Predict Outcomes within the Health Plan Population and Identify Key Risks

It’s that time of year where we’re busy heading into the final quarter of the year. In 2018, we have witnessed a fundamental change in the concept of value, going far beyond pharmacological properties and clinical effects, to not only to include patient value, quality of life and patient management, but also to integrate systems and societal value dimensions more broadly.Where are we in our journey towards the new frontier in the ‘paying for value’ discussion in the US?

Stage 2 Predict Outcomes within the Health Plan Population and Identify Key Risks

Therapies often perform differently in the real-life compared to the clinical trial setting. The phenomenon has been described as the “effi cacy-toeffectiveness” gap mentioned earlier. A commonly-held belief is that performance is always reduced in the real-life, but there are numerous examples of treatments performing better than in the RCT (e.g., long acting dopamine agonists in schizophrenia, anti-IgE mAb in asthma). There are several factors that can infl uence real-life effectiveness, and they can be grouped into two categories: drug use factors and patient population factors. Drug use factors include patterns of use, dose, duration of treatment, past history of exposure, co-prescription, and adherence to treatment. Patient population factors include age, gender, behaviors, comorbidities, disease stage and severity, genetic and risk factors relevant to the disease. Furthermore, these factors are in turn influenced by the health system (e.g., coverage/reimbursement, medical practice, or screening policies influence which patients receive a treatment and how it is used). The so called “Drivers of Effectiveness” (DoE) framework, which originated from Analytica Lasers’ participation in “GetReal”, part of the EU Innovative Medicines Initiative, is illustrated in Figure 18.

For real-life effectiveness to differ from clinical efficacy, the distribution of patients along some of these factors has to differ significantly in the real-life compared to the clinical trial setting and there needs to be a meaningful interaction between the factor(s) and the outcome. Based on our experience, there is usually a limited set of factors that truly influence effectiveness and the interacting effects are mostly universal. The relevant factors can be identified through previous studies reported in the literature, sometimes based on your own clinical data, or by setting up ad-hoc studies.

What needs to be remembered is that the interaction between these factors and the drug efficacy is actually the same – whether you are looking at pivotal clinical trials or the real world. While the drug properties don’t change from population to population, the distribution of risk factors and effect modifiers does. That is why drug effectiveness appears different in the OBA context. A clear understanding how this framework plays out in the relevant context enables robust estimates of effectiveness as we concentrate on exactly these interactions. Of course, the more data that can be integrated, both ‘on the drug’ and on informing the characteristic of the population in real life, the better.

Once the factors are identified and their effects understood, it becomes possible to build a model that will be able to predict the real-life effectiveness. This model can then be used for different countries and health plans, provided the distribution of the influencing factors is understood in the populations of interest. The key benefit of the predictive approach is that it enables the manufacturer to quantify the multiple sources of variance and uncertainty that will be encountered in the execution of an outcomes-based agreement, which are related to:

■ the difference between the real-life versus clinical trial setting discussed above
■ the inter-patient variability
■ the effect of time, including both the intrinsic evolution of the disease and outcome, as well as potential changes in how, or by whom, the drug is being used.

While any model in itself does not reduce the uncertainty (without which an OBA would be rather pointless), it is essential to understand source and magnitude in order to frame and provision the contract adequately and manage the risk properly. Once the model offers a solid foundation of your understanding on real world performance, it can be useful to set up a baseline pilot for a preliminary discussion of mutual perspectives, data management and financial implications (paid for under “Fair Market Value” fees for staff time and resource needs to analyze data). This would precede the execution of the full-fledged agreement and can enhance further stages of the implementation.

Meet Our Experts

Ulrich Neumann
Ulrich NeumannSr. Director & Head of US Value & Access
Ulrich Neumann is the Senior Commercial Director of Analytica Laser, a global research consultancy with offices in eight countries. In addition to client advisory in US-based market access, his responsibilities include corporate affairs, marketing, product development and customer-facing growth strategies across all of the company’s target markets. Analytica is part of Certara, the global leader in model-informed drug development with over 700 professionals in 20+ countries.

With a dual background in business management and public administration, Ulrich focuses his work as a pharmaceutical market access expert on reimbursement and pricing, as well as activities on the US political, legislative and regulatory landscape to assess policy drivers, enablers and challenges to market access. He has given conference keynotes and university seminars, published research in whitepapers and articles in the pharmaceutical press, as well as two books in the field of media and terrorism policy.

