FDA – Incorporating cost effectiveness & societal outcomes into FDA opioids model (U01) Clinical Trials Not Allowed

March 16, 2020 by dld5dt@virginia.edu


The purpose of this funding opportunity is to incorporate social outcomes (quality of life, untreated pain, etc.) and cost effectiveness into FDA’s existing model and policy analysis tool. Work resulting from this funding opportunity should provide more holistic insight into the opioid crisis and enhance decision makers’ ability to propose and consider policy options. Such work will require the integration of new structural components, new data, and new analyses into the model. Given the paucity of data surrounding opioids interventions, this effort will also require studies to collect information about the costs, social outcomes, and other details associated with potential policies.

It is anticipated that 1 award will be made, not to exceed $1,000,000 in total costs (direct plus indirect).


In 2018, FDA’s Center for Drug Evaluation and Research (CDER) began an effort to develop a comprehensive systems model of the opioid crisis. The purpose of the model is to help FDA better understand a) the complexity of the underlying mechanisms of the crisis, and b) potential short- and long-term impacts of possible policy actions — both desired outcomes and unintended consequences. FDA intends to leverage the model as a practical tool to inform FDA priority-setting and decision-making about potential policies. Given the model’s integrative perspective, this effort may eventually provide insights into policy-making beyond FDA. FDA’s opioids systems model project coincides with complementary research and modeling efforts undertaken by both the National Institute on Drug Abuse (NIDA) and the Centers for Disease Control and Prevention (CDC).

FDA’s current opioids systems model employs a system dynamics modeling approach[1], which is particularly well-suited to the opioid crisis for two reasons. First, the opioid crisis is complex, with multiple interacting feedbacks, time delays, and dynamics that evolve over time and lead to unforeseen or unintended outcomes. A systems model can provide the integrative perspective needed to guide regulatory decision-making under such circumstances. Second, there are large gaps and uncertainties in data relevant to the crisis. Simulation models allow rapid and extensive sensitivity analyses to help address these uncertainties.

The formal architecture of the current model has taken shape based on a review of the literature and numerous interviews with experts in government, academia, and professional practice. This architecture is then transformed into a simulation model that is currently being quantified, calibrated, and validated. The model will ultimately be used as a tool to support policy analysis. FDA’s opioids systems model focuses on trajectories of opioid use, including, but not limited to: initial exposure to prescription opioids; opioid use, abuse, and misuse; development of opioid use disorder (OUD); use of illicit opioids; opioid overdose, death, and harm reduction; and OUD treatment and relapse. The model identifies key influences on these processes, including situational, behavioral, and motivational factors. FDA’s opioids systems model will be fully transparent and documented, as well as publicly available, for further reproducibility and validation.

FDA’s opioids systems model is built upon academically rigorous standards and careful assessment of the best available evidence of the opioids system. At its initial stage, the systems model is considered a “baseline” dynamic model limited in current scope and quantification.  The model has been developed to enable continual improvements, expansion, and adaptation as the complexities of the crisis change. There is a continual need to enhance the model by refining the underlying model structure, refining the quantification of the model parameters, expanding the scope as appropriate, and transforming the model into a practical policy analysis and communication tool.

In September 2019, FDA awarded a grant under RFA-FD-19-026 to continue enhancing and improving FDA’s systems model focusing on the following seven areas: 1- Enhance modeling of utilization, effectiveness, and outcomes of treatment for opioid use disorder (such as Medication-Assisted Treatment (MAT) and behavioral therapy); 2- Enhance modeling of opioids prescribing and prescription opioids utilization; 3- Incorporate modeling of mental health and other comorbidities of opioid use disorder; 4- Research and enhance the modeling of potential interventions of most interest to FDA; 5- Research and enhance modeling of the supply utilization of illicit opioids such as heroin, synthetic opioids such as fentanyl, and polysubstance use; 6- Research and expand integration of social determinants into the systems model; and 7- Appropriately harness social media data to inform FDA’s systems model. FDA has been working closely with the awardee on these enhancements since September 2019.  RFA-FD-19-026 is anticipated to conclude in September 2020 with a second version of the model that includes the enhancements described above.

FDA has also awarded a separate contract, which also began in September 2019, that includes a third-party review and validation of FDA’s model. The contractor will also develop a policy tool based on the underlying systems model developed by the FDA and enhanced by RFA-FD-19-026. The current efforts are expected to result in a systems model and policy analysis tool to explore the intended and unintended consequences of potential opioids policy options. Under the scope of the current effort, FDA does not anticipate that the model and the policy tool will yet be equipped to explore social outcomes (quality of life, untreated pain, etc.) or the cost effectiveness of policy options. These analyses are important steps toward understanding the true effects of the opioid crisis on the U.S. population.


Posted Date – March 13, 2020
Open Date (Earliest Submission Date) – March 16, 2020
Letter of Intent Due Date(s) – April 6, 2020
Application Due Date(s) – May 18, 2020

Filed Under: Funding Opportunities