Leveraging the latest technologies to improve treatment regimens, store patient data, and track outcomes is essential for a country to reach their healthcare goals amidst changing environmental, economic, and social conditions.
Precision medicine uses personal information, such as DNA sequences, to prevent, diagnose, or treat disease. From targeting late-stage cancers to curing rare genetic diseases, precision medicine is poised to impact millions of people within the next decade. Despite such promise, this form of healthcare is not without unique challenges.
…traditional governance and oversight mechanisms… can successfully regulate and monitor advancements in healthcare, additional processes, collectively referred to as “agile governance,” play a vital role in helping governments keep pace with emerging technologies while serving the greater good. Agile governance, as defined by the World Economic Forum, “calls for a multistakeholder effort, in which the public and private sectors collaborate to develop forward-thinking solutions to encourage industry growth, while addressing the concerns of a fast-paced digital world.” Agile governance also ensures accountability through human-centric processes designed for the benefit of citizens.
Precision medicine would benefit from agile governance processes to improve government coordination, leverage public-private partnerships, and actualize the potential of personalized healthcare without further exacerbating health inequity.
…the recent Agile Nations agreement between Canada, Denmark, Italy, Japan, Singapore, UAR, and U.K. aims to foster multi-jurisdictional cooperation by “helping innovators navigate each country’s rules, test new ideas with regulators and scale them across the seven markets.” Similarly, the Global Financial Innovation Network (GFIN), composed of over 60 organizations, was established to help firms “test innovative products, services or business models across more than one jurisdiction.”
…the World Economic Forum defines agile governance as adaptive, human-centered, inclusive and sustainable policy-making, which acknowledges that policy development is no longer limited to governments but rather is an increasingly multistakeholder effort.
In “A blueprint for technology governance in the post-pandemic world,” Landry Signé and Steven Almond provide seven pillars (processes) constituting a blueprint for regulators seeking to respond effectively and efficiently to technological innovation worldwide (see Table 1 below).
- Anticipate innovation and its implications.
Regulators who can employ foresight mechanisms will be better able to capitalize on opportunities that new technologies create while mitigating the risks.
Although precision medicine holds considerable promise for treating previously incurable diseases, governments must consider its full spectrum of uses and implications to maximize benefits while minimizing undue risk. …governments are using anticipatory governance processes to consider both the short- and long-term implications of precision medicine on individual and society-levels.
Singapore’s Ministry of Health setup the Agency for Care Effectiveness, which launched a horizon scanning system (HSS) in 2019 to identify emerging technologies and predict their impact on patient health and costs to society and Singapore’s healthcare system.
- Focus regulations on outcomes.
This promotes innovation by allowing businesses to achieve regulatory goals in the most efficient ways, while still complying to guidelines.
The EU fast-tracked vaccine development by temporarily suspending certain clinical trial restrictions, recognizing the need to adapt rules in favor of essential outcomes. Within these agile processes, vaccines were still appropriately tested for safety and efficacy.
With a focus on outcomes over processes, the FDA is considering virtual inspections, which would minimize delays of future approvals and allow simultaneous inspections by multiple agencies at once.
- Create space to experiment.
…regulation should not be written in isolation but instead developed in conjunction with the technology it ultimately seeks to regulate.
Once therapies make their through regulatory approval and reach market, payers and providers must negotiate how to bring these treatments to patients. Rather than paying for volume, healthcare systems are looking to pay for value, which requires new payment and financing models.
…government provides the funding, learning tools, and expert technical assistance, while the states “utilize policy and regulatory levers to accelerate health system transformation.”
- Use data to target interventions.
…finely targeted regulatory interventions would grant businesses greater flexibility to innovate, as regulators can rapidly intervene through data-driven technologies.
Data does not only form the foundation of precision medicine; it can also be used to improve regulation. … A broader perspective is particularly useful in healthcare, especially when a country’s medical system is overseen by several different agencies, regulated under multiple frameworks, and decentralized across cities and states.
Research and development programs are fragmented across 14 different ministries within the Korean government. … the Korean Research and Development Platform for Investment and Evaluation (R&D PIE), uses big data analytics and machine learning to identify overlaps and potential opportunities across Korean ministries.
In addition to intra-governmental insight, data can provide a valuable perspective across countries. The FutureProofing Healthcare initiative monitors and quantifies the sustainability of healthcare systems across the EU, Africa, and Asia Pacific regions.
- Leverage the role of business.
Regulators need to harness the role that the private sector can play in the governance of innovation.
The market for direct-to-consumer genetic testing offered the public a new way to explore personal health, yet despite the reach of consumer genetics companies this sector suffers from a lack of regulation. …a nonprofit promoting data privacy, partnered with consumer genetics companies to announce best practices for protecting consumer genetic data privacy.