His business experience lies in defining product strategy, value messaging and brand development. The founder of several ventures, Ulrich has worked on go-to-market projects for close to 15 years and most recently ran the US division of a respected global provider of pharma market insights, b2b networking and conferences. Ulrich is a graduate of the London School of Economics (MSc), University of Southern California (MA), University Twente (BSc) and University of Muenster (BA). At USC, he held the Roger Silverstone Fellowship, the Annenberg School’s highest-sponsored grant, conducting quantitative and qualitative research on the economics of digital platform markets, competitive strategy as well as marketing and communications strategies. Active in the NY health tech community, Ulrich is a supporter of the open health data movement and member of ISPOR and DIA. He is a nominated Fellow of the Royal Society of Arts and Commerce since 2014.

Isha Bangia
Isha BangiaUS Access Specialist
Isha brings a combination of clinical and strategic experience to Analytica. She holds a PharmD from Rutgers University and an MBA from Johns Hopkins. Her background as a pharmacist provides clinical perspective into retail and hospital settings where she has been involved in market access and clinical care at the provider level. Her managed care expertise includes work at Hunterdon Medical Center, Bayshore Community Hospital, Princeton Houser Psychiatry, Hackensack University Medical Center, Bayonne Medical Center. Isha has also participated in P&T committee meetings for formulary decisions. Prior to joining Analytica, Isha was a consultant at Prescient Healthcare Group, focused on competitive landscaping and go-to-market strategies. She has past experience from Zitter Health Insights where her work focused on payer primary and secondary market research to better understand market access barriers (e.g. step therapies and prior authorizations) for manufacturers. She has worked across multiple therapeutic areas including oncology, biosimilars, diabetes, vaccines and rare diseases.

Roman Casciano
Roman CascianoGeneral Manager of Analytica Laser & SVP, Market Access, HEOR and Real-world Evidence, Certara
Roman Casciano is the General Manager of Analytica Laser, leading Certara’s Market Access, HEOR and Real-world Evidence Group in the capacity of Senior Vice President. Based in New York City, Roman’s role comprises the executive management of all strategic and scientific teams across the company’s eight offices and three divisions. Roman continues to actively participate in client engagements as a market access strategist, ensuring that customers can leverage all of the benefits of our multidisciplinary team and integrated offer.

Roman was one of the founding members of the Analytica Group in 1997 and remained with the company for over 14 years, serving in various capacities during his tenure ultimately serving as President from 2008 onwards. Prior to joining Analytica, Roman worked for 4 years as an environmental engineering consultant with HDR Engineering Inc. in White Plains, NY. Roman went on to participate in the founding of the Analytica Group in 1997, initially serving as Director of Operations and subsequently serving as General Manager for the German subsidiary from 2004-2006. After returning to the US from his tenure as General Manager in the German subsidiary and prior to taking on his role as President of Analytica, Roman worked for 2 years in Analytica’s previous parent company, Accentia Biopharmaceuticals as the Sr Vice President and head of the NY-based product development team with full responsibility for the conduct of its two on-going phase III clinical programs.

As an applied health economist and market access strategist, Roman has personally led hundreds of engagements in the international market access and HEOR context related to product value demonstration and has deep experience in the management of multidisciplinary teams of scientific and strategic consultants across continents. Roman’s experience ranges from analytical activities such as economic modelling and database studies, to strategic activities such as developing product pricing and market access plans and communicating evidence internally and externally for our customers. Roman has experience across the product lifecycle, including assessment of early phase assets, licensing due diligence, and go/no-go decision-making support, as well as launch planning and commercialization activities.

Roman received both a Bachelor of Science degree in Mechanical and Aerospace Engineering and a Master of Engineering Degree in Mechanical Engineering and Engineering Decision Making from Cornell University.

Lee Stern
Lee Stern Global Vice President, Business Development
Lee Stern is a Vice President at ANALYTICA LASER, formerly Senior Vice President of Analytica International. In this role, she is responsible for all health economic and market access engagements for global clients. She also oversees the full operations of the New York office. Lee is a strategic and actionable leader managing highly qualified and scientifically robust cross-functional and geographically disparate teams.

Her expertise in solving client issues via action-oriented deliverables is a strong asset in the context of a cost conscious payer environment. She has extensive experience in global market access and launching products in competitive landscapes. As a strong operations leader, Lee is able to deploy a team of specialized scientists to ensure timely, relevant and scientifically robust deliverables for her clients. Her 10+ years of experience in the industry enables her to draw upon a vast repertoire of project deliverables to produce solutions for complex study designs and market access issues. For example, she has coordinated multiple local country submissions for major oncology products, managed international payer research in diabetes, and published numerous retrospective data analyses, models and literature review studies in peer-reviewed journals.

Prior to joining ANALYTICA LASER, Lee served as the primary nutritionist in a large physician practice, closely affiliated with NYU Langone Medical Center where she has maintained close ties.

Lee received her Bachelor of Arts degree in Neuroscience at The University of Pennsylvania. She also completed her Masters in Clinical Nutrition at New York University School of Education.

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