In 2013, the U.S. Food and Drug administration (FDA) selected Huntington’s disease (HD) and Parkinson’s disease (PD) as the focus of a three-year public consultation project for Patient-Focused Drug Development. This initiative involves working toward a better understanding of patient perspectives on the severity of these diseases and evaluation of therapies available for their treatment. Through the end of 2015, the FDA wants to hear about symptoms that most impact people with HD or PD and their families, and what stakeholders are hoping for in new treatments for these disorders.
The Patient-Focused Drug Development initiative is being conducted to fulfill FDA performance commitments that are part of reauthorization of the Prescription Drug User Fee Act (PDUFA), first enacted in 1992 and renewed in 1997 (PDUFA II), 2002 (PDUFA III), 2007 (PDUFA IV), and 2012 (PDUFA V). The PDUFA authorizes the FDA to collect fees from companies that produce certain human drug and biological products to provide it with the necessary resources to maintain a predictable and efficient review process for human drug and biologic products under Title I of the Food and Drug Safety and Innovation Act (Pub. L. 112-144).
The full set of performance commitments is available at http://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm270412.pdf
As directed by Congress in the FDA Amendments Act of 2007, the FDA developed the proposed enhancements for PDUFA V in consultation with drug industry representatives, patient and consumer advocates, healthcare professionals, and other public stakeholders from July 2010 through May 2011. The FDA transmitted these proposed enhancements to Congress on Jan. 13, 2012, which are referred to in Title I of FDASIA. The law ensured that FDA would continue to receive consistent funding in fiscal years 2013-17 to allow it to fulfill its mission to protect and promote public health by helping to bring to market critical new medicines for patients.
The FDA has committed to obtaining patient perspectives on 20 disease areas during the course of PDUFA V, with the Agency conducting public meetings to discuss the diseases and their impact on patients’ daily lives, which types of treatment provide the most benefit to patients, and to hear patients’ perspectives on the adequacy or otherwise of available therapies. The meetings include the participation of FDA review divisions, relevant patient communities, and other interested stakeholders.
On April 11, 2013, the FDA published a notice (78 FR 21613) in the Federal Register announcing the disease areas for meetings in fiscal years (FY) 2013-15, the first years of the 5-year PDUFA V time frame. The Agency used several criteria outlined in that notice to develop the list of disease areas, and obtained public comment through a public docket and a public meeting on Oct. 25, 2012. The FDA is to initiate a second public process for determining disease areas of focus for FYs 2016-17 by the end of FY 2015.
More information, including the list of disease areas and a general schedule of meetings, is posted at: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm326192.htm
One of the series of public meetings was held Sept. 22, 2015, at the FDA’s White Oak Campus in Silver Spring, Maryland, providing an opportunity for public comment on Patient-Focused Drug Development for Huntington’s disease and Parkinson’s disease, and allowing the agency to obtain patient perspectives on the impact of these diseases on daily life, and their views on treatment approaches.
The FDA, recognizing that while both PD and HD are neurological diseases that have quite distinctive characteristics, structured the meeting into two distinct sessions — with the morning session, from 9 a.m. to 1 p.m., devoted to hearing patient perspectives on Huntington’s disease, and the afternoon session, from 1 p.m. to 5 p.m., focused on the impact of Parkinson’s disease and its treatments on patients.
Huntington’s disease is a fatal genetic disorder that causes progressive degeneration of nerve cells in the brain, resulting in uncontrolled movements, loss of intellectual faculties, and emotional disturbance. Each child of an HD parent has a 50-50 chance of inheriting the HD gene, and a person who inherits the HD gene will eventually develop the disease. Physicians may prescribe various medications to help control emotional and movement problems associated with HD, but while these medicines may help keep clinical symptoms under control, there is currently no known treatment to stop or reverse the disease’s course.
Parkinson’s disease belongs to a group of conditions classified as motor system disorders, which result from a loss of dopamine-producing brain cells. As nerve cells become impaired or die, affected individuals experience progressively worsening symptoms, including tremor, muscle rigidity or stiffness, slowing of movement, and impaired balance and coordination. The cause of PD is unknown, but it is thought that factors such as genetics and environmental triggers play a role. Although there is no cure for PD, medications such as the dopamine substitute levodopa can help manage levels of dopamine and other neurotransmitters in the brain to improve symptoms. A surgical procedure called deep brain stimulation may also be employed to manage symptoms if medications prove ineffective.
According to a Parkinson’s Action Network report, nearly 100 people attended the Sept. 22 meeting in person, with more than 200 participating online from around the country.
The meeting included a question-and-answer session with patients and patient stakeholders, followed by a facilitated discussion inviting comments from other participants. You can access a recording of the full meeting on the FDA website at: https://collaboration.fda.gov/hpm922
In addition to input generated through public meetings, the FDA is interested in receiving patient input addressing these questions through written comments, which can be submitted to the public docket. The FDA will be using information gathered at the Sept. 22 meeting, along with comments received by Nov. 23, to produce a summary report to be made public and to inform the agency’s approval processes for new treatments for Parkinson’s and Huntington’s.
Questions available for electronic or written comment can be found either at http://www.regulations.gov or by using this link, or by writing to the Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. All comments should be identified with docket number FDA-2012-N-0967.
Comments can be found at:
U.S. Food and Drug Administration (FDA)
Parkinson’s Action Network