Public Policy Matters in Clinical Research
The work of drug development is more than clinical trials and volunteers. It’ s important to stay informed about public policy— government laws and regulations— because it can directly affect how treatments move from lab bench to pharmacy shelves, and how they are evaluated and approved.
Upholding Rigorous Approval Standards
Clinical trials test potential therapies for two important factors: Are they safe for patients to use and do they actually work? Researchers spend significant amounts of time and money investigating these questions. At the end of the clinical trial process, the U. S. Food and Drug Administration, the European Medicines Agency and governmental organizations in other countries review medications to ensure they meet strict safety and efficacy standards. While these regulatory agencies make the final approval decision, public policy can influence their evaluation criteria.
Encouraging Drug Developers to Meet Patients’ Needs
People living with Parkinson’ s disease( PD) are well aware of the gap in treatment options for many symptoms, such as cognitive impairment, balance problems and constipation. In the United States, policymakers created pathways to get crucial drugs to market quickly while maintaining strong FDA safety and efficacy measures.
The“ breakthrough therapy” designation, for example, allows the FDA to expedite the development and review of a therapy that may offer substantial improvement over existing treatments. Nuplazid( pimavanserin), the first drug approved to treat hallucinations and delusions associated with PD, got to market faster through this program. Up to 50 percent of people with PD can experience these symptoms at some point( they’ re more common in those with longer duration of disease, dementia or older age), and no approved treatments were previously available. Based on preliminary evidence, the FDA shortened Nuplazid’ s clinical trial development and review timeline while still preserving strict safety and efficacy guidelines.
Recognizing Changing Research Trends and Tools
The Parkinson’ s drug development pipeline encompasses a broad array of experimental therapies, including motor and non-motor symptomatic options, and varied disease-modifying therapies( ones that could slow or stop PD progression). Therapies in testing include everything from antibody infusions that clear out alpha-synuclein( the sticky protein that clumps in the brains of people with PD) to surgical procedures to drugs for people with specific genetic mutations. Research is evolving to address patient priorities and incorporate new devices and technologies. Regulators at the FDA must be in tune with patients’ needs and the latest ways to meet them, including unique trial designs and the right measurements to determine if they are successful.
When it comes to priorities, treatments to lessen or prevent dyskinesia( involuntary movements that can occur as a complication of long-term PD drug use) are toward the top of the list. But to make sure these therapies work, we need a way to measure their impact in trials. When a standardized approach to rate dyskinesia did not exist, The Michael J. Fox Foundation funded the development and validation of the Unified Dyskinesia Rating Scale. This tool is now widely accepted by clinicians, researchers and regulators as a method for evaluating potential benefit of new dyskinesia therapies.
Genetic research is another area where we’ re all working together. Parkinson’ s genetic clinical trials are just beginning, and they’ re an avenue toward precision medicine. As this research progresses, the FDA and researchers have to determine the right numbers and types of participants( with and without genetic mutations) to include in which trials so they can gather the best data.
Policy and research also intersect with technology. Data collected through online studies and smartphones can help scientists form a more complete picture of what life is like with Parkinson’ s. Wearable devices can capture how movement changes in real time when medication is taken. The FDA must determine how trials can use new and existing technologies and what types of data they can gather to achieve the most robust results.
To learn more about policy and get involved in policy activities that can affect Parkinson’ s research, visit advocate. michaeljfox. org.
10 Navigating Clinical Trials: A Guide for Parkinson’ s Patients and Families