One example of coalition movement is the Decentralized Trials Research Alliance (DTRA), which unites healthcare companies, regulators, and patient groups across jurisdictions to improve clinical trial participation, through identifying best practices, resolving regulatory gaps, and building a data and knowledge repository.
- Work across institutional boundaries.
…a more localized “one-stop-shop” introduced by governments would help companies to engage more directly with national regulators.
Establishing one-stop points of guidance, where companies can receive feedback on product plans, helps streamline approval processes and promotes innovation.
The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) established an Innovation Office that fosters dialogue across institutional boundaries, providing advice on how to navigate U.K.’s regulatory landscape. The Innovation Office offers advice that can save precision medicine companies significant time and money.
- Collaborate internationally.
Cooperating across borders allows regulators to address shared challenges, share foresight, experiment jointly, and facilitate trade and investment more efficiently.
When bringing precision medicine to international markets, companies must navigate complex networks of policies, standards, and approvals. International coordination between regulators and in consultation with companies can accelerate innovation and improve patient access, while simultaneously preserving safety standards.
A model example of international coordination is the Access Consortium, a coalition of regulatory authorities from Australia, Canada, Singapore, Switzerland, and the United Kingdom which forges international cooperation through information and work sharing, reducing duplicative efforts and increasing each agency’s functional capacity.
The European Medicines Agency (EMA) and the U.S.’s FDA support a parallel advice program in which companies can engage with both regulatory agencies early on or prior to product development.
Applying agile governance processes can follow two unique methods designed to achieve a more adaptive and coordinated approach to regulation in healthcare. A “design method” can identify and address problems as they arise- for example, adjusting clinical trial design during a global pandemic. In contrast, a “system method” takes into account the whole system and addresses foundational problems.
It can create robust frameworks that can solve multiple issues –for example, establishing cross-national systems between regulatory bodies to more quickly and efficiently approve personalized therapies.
Countries looking to leverage agile governance process to help integrate precision medicine in their healthcare systems must begin by understanding the unique challenges posed by genomics-based medicine.
Precision medicine is not yet a common route for disease diagnosis and treatment, an unfortunate reality for those confronted with cancer, rare genetic disorders, or neurodegenerative diseases. …precision medicine entering the clinic requires a critical examination of the precision medicine “pipeline”:
- Data acquisition and storage
- Information access and research
- Clinical trials and commercialization
- Societal benefit
Big data forms the foundation of precision medicine. …Obtaining this information from diverse cohorts, including underrepresented minority groups, raises concerns of data ownership and privacy. Once acquired, DNA sequencing data is stored in a wide variety of formats across thousands of online repositories, ranging from small academic databases to large repositories controlled by companies like 23&Me. …variability in formatting and nomenclature can make it difficult to merge data across repositories and integrate files with electronic health records (EHR). Storing genomic information and merging that data to a patient’s EHR requires infrastructure and expertise…
While some repositories make their data freely available, others are restrictive, like Genomics England in London, limiting access to select institutions. …the Global Alliance for Genomics and Health is developing frameworks for the responsible sharing of genomic and clinical data internationally to improve precision medicine and cancer research. Data regulations and research review processes vary across healthcare organizations, direct-to-consumer (DTC) companies and other entities collecting human health data, creating incongruence when data is shared for research purposes and may not be transparent to individuals providing their personal information.
Precision medicine treatments pose considerable promise for patients with rare diseases, like sickle cell disease or muscular dystrophy, due to an ability to make targeted changes to a person’s genome. …the International Rare Diseases Research Consortium (IRDiRC) and the Privacy-Preserving Record Linkage (PPRL) Task Force are creating standards and processes to improve international data sharing and enable global harmonization of data sharing to facilitate research from diverse clinical sites. Once precision medicine therapies make their way through regulatory approval, processes which can vary across countries, companies must find ways to bring their products into the market. The individualized nature of precision medicine products, such as cell and gene therapies, creates unique commercialization hurdles, particularly in the supply chain pipeline.
… cell and gene therapies are some of the most expensive treatments in the world, costing millions of dollars… The high cost of these life-saving treatments are forcing insurers, hospitals, manufacturers, and governments to experiment with payment models. The complexity and potential risk of precision medicine therapies means that a limited number of hospitals have the necessary equipment and trained personnel to carry out these procedures …Expanding the benefits of precision medicine beyond major health centers in and into lower-resourced areas means training a new workforce and investing in healthcare infrastructure.
Despite its potential, precision medicine has not broadly integrated into healthcare systems as quickly as many people had hoped. While innovators across genomics, data science, and AI aim to overcome technological hurdles limiting the impact and deployment of precision medicine, policy-makers and regulators must update systems in a way that promotes innovation while ethically and equitably protecting society. In order to strike this balance, policy-makers and regulators should look to the seven pillars of agile governance as a blueprint and a guiding structure. These agile governance processes can be applied to each stage of the precision medicine pipeline, recognizing that precision medicine encompasses a wide range of activities from basic research to care delivery.