The Effective Statistician - in association with PSI

Interview with Rachel Tham What is deep work When have you stepped over this concept of deep work? Which aspects of deep work have helped you most? Were there any guidances in deep work, that you found difficult to follow? How do you use deep work concepts currently in your day-to-day work? References: Deep Work Atomic Habits

Interview with Anja Schiel Are you seeing a return to single arm studies from an HTA perspective? What potential drawbacks should be considered when opting for non-randomized comparative trials instead of randomized control ones? If a full RCT isn't feasible, what other viable approaches are available as substitutes? How can sponsors best communicate their research with HTAs on these alternative solutions and explain the value they present in comparison to an RTC design study? Listen to this episode and share them with your friends and colleagues!

Interview with Heidi Seibold Document Everything Create Reusable Code Share Results with Others Making sure that your research is reproducible is important in the pharma industry because it allows others to understand what you have done and how you arrived at those conclusions. By following these three steps—documenting everything, creating reusable code, and sharing results—you will be able to make sure that your research is reproducible for anyone who needs access to it now or in the future! Resource: Transparimed Share this episode with your friends and colleagues who can learn from this!

Katrin Kupas and Jen Min-Hua discuss the role of health economics in EU HTA, the influence of joint clinical assessment on economic modeling, the need for pre-specification of statistical analyses, and the importance of statisticians having a broader perspective in health economics.

Interview with Katrin Kupas and Dr. Arthur Allignol All statisticians in the Pharmaceutical Industry will be impacted by the new EU HTA regulation. Activities around HTA submissions will happen earlier than currently, in parallel to the regulatory approval process for marketing authorization. There will be an increased scope of evidence for the joint clinical assessment to fulfill the needs of all EU member states, and so there will be a large package of statistical analyses that need to be provided in addition to the submission to the regulatory bodies. This will redefine how you, as a statistician, work, and with whom you need to collaborate. Both HTA and clinical development statisticians will need to join forces to define the value story for the complete lifecycle of the drug. HTA specific analyses will need to be planned in parallel with clinical development. In this episode, we will discuss the future skill sets that statisticians in the pharmaceutical industry need to adopt with the new EU HTA regulation being applied in 2025 already.

Dr. Arthur Allignol, a statistician working in a regulatory setting, and Anders Gorst Rasmussen discuss the role of estimands beyond regulatory approval, particularly in a Health Technology Assessment (HTA) setting. They explore current draft guidelines for EU HTA and the challenges that estimands pose in treatment policy estimates. The chapter also emphasizes the importance of specificity in research questions and the involvement of statisticians in the HTA process.

Bonus Episode The Effective Statistician will be hosting their first full conference on April 25th, 2023 as a virtual event. The conference is free to attend and will be five hours long, with a variety of presentations ranging from 15-20 minutes each. Some of the topics that will be covered include estimands, bayesian approaches in early development, medical affairs, digital health applications, study program simulation, influencing skills, and data visualization. For more information or to register for the conference visit: theeffectivestatistician.com To learn more about the conference, CLICK HERE. If you want to sponsor the conference, CLICK HERE for the details.

Interview with Lara Wolfson and Anders Gorst-Rasmussen References: D4.5 - EUnetHTA Antonia Morga, Nicholas R. Latimer, Martin Scott, Neil Hawkins, Michael Schlichting, Jixian Wang, Is Intention to Treat Still the Gold Standard or Should Health Technology Assessment Agencies Embrace a Broader Estimands Framework?: Insights and Perspectives From the National Institute for Health and Care Excellence and Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen on the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use E9 (R1) Addendum. Value in Health. S1098-3015(22)02148-9. doi: 10.1016/j.jval.2022.08.008.

Interview with Greg Ball The US FDA has been advocating for aggregate program-level safety evaluations since 1988 when the ISS was introduced (the IND Safety Reporting Final Rule). Aggregate data analysis looks at all available data holistically rather than relying on individual case reports. This allows for a more complete understanding of potential risks associated with a drug or vaccine. In the near future, more companies will adopt a systematic approach to aggregate safety assessment planning and IND safety reporting. This process will develop a better understanding of the safety profile of drugs and ensure that patients' safety is well-thought-of.