Washington DC

The International Society of Pharmacoeconomics and Outcomes Research (ISPOR) is hosting its 21st annual meeting this May in Washington, and I’m excited to see how many sessions, workshops and presentations are focused on the need for more real world data in the clinical development process.

Over five days, thousands of academic, industry and government leaders with gather to discuss health economics and outcomes research, and how we can use real world data to make evidence based decisions that shorten time to market while maintaining the optimal balance between benefits, risks and costs in trial designs.

Closing the evidence gap

The first plenary session directly addresses the need to find this balance when pursuing an accelerated approval pathway. Legislative proposals aimed at accelerating access to biopharmaceutical and other medical products have had bipartisan backing, as well as support from various stakeholder groups. However, these efforts have also drawn fire from opponents concerned they may circumvent patient safety. Several industry experts, including Dr. Scott Gottlieb, resident fellow at the American Enterprise Institute, and Dr. Jerry Avorn, professor of Medicine at Harvard Medical School, will explore the benefits of allowing earlier approval for drugs that treat serious conditions and fill unmet medical needs, as well as the potential risks that can arise from bringing drugs to market with less evidentiary support.

This is an important topic that will help us advance the conversation about how to gather the right evidence as quickly as possible to support the approval and commercialization process, and how developers can use observational data, including patient reported outcomes (PROs), registries, and naturalistic studies to demonstrate continued safety and effectiveness after these treatments come to market.

Making the best use of all available data can help the industry ensure these products are safe and effective for the target population, and provide evidence to support its use in sub-populations, including older patients or those with co-morbidities who may be under-represented in typical randomized controlled trials.

Assessing Value Frameworks

Value frameworks are another important topic being covered through several presentations and posters, including several Quintiles experts. These frameworks, which have been introduced by many health-related organizations, including the American Society of Clinical Oncology (ASCO), European Society for Medical Oncology (ESMO) and National Comprehensive Cancer Network (NCCN), are designed to help industry stakeholders measure and compare the value of different treatments and medical devices so they can make more informed decisions about pathways, pricing and prescribing. These tools represent meaningful progress in our country’s efforts to create a value-based healthcare system that is both objective and transparent in the way it assesses the worth of a product or treatment. However, the framework trend is still in its infancy, and will require much discussion and collaboration amongst industry stakeholders to ensure they function in a consistent, meaningful and balanced way. The current frameworks need to evolve to become more patient-centric and holistic in their evidence appraisal.

My colleagues and I are running a workshop as ISPOR analyzing the heterogeneity of outputs from oncology value frameworks, including ASCO, NCCN and Drugabacus. In this session we will compare the pros and cons of these tools, and demonstrate and how each one can generate different ratings for the same drug, which highlights the need for further discussion about harmonizing these tools and how they should be used, and improved upon, going forward. Value frameworks promise to be an important part of our transition to a value-based healthcare system, but industry leaders need to have open conversations with regulators and the health organizations developing these tools about the evidence and weighting tools being used to support these scores. By getting involved now, industry stakeholders can work collaboratively to ensure these frameworks are fair and balanced. It can also provide sponsors with a better sense of how their products will be rated by these tools so that they can make more informed decisions about the data they collect and the end points they track to meet the value criteria defined by these organizations. We have an opportunity right now to shape the policy environment around the use of valuation frameworks and we can’t afford to waste it. ISPOR will provide a vital platform to progress these conversations.

The patient perspective

Finally, there will be many sessions on the increasing importance of PROs throughout the clinical research process. Biopharma leaders are uniquely positioned to provide insight into how patients navigate the treatment journey in the healthcare system. We need to do a better job of capturing these data, and translating the information into intuitive metrics that provide clear measures of what patients’ value. It is no longer enough to capture anecdotal evidence from the occasional patient interview or panel discussion. PROs need to become a formal part of the evidence gathering process to ensure the patients are heard and that the products being developed deliver benefits that are relevant to their experience and belief system. As one example, my colleague Dr. Jean Paty and his team will present a poster on their work with Astellas on how they were able to enhance interpretation of PRO scores for prostate cancer patients using intuitive metrics. His team developed a standardized measure to compare results from four enzalutamide clinical trials, and translated the data into results that clearly demonstrate the value these patients place on maintaining a high quality of life longer as they live with their disease.  Tying PROs to real world outcomes doesn’t just benefit patients; it ensures that developers are able to prove and communicate value propositions that will be meaningful to regulators, payers, physicians and patients, which can help them differentiate their products from competitors.

These are just a few of the many important themes that will be discussed at ISPOR this year. It’s going to be a groundbreaking event that will further our ability to generate greater value for patients and greater collaboration between industry stakeholders, ensuring we can continue to bring safe and efficacious products to market as quickly and efficiently as possible.

Topics in this blog post: CER, Evidence, Healthcare Cost, HTA, Population Health, Value