Event Schedule
ALL TIMES ARE IN EST

There is positive momentum for using RWE across a spectrum of clinical development, regulatory approval and access. If properly wielded, RWE can boost trust in both the data itself and the outcomes they recommend.

  • Ensure more transparency into the way protocols are designed
  • Detail the procedural aspects of analyzing data
  • Use RWD to answer uncertainties in initial product profile
  • Launch new product data that contextualizes the benefits of the new product

Prepare RWE to Gain Regulatory Approvals and Optimize Clinical Trials Recent successful uses of RWE in regulatory approvals amount to key new case studies that can benefit your own approach. By reviewing key lessons, you can help innovate your methods for clinical trial design and patient recruitment.

  • Consider lessons learned from recent approvals where RWE was used as a complement to a randomized trial
  • Understand when and how to use RWE as a synthetic control, particularly in situations like rare disease and gene therapy
  • Leverage different technologies, as well as databases to systematically target the right sites and patients for clinical trial enrollment

HEOR teams face intricate challenges in their work to show cost effectiveness in variety of different circumstances. Get a bird’s eye view of how they lay out a strategy for submitting clinical and economic evidence in support of formulary considerations using real world evidence.

  • Execute early discussions with payers and decide what to communicate
  • Create a budget impact and model indirect costs
  • Consider standardization of parameters for discussion with payers
  • Improve publication quality using RWE

Collecting real world data (RWD) from Expanded Access programs is of growing interest, but the question of which types of data to collect, how the data may be subsequently useful, and addressing patient needs are all important considerations. Take a deep dive into considerations when collecting RWD and learn which data elements have the capacity to provide real world evidence (RWE), all within the context of Expanded Access programs.

  • Discuss ethical and statistical considerations when collecting RWD
  • Determine ways to ensure RWD data can provide RWE
  • Examine regulatory and practical uses of RWD in Expanded Access programs 

Payers, employers, providers, and regulators are increasingly more receptive to expanding the use of RWE, and RWE is playing an important role in health care decisions and innovation. Now more than ever, demonstrating the value of a therapy is crucial for stakeholders.

  • Learn best practices for communicating health economics / value
  • Understand where health economics / value fit into the larger RWE communication picture
  • Identify the health economics / value needs of stakeholders and how they vary across stakeholder type 

Federated data networks (FDNs) apply novel technology to enable the conduct of RWE studies with multiple partners, without the need to share the individual partner’s data set. Gain insight on which types of research questions can best be addressed through FDNs, specifically in the field of oncology.

  • Raise awareness of the important role that FDNs can play in leveraging readily available RWE to address key research questions of interest in cancer and the benefits to the research community in engaging in federated data initiatives with a long-term perspective.
  • Gain insight into the challenges in combining data from databases with different data structures, variables, and coding practices, particularly in Europe where there is a variety of rich RWD sources of heterogenous nature
  • Assess how FDNs are likely to play a key role in improving knowledge and understanding of real-world treatment patterns and outcomes for patients with cancer

The real world data collected to gain expedited FDA approval of vaccines for the coronavirus is perhaps the largest and best example of why and how to use RWE to inform critical decision making. This case provides very valuable lessons moving forward for handling preventative and therapeutic needs.\

  • Assess the damage the virus has done and implications for stakeholders including hospital networks, payers, regulators and patients
  • Discuss building models to predict how populations are impacted and ideas for how to protect the most vulnerable such as immunocompromised
  • Identify sub cohorts (compare as example Medicaid and commercially insured) which will have higher challenges in front of them and consider how to achieve greater health equity
  • Drive for better data and evidence regarding changing factors including omicron, delta and what’s next 
ALL TIMES ARE IN EST

Approximately 5 percent of stem cell transplant patients are diagnosed with Bronchiolitis Obliterans Syndrome (BOS). Extracting real world evidence from retrospective studies as well as claims data to identify the health economic burden is an incredible challenge. Hear first- hand about the challenges collecting accurate data in rare disorders.

  • Discuss the difficulties using coding data particularly where codes do not always exist in rare diseases
  • Identify limitations of claims data with respect to disease staging or progression
  • Hear about challenges using RWE in finding patients who are trending to potentially get the disease

Dashboards are an excellent tool for assessing data and drawing conclusions. It’s important for an organization to have a solid strategy and framework for understanding what the data means and the best way to use it.

  • Evaluate the metrics used to assess whether the data is fit for purpose
  • Implement a plan to communicate the data analytics
  • Discuss ideas for what should be included in a dashboard

There has been interest from both regulatory and reimbursement bodies outside of the United States around incorporating RWE into their assessments, however, perspectives can differ so it is critical to understand the global environment around this key topic.

  • Learn about integrated data networks e.g. DARWIN
  • Understand the implications from IMPACT HTA, an initiative funded from the European Union’s (EU’s) Horizon 2020 program, is a collaboration between top EU academic institutions and HTA bodies
  • Identify the key aspects from the EMA sponsored article on enabling use of RWE in EU Medicines regulation

Supporting products with the dossier is highly time- intensive. Envisioning in advance the best methods for dossier design can ensure both better representation of your product and the most efficient use of resources.

  • Identify key ingredients for a successful dossier
  • Determine when updates may be needed
  • Consider best ways to represent values and modeling

Now more than ever it is important to expand expertise to facilitate earlier diagnosis and intervention. Tap into ways to use RWE in preventive approaches for providing solutions for patients who are currently in need.

  • See how published data reveals unmet population needs for therapies
  • Target trends about specific patient populations using large data sets
  • Build predictive models to identify undiagnosed and high risk patients

FDA released four new draft guidance documents at the end of 2021 addressing the use of RWE to support regulatory decision-making in drug development. As FDA’s approach to RWE continues to evolve, it is critical to stay abreast of these developments and take FDA’s new draft guidance into account when planning for and executing RWE initiatives.

  • Analyze FDA’s new RWE draft guidance documents addressing (1) electronic health records and claims data, (2) registry studies, (3) data standards for RWE submissions to FDA, and (4) considerations for the use of RWE to support regulatory decision-making
  • Key remaining questions and challenges with the use of RWE to support regulatory decision-making in drug development
  • Next from FDA on RWE

Drug-based treatments and most medical device treatments face a challenge collecting prospective and accurate real-world data regarding the usage, efficacy, and safety of their therapy. Hear how Theranica copes with this challenge by strongly coupling the medical device with a digital health app (Nerivio):

  • Discuss how a medical device can be fully controlled, in time and space, by a digital health app to provide prospective, accurate real-world disease and treatment data
  • Familiarize with passive and active methods of data collection from users, and data sharing with users (and providers) for better disease management
  • Learn about the largest real-world evidence analysis of a migraine device, with data collected over 20 months, from over 120,000 treatments by over 12,000 users