4 agile ways policy-makers can advance precision medicine

This article is brought to you thanks to the collaboration of The European Sting with the World Economic Forum.

Author: Kevin Doxzen, Hoffmann Fellow, Precision Medicine and Emerging Biotechnologies, World Economic Forum, Landry Signé, Professor and managing director, Thunderbird School of Global Management & Diana Bowman, Associate Dean and Professor, ASU Law, Arizona State University

• A combination of technological, ethical, and regulatory challenges has limited the impact of precision medicine.
• To overcome this, investment in health care innovation is vital, but the risks need to be managed with agile governance.
• Governments should work with industry, build on best practice, and collaborate internationally to ethically accelerate innovation.

A new report from the Brookings Institution explores how agile governance can help promote innovation in precision medicine while protecting the public from unintended consequences. Precision medicine uses personal information, such as DNA sequences, to prevent, diagnose, and treat disease. This can present major challenges, including genomic data privacy, clinical trial design, and equitable therapeutic delivery. The report presents global case studies demonstrating how governments, companies, and other stakeholders are using agile governance processes related to healthcare, and asks what the implications could be for precision medicine.

Technological innovation can quickly outpace laws and regulation. This dilemma is known as the “pacing problem” and poses unique problems for governments looking to develop advanced technologies, while making sure that risks are managed with the right policies. Precision medicine has been vulnerable to the “pacing problem” – but agile governance could help harness it for the public good. Building on a blueprint by Landry Signé and Steve Almond, this new report explores the roles that agile governance can play in overcoming the regulatory challenges posed by precision medicine.

The precision medicine pipleline

Precision medicine can be thought of as a pipeline, moving from research and development in labs to treating patients in hospitals. The report divides the pipeline into four stages, each of which has its own ethical, legal, and technological challenges, which could benefit from agile governance.

Data acquisition and storage

Large genomic data sets help form the foundation on which health discoveries are made and personalized therapeutics are designed. However, acquiring and storing personal information is fraught with a number of ethical concerns. For example, some indigenous communities shared concerns about data protection and ownership in the US’s All of Us research program. The use of agile governance tools could help create dynamic systems and infrastructure that allow indigenous groups to keep ownership of their genetic information, while still benefiting from corporate innovation.

Information access and research

Understanding how genetic information is accessed and anticipating how it may be used in domestic and international research is vital to creating effective regulatory frameworks to safeguard human rights and personal privacy. For example, the UK’s Human Fertilisation & Embryology Authority uses horizon scanning exercises to anticipate how precision medicine tools may alter the future of reproduction both inside and outside the UK.

Transforming clinical trials

Precision medicine is a powerful approach for treating rare diseases, but small patient populations can make it difficult to design and carry out clinical trials. Industry-led coalitions like the Decentralized Trials Research Alliance are transforming how international clinical trials are conducted.

Societal benefit

Precision medicine treatments, particularly cell and gene therapies, can be prohibitively expensive, limiting their societal benefit. The US’s Center for Medicare and Medicaid Innovation is testing innovative health care payment models by partnering with states to help improve access to life-saving treatments.

Recommendations for policymakers

Precision medicine has not been integrated into healthcare systems as quickly as patients, doctors, technology developers, or governments had hoped. In part, this is due outstanding scientific research questions and technological limitations, but there has also been a lack of coordination. There are four key ways that policy-makers can implement agile governance processes to advance precision medicine.

1. Engage the market

Governing bodies must work with industry to maximise the public benefits of precision medicine. This vital line of communication accelerates innovation by ensuring that bold ideas fit within safety standards imposed by regulatory agencies, before companies set on product development and testing.

Active engagement was imperative for Novartis, an international pharmaceutical company, to bring a gene therapy for vision loss to the UK market. Meindert Boysen, director of the Centre for Health Technology Evaluation at the National Institute for Health and Care Excellence, said: “the company’s willingness to work with us early and constructively has allowed us to publish this guidance on a much faster timeline than normal, which is good news for patients.”

2. Build on good practice

Projects that incorporate the public into the research process tend to align best with community needs. For example, Canada’s Silent Genomes Project is the result of years of relationship-building, and it reduces access barriers to diagnosis of genetic disease in Indigenous children by working with local communities.

Health and healthcare

How is the World Economic Forum bringing data-driven healthcare to life?

The application of “precision medicine” to save and improve lives relies on good-quality, easily-accessible data on everything from our DNA to lifestyle and environmental factors. The opposite to a one-size-fits-all healthcare system, it has vast, untapped potential to transform the treatment and prediction of rare diseases—and disease in general.

But there is no global governance framework for such data and no common data portal. This is a problem that contributes to the premature deaths of hundreds of millions of rare-disease patients worldwide.

The World Economic Forum’s Breaking Barriers to Health Data Governance initiative is focused on creating, testing and growing a framework to support effective and responsible access – across borders – to sensitive health data for the treatment and diagnosis of rare diseases.

The data will be shared via a “federated data system”: a decentralized approach that allows different institutions to access each other’s data without that data ever leaving the organization it originated from. This is done via an application programming interface and strikes a balance between simply pooling data (posing security concerns) and limiting access completely.

The project is a collaboration between entities in the UK (Genomics England), Australia (Australian Genomics Health Alliance), Canada (Genomics4RD), and the US (Intermountain Healthcare).

3. Think holistically

Precision medicine relies upon personalized care delivery, an aspirational goal for many countries. Governments aiming to develop and maintain precision medicine must take a holistic perspective, monitoring each step from beginning to end. This will help them to identify weaknesses that may require attention and investment, and also protect against disruptions in the precision medicine pipeline.

The COVID-19 pandemic was one such disruption, impacting many countries’ ability to develop, test, and deploy precision medicine-based health care. To increase resilience, innovators and regulators may need to adjust clinical trial protocols, such as incorporating telemedicine, to minimize patient travel.

4. Evaluate and learn

Effective governance of precision medicine is a balance between promoting innovation and preventing unnecessary risk. Governments must continually evaluate this balance either through periodic auditing or maintaining a direct line of communication with innovators, constituents, and other stakeholders. Iterative improvements based on key learnings can help policy keep pace with rapid technological advancements.

In Switzerland, the Geneva public hospital system implemented the Health Information Exchange project to store personal data in a centralized database. After pushback from the public, it was replaced by a fully distributed database, which people could opt in to. The Swiss government evaluated its healthcare infrastructure and the trust of its users, using this to update systems based on new technologies.

Landry Signé is professor and managing director at Thunderbird School of Global Management, a senior fellow at the Brookings Institution, a distinguished fellow at Stanford University, and a World Economic Forum Young Global Leader

Kevin Doxzen is Hoffmann Fellow, Precision Medicine and Emerging Biotechnologies, World Economic Forum; Thunderbird School of Global Management; ASU Law