Response to "Repairing the Parkinson's Pipeline"
by Robin Elliott in PDF's
Summer 2008 issue of News & Review
The article "Repairing the Parkinson's Pipeline" by Robin Elliott in PDF's
Summer 2008 issue of News & Review provides a very good summary of the
successes of the past decade as well as the tough challenges that the PD
community faces in order to continue progress toward better treatments and cures
for people with Parkinson's (PWP.) The statement offers a historical context for the recent
disappointments, and it offers general concepts to begin to answer the growing
bewilderment and frustration of activist PWP whose first-hand experience as
research participants in clinical trials has too often been at odds with the
scientific, regulatory, and business decisions of the other main constituents in
the process of therapy development. This comment intends to illustrate how a
patient advocacy organization can help break through these scientific and
regulatory roadblocks through true collaboration and mutual respect for the
power of independent patient perspectives.
The Parkinson Pipeline Project was initiated in 2002 to address the challenges
of therapeutics development. We start from a fundamental and essential concept
that "the missing ingredient in development of new therapies is the voice of the
patient," as Clifton Leaf has concluded in "Deadly Caution," Fortune Magazine's
February, 2006 review of medical science innovation and FDA regulation. In
addition to participation in clinical trials as research subjects, PWP advocates
must be included as full collaborators at every stage of new therapy
development. PWP involvement is necessary to help specify outcomes, to help
design research protocols, and to help bridge the wide gaps in communications
and understanding among patients, regulators and scientists.
The Pipeline Project was established with support from PDF to provide this
ingredient and in collaboration with PDF has established new advocacy roles to
facilitate the process. Roles to assist sponsors and regulators on study design
and analysis and roles to assist clinical researchers on the conduct of studies
to evaluate therapies have been identified. PDF has also collaborated with
Pipeliners on (1) the development of a Declaration of Clinical Research Rights
and Responsibilities to establish criteria for PWP participation in clinical
studies, (2) formal training of PWP for advocacy roles and (3) a series of Round
Table discussions to apply the most informed expertise among scientists,
industry, and PWP to solve the key problems that interfere with timely
evaluation of new therapies.
The recent FDA Amendments Act (FDAAA) goes a long way to improve the
process of regulation which determines the pace at which studies can proceed.
Taking into account the limitations of methods for randomized controlled
clinical trials that are necessary for approval of new therapies, this new law
emphasizes the need for closer monitoring of interactions among medicines
throughout the product's life cycle, and reinforces the value of transparency of
data and results for the advancement of science and new therapies. However, the
law did not change the proprietary interactions between industry and FDA in the
pre-approval phases of the pipeline. Opening up this process to the light of
public and patient interest is an important precursor to a more efficient and
effective regulatory processes.
Based on the successful advocacy of the Pipeline Project, PD is the first
disease to expand the FDA's pilot efforts in oncology that seek input from
patient consultants on pre-approval study design and analysis issues. PD patient
consultants have been selected by FDA and trained in collaboration with the
Pipeline Project to provide the patients' view in these critical decisions.
Also, PD along with AD have been identified as pilot examples of a new effort
by the Critical Path Institute and the Regan-Udall Foundation (set up by the new
law) to test analytic methods and build the information infra-structure
necessary to support patient centered medical research and health care. This is
a wonderful opportunity to establish PD as a leader among chronic diseases in
the collection and use of population data and clinical findings, which for PD
has been a large gap in the knowledge necessary to assess risk and deliver
cures.
PDF has taken a lead among PD organizations in recognition of the power and
importance of the insight and motivation of activist PWP. We still have a long
distance to go for the independent voice of patients to be consistently and
loudly heard in all decisions about medical research and patient focused health
care, but movement in the right direction gives us hope.
Perry D Cohen, PhD
Founder and Project Director
Parkinson Pipeline Project
|
