Real World Data Europe

Apr 28, 2014 - Apr 29, 2014, London, England

Demonstrate the true effectiveness of your drugs to satisfy payers, HTAs and improve patient outcomes

Embracing the Age of Data-Driven Medicine

Deirdre Coleman assesses the challenges Real World Evidence poses for pharma and the path towards gaining meaningful insight from its usage.



The value of Real World Evidence is undeniable and provides the missing pieces in the puzzle of delivering high quality healthcare in an efficient way; yet we are only at the infancy stage of leveraging the opportunities it opens up for all stakeholders. Getting the architecture in place to utilise real world evidence from disparate systems and realize its value is one such challenge but the greater challenge for pharma is employing an active role at the table alongside other stakeholders and moving towards the thoughtful integration of Real World Evidence into their processes to deliver patient insight and inform strategy.

Challenges to Pharma

According to Jon Resnick, Vice President and General Manager, Real World Evidence solutions for IMS Health, one of the key challenges for pharma will be the change in mindset required to truly embrace Real World Evidence and the opportunities it presents:

“Traditionally, much of the work already completed for market access at the time of launch was based on evidence from randomized clinical trials (RCTs). While these remain the gold standard for demonstrating product safety and efficacy, the limitations of their data such as small sample size, controlled environment and focus on short-term outcomes meant negotiations were primarily focused on qualitative documentation of value. Quantification was mainly extrapolated using health economic modeling. Increased payer interest in Real World Evidence (RWE) reflects a shift toward more quantitative measurements of value and acceptance of data sources and endpoints that are not explicitly derived from RCTs. This comes with growing acknowledgment that while RWE may have limitations in data quality relative to RCTs, its findings are more relevant to the payer perspective because it reflects actual use and effectiveness in their patient population. Regulators, too, recognize the value of RWE on safety, relative to that offered by RCTs. The future for RWE is one of certain challenge for all key stakeholders as scientific advancements and external dynamics place increasing demands on decision making. However, it is also one of tremendous potential for greater insight into patient care as RWE use becomes more standardized”.

The biggest threat from RWE is to ignore it because our customers, payers and providers will use it.  Pharma needs to  participate in the process and collaborate on how best manage to it".

The field of Real World Evidence is still quite young and there are a number of challenges within the healthcare environment to be faced as the phenomenon of RWE gains momentum. “The evolution of a regulatory framework surrounding the appropriate use of RWE, appropriate methodologies as to how best to structure studies, technology challenges – how to collect and synthesize data sets, all need to evolve and over time these will be worked out. Internally within pharma, there is a long legacy of working with RCTs and a certain level of comfort around it. It will take time to work out where RWE best fits within the organisation and how to get the most out of it. It will impact on all functions within the organisation, R&D, medical affairs, commercial and it will be a process of working out how to scale different studies in different markets. There is no simple answer, it will be a process of experimentation, trial and error to work out how it fits within the structure of the organisation, but as the external barriers come down it will require time and investment internally to get to scale and learn how to leverage it. The biggest threat from RWE is to ignore it because our customers, payers and providers will use it.  Pharma needs to  participate in the process and collaborate on how best manage to it”, comments Resnick.

Increased Complexity

Current scientific and regulatory approaches to marketing authorization generally utilize randomized controlled trials (RCTs) to provide information on safety and efficacy, yet real world evidence such as effectiveness studies are required to have comprehensive information about the product in daily practice.

 Taken together, these issues have led to more data being required during the initial development of a new product. To meet this demand, pharmaceutical firms have increased their investment both in research and development and in the number, size, duration, and design complexity of clinical trials.

According to Bernadette Hendrickx, Senior Medical and Scientific Advisor to the CEO, Sanofi Pasteur: “Real world data and its incorporation will only increase in importance in the coming years which will have implications on all areas from development through to post-licensing; the onus will be on pharma to develop value propositions supported by real world evidence in collaboration with regulators and payers”.

Under the traditional regulatory paradigm, the life span of a drug is divided into two distinct phases: pre-licensing and post-licensing. During the pre-licensing phase, patients are exposed to a new drug only if they enroll in clinical trials with informed consent procedures, meet specific enrollment criteria, and are randomized to the investigational product. The situation changes abruptly upon licensure. Often, this single event expands the exposure of a new drug from a relatively small number of selected trial subjects to millions of real-world patients who might not fit treatment eligibility requirements as specified in the label or the clinical trials. The unpredictability of the confounded real-world populations and usage combined with the unrealistic expectation of perpetual safety based on the extrapolation of limited data is not generally acknowledged in the current regulatory-decision framework.

The costs and resources for the post-licensure data in many cases is as significant as the production of pre-licensure data".

The demands of Real World Data are therefore many. “The world has changed vastly in the last year, mainly due to safety issues. The clinical data you submit to obtain a registration is now much more important than it was previously. The risk management plan that is now required by the authorities is to have much larger clinical studies looking at safety and effectiveness in a real world environment.  The costs and resources for the post-licensure data in many cases is as significant as the production of pre-licensure data”, explains Hendrickx.

The late phase research environment has changed dramatically in recent years. Regulators are demanding a much more proactive approach to safety and risk management. Addressing those mandates requires longer studies, larger patient pools and comprehensive risk evaluation and mitigation strategies. Payers likewise are demanding data to support their therapeutic choices. These demands, and the ever-expanding pool of data sources, have made late phase research studies increasingly important and complex. Effective planning must include a comprehensive strategy for identifying and addressing the therapeutic, regulatory and economic concerns of diverse healthcare stakeholders. Failing to take early action and develop an integrated RWD strategy can result in expensive missteps, wasted time and failure to obtain drug approval.


Bernadette Hendrickx will be speaking at eyeforpharma's Real World Data Europe in April. For more information on her presentation, click here.

 

 



Real World Data Europe

Apr 28, 2014 - Apr 29, 2014, London, England

Demonstrate the true effectiveness of your drugs to satisfy payers, HTAs and improve patient outcomes