins University and his JD from Georgetown University Law Center.
Ethan D Leder
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