Open Outcomes Based Regulation
The U.S. Needs a New Approach to the Validation and Use of Medical Products and Health Care Services
It is almost a truism that the U.S. health care systems is “broken” yet the solutions offered tend to be either supportive of the pre-PPACA status quo or offer solutions based making the existing system better. Neither approach will be effective unless the fundamentals are changed. What are the fundamentals? In relation to medical products and health care services, society needs informed purchasers and accountable sellers. (We also need accountable consumers but that will be the subject of a future conversation.)
Government has an important role in this effort. Its responsibilities include:
- Protecting the public health by reasonably assuring their safety, efficacy, and security;
- Promoting public health by promoting useful innovation and ensuring that there is timely access to new technologies and knowledge; and
- Helping the public get the accurate, science-based information they need to manage their own health.
In carrying out these responsibilities, agencies face the significant challenge that the pace of change in technology, medical knowledge, and health behavior is increasing while regulatory processes are slow and isolated from post-approval real world results. The concept of “risk” is central to existing regulatory program but it is complicated. There are two types of risk: (1) the risk to a patient from the use of a product (including services and integrated solutions); and (2) the public health risk from lack of access to a product).
At the same time, risks change over time due to the dynamics of people and market:
- Technology improves:
- Knowledge improves;
- Average user competence generally improves;
- Support systems may improve or decline; and,
- External factors change for better and worse, both rapidly and slowly (e.g. environmental conditions, diet, epidemics).
For purposes of regulation of a specific product, the two types of risk should both be considered and risk should be regularly evaluated over time in light of the five dynamic factors. In light of these factors, long delay or failure to act may be equally or more harmful than approval. Moreover, the true risks and efficacy of products are best discovered in real world use.
Current health care and medical regulatory processes are not efficient or effective in maximizing the twin goals of protecting patients and promoting health. However, the tools of connected health offer the resources to create a dynamic system in which safety and efficacy can be easily monitored and are more transparent in near real time, thus leveraging the power of markets to support regulatory goals.
Outline of Open Outcomes Based Regulation
Open Outcomes Based Regulation (“OOBR”) leverages the power of connected health technologies and has the benefit of establishing a framework that can be applied to improve the regulation of drugs as well as health IT and medical devices. It is sufficiently flexible to deal with the fact that the boundaries between hardware, software, and drugs are changing rapidly. The future of consumer health and health care services belongs to combination products that are utilized within the unregulated consumer sector and regulated health care systems. The formerly bright line between unregulated consumer health and regulated health care is being erased by the activities of both consumers and traditional health care players (providers, payors).
The principles of OOBR are based on a system for continuously improving risk and efficacy estimates that incorporates the investigational and computational power of the research community in addition to agency resources. Essentially, the system would include these steps:
- A product is approved by an agency or is self-certified based on defined categories, sometimes including pre-market clinical research;
- Initial market authorization may be limited, based on estimates of the likelihood and degree of unanticipated risks;
- Once the product is in the market, data about its safety and efficacy are collected in a de-identified database that is available to approved researchers under defined rules for access and publication;
- As appropriate, the marketing approval and labeling of the product are revised in accordance with the data.
- In addition, customers and users will have access to analysis that equips them to make informed purchasing decisions.
We envision this approach to start as a voluntary program and alternative to traditional regulation of Class I, II and III products. It would be applicable to products that by their nature or based on evidence do not pose a material risk of significant unavoidable harm to users taking into account the restrictions on use, the anticipated learning curve based on the analysis of data gathered through use of the product, and the harm (if any) associated with its not being available. In order to effectuate this continuous learning process, manufacturers would agree to the continuous/regular sharing of data on product utilization, efficacy and outcomes. This responsibility may be shared with clinical organizations that utilize or prescribe the product. Liability issues require specific study, but in general we support an approach that takes into account the willingness of a clinical provider to accept legal responsibility for the safety and efficacy of a product.
WLSA recognizes that the implementation of Open Outcomes Based Regulation would require a substantial effort, including participation by government agencies, research funders, manufacturers, and health care organizations. By leveraging the power of big data, the research community and the market, OOBR offers a pathway to move promising products to market more quickly, modify the permitted uses of these products more quickly and enable the health care community to more quickly identify the most efficacious use of approved products.
OOBR may be considered as a modernization of the established regulatory concept of post-approval studies with the objective of using current technology and knowledge platforms to expand its use and make the process as open, continuous, and timely as possible. This can be a voluntary system that will be improved over time due to ongoing improvements in technology, regulatory principles, and consumer/industry acceptance. The goal is to establish a dynamic regulatory system that speeds the entry of promising medical products and services to market while it frequently reevaluates the risks associated with the devices and adjusts the approved uses accordingly. The open nature (”transparency”) of the system will improve levels of self-enforcement by manufacturers and rely as much as possible on the marketplace to determine the value and efficacy of devices.
There is an example of another major sector in the U.S. economy that operates on a similar basis. Automobiles are primarily brought to market through a self-certification process. Adverse events such as accidents and mechanical failures are analyzed by private parties, regulatory agencies, and researchers. Products are continuously improved and marketed for different intended uses. Mis-use is sanctioned. As a result, automobiles have continuously become safer, more efficient, more comfortable, and cheaper over the last few decades, and drivers have improved. The U.S. auto industry has become competitive in the world market and users have modified their behavior (seat belts, impaired driving) to improve safety. I believe that, over time, U.S. regulation of all aspects of health care move toward this model which is based on standard setting, self-certification, adverse event analysis, and shared liability.
The U.S. is a global leader in the life sciences and it can retain its role as the global leader in mobile and wireless health with the support of the agencies. Wireless health has significant policy benefits for the U.S. and the entire world:
- Improve access to services.
- Lower the cost of healthcare.
- Improve the quality of healthcare.
- Make healthcare services transparent and thus measurable and accountable.
- Improve public health.
