Getting Real World About Outcomes Data

I’ve just returned from a cold, blustery and wet London where I attended the annual Pharma Access Leaders Forum.

But the meeting was hot.

In fact, piping hot when you consider the tectonic changes being felt in the world of healthcare technology assessment (HTA) and their implications on both patient access and innovation. And the linkages couldn’t be more profound.

Wither HTA in the EU? A key red thread through a series of potent discussions was real world evidence (aka, “outcomes data”). Head honcho HTA officials from across Europe (including England and Scotland – both still in Europe as of last report) returned again and again to the value of outcomes not just for the evolving world of Risk Sharing Agreements, but for the acceleration of reimbursement science.

Reimbursement science? In July 2012, when Sir Michael Rawlins was chairman of NICE, he told the House of Commons that value, “is based on the collective judgment of the health economists we have approached across the country. “It is,” Sir Michael said, “elusive.”

When it comes to HTA – the times they are a-changin’.

One key insight came from Finn Børlum Kristensen, Chairman of the Executive Committee, European Network for HTA (EUnetHTA), who reminded the conference of JM Eisenberg’s advice, “Globalize the evidence, localize the decision.”

Just as we’ve finally come to realize the urgency of regulatory science (adaptive clinical pathways, imaging, bioinformatics, biomarker validation, 21st century bioequivalence, etc.) to the future of healthcare, so too must we understand and encourage the development of reimbursement science. And high up on the list of items to consider is the validity and utility of outcomes data. We’ve come a long way from the 2007 NICE/Velcade risk-sharing arrangement. Today the stakes are higher and the battle has ratcheted up to a higher level.

When it comes to reimbursement science, we are still in early days. For example, while many HTA bodies in Europe have moved beyond accepting evidence exclusively “the old fashioned way” (via the traditional RCT “gold standard”), there are exceptions – such as Germany. As Detlef Parow (Head of Care Management at DAK-Gesundheit) pointed out, IQWiG will only look at outcomes data “only in exceptional cases.” (This is in keeping with the IQWiG “Principle of Prohibition,” where “Everything is forbidden if it is not expressly permitted.”)

The always-insightful Francois Meyer (Advisor to the President, Director of International Affairs for HAS — France’s Haute Autorité de Santé) wisely suggested more formal early dialogues on scientific advice – similar to the regulatory science initiatives that drive the conversations between FDA and innovators. This would give HTA bodies the opportunity to identify key issues and receive draft company positions, discuss divergent views and try to reach consensus before any final submissions or decisions are made. Sometimes the most common-sense recommendations are the toughest to implement.

But, as with FDA and the advancement of regulatory science, much depends on willingness and ability to implement based on infrastructure, capabilities, and trust. The end goal is the same for all stakeholders – ensuring optimal use of resources for healthcare systems; improving access to value-adding medicines for patients; and appropriate reward for innovation.

In a 2009 white paper, I wrote, “We need to develop proposals that modernize the information used in the evaluation of the value of treatments. Just as the key scientific insights guiding the FDA Critical Path program are genetic variations and biomedical informatics that predict and inform individual responses to treatment, we must establish a science-based process that incorporates the knowledge and tools of personalized medicine in reimbursement decisions: true evidence-based, patient-centric medicine.”

Today, right now, we need a Critical Path for Healthcare Technology Assessment to begin the process of developing a similar list of ways new discoveries and tools (such as electronic patient records) can be used to improve the predictive and prospective nature of clinical outcomes. In an era of personalized medicine, one-size-fits-all treatments and reimbursement strategies are dangerously outdated. Accepting real world evidence does not mean discarding the randomized “gold standard” – it means augmenting it.

It’s a complicated proposition—but such a goal is as simple as it is essential – cost must never be allowed to trump care, and short-term savings must not be allowed to trump long-term outcomes. Just as we need new and better tools for drug development, so too do we need them for HTA.

A health technology assessment model for the 21st Century should reflect and measure individual response to treatment based on the combination of genetic, clinical, and demographic factors that indicate what keep people healthy, improve their health, and prevent disease. A rapidly aging society demands a new healthcare paradigm capable of providing for its needs in the 21st Century. Equality of care must be matched with quality of care.

Peter J. Pitts, a former FDA Associate Commissioner, is President of the Center for Medicine in the Public Interest