Drug Development and Approvals
Clinical trials aren't the only step to bringing new medications to market. Drug developers must work closely with federal regulators throughout the testing process and then receive approval of their drug or medical device before they can market it to the public. The price of drugs and coverage by insurance companies and Medicare/Medicaid also can impact if patients are able to access the drugs they need. The Michael J. Fox Foundation (MJFF) works with regulators and payers to design efficient studies and to convey the patient need for and impact of new therapies.
Food and Drug Administration
The Food and Drug Administration (FDA) is the federal agency responsible for protecting the public health through the regulation and supervision of a variety of areas, such as our nation's food supply, tobacco products, vaccines and medications. See more on the FDA website on the process of drug development and approval.
In addition to continued funding for the FDA, MJFF advocates for conversation and collaboration with regulators to support treatment of Parkinson's disease (PD). In 2013, the Foundation held a meeting with the FDA to discuss clinical scales used in trials of drugs for cognitive impairment, and MJFF staff testified at a 2016 FDA Advisory Committee Meeting evaluating a new drug for Parkinson's psychosis.
The Foundation also helped prepare for the September 2015 FDA Patient-Focused Drug Development meeting on Parkinson's disease where patient panels discussed the experience of Parkinson's disease and unmet needs. Read the FDA's report on the meeting, or watch the full meeting on the FDA website. (The Parkinson's meeting is listed as "Meeting Recording Part 3" and "Meeting Recording Part 4.")
Drug Developers and Payers
Throughout every stage of the drug development process, patients need a representative to highlight their voice and make their needs known. This allows drug developers to better understand which symptoms or area of Parkinson's can be targeted to best improve quality of life. Regulators and payers can make the most informed decisions when they hear the patient voice and take into account the needs of the community.
As a trusted and credible ally to both researchers and patients, MJFF is uniquely positioned to play this role, ensuring developers, regulators and payers understand important areas of unmet need.
As one example, MJFF advocated for patient needs around the treatment of "off" episodes. Facing a lack of knowledge among decision makers and a handful of therapies in late-stage testing for avoiding or treating "off" episodes (resurgence of symptoms when on medication), MJFF gathered patient voices to elevate the need for and potential impact of a drug to address this aspect of PD. In the spring and summer of 2014, MJFF surveyed 3,000 patients on the prevalence and impact of "off" episodes, and in August 2014, MJFF convened a meeting of more than 20 payers and policymakers with members of the MJFF Patient Council to share survey results and educate on "off" episodes.