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