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Lessons Learned From Failure Of The National Children’s Study Are
Guiding New Initiative To Recruit One Million Study Participants
In his 2015
State of the Union address, President Barack Obama announced
the Precision Medicine Initiative (PMI), an ambitious plan to advance
the emerging field of precision medicine through research and
development of technology and policies that improve individualized
care. Precision medicine is a personalized approach to disease
treatment and prevention that is tailored to individuals based on
specific genomic, environmental and lifestyle data.
Topic Areas of
Interest
The PMI will look at
genomic, environmental and lifestyle data to achieve a better
understanding of disease risk, mechanisms of disease and individual
response to therapeutics. As the White House describes it in an
official statement, the overarching goal of the PMI is to “leverage
advances in genomics, emerging methods for managing and analyzing
large data sets while protecting privacy, and health information
technology to accelerate biomedical discoveries.” More specifically,
NIH director Francis Collins has written in a perspective in
the New England Journal of Medicine that the initiative will have a
“near term focus on cancers and a longer-term aim to generate
knowledge applicable to the whole range of health and disease.”
Key Component
A central component of the PMI
involves
the development of a longitudinal research cohort