Chris M. Slawecki
DIA Senior Digital Copyeditor

rescribing physicians, patients, and payers around the world have come to recognize that their healthcare systems are increasingly vulnerable to social and economic pressures. Aging populations who have to be treated for chronic diseases for decades, the emergence of groundbreaking immunotherapies and other weapons in the fight against cancer, and other trends, have created a dangerous imbalance between the demands for healthcare delivery and the resources available for that purpose. Will realigning these systems “from volume to value” save them?

Key Takeaways

• Defining an “efficient system” or a “valuable outcome” must be based on what outcomes matter most to the patients these systems serve
• Personalized medicine and digital patient engagement will ultimately deliver therapeutic and cost benefits throughout these systems:
  • Rigorous collection and analysis can convert patient data into compelling evidence
  • Compelling evidence can drive meaningful improvements to standards of care
  • Meaningful improvements to care can drive healthcare policy, pricing, and related decisions
• Value does not automatically convert into price; neither does the reverse.

Why this is important: Innovative new therapeutic options can also introduce new clinical and economic uncertainties. Consequently, innovation in healthcare must mean more than delivering more complex technologies more quickly to more patients; it must also ensure that the system delivering these technologies can sustain them. The safest and most efficacious medicine in the world has no value if patients cannot afford it or access it.

“It’s through the collaboration across multiple stakeholders that we find ways all together to address those new questions and challenges that come to us,” said Claudine Sapède, Global HTA & Payment Policy Lead, F. Hoffmann-La Roche.

Is it FINALLY Time for Big Data?

The comprehensive European research program Big Data for Better Outcomes (BD4BO) aims to improve health outcomes and healthcare systems in Europe by maximizing the potential of big data. BD4BO will develop platforms for integrating and analyzing diverse data sets, focusing on outcomes that matter to patients, and will generate other insights (e.g., protocols, processes, methodologies, metrics, and analytics) that will help improve health care in Europe.

How Can Real-World Data Improve Clinical Trials?

While collection and analysis of real-world data (RWD) is most often discussed in the post-marketing phase, RWD is now improving many aspects of clinical trials:

• Formulate more clinically effective and operationally efficient study designs
• More targeted identification of investigators and study sites
• More predictive recruitment by better matching patients with protocols
• Supplement electronic medical record (EMR) and randomized clinical trial (RCT) data.

Direct Digital Patient Involvement

In addition, digitally captured real-time patient data and related analytics are enhancing new drug development by increasing the accuracy and frequency of data capture, and by asking/answering these and related questions:

• Did the patient take the medicine?
• What else was impacting the patient’s health?
• Did the patient feel better?
• Did the markers of the patient’s disease improve?
• Was it the right medicine for the right patient?

“Patients who define what is important, where is the need — that guides research and development,” Claudine said. “They are at the center and they are also at the receiving end in terms of the medicine that gets to them and whether those medicines [do] get to them.”

This article summarizes key takeaways from sessions in “Topic B: What Are Necessary Steps Towards Outcome-Driven Health Systems?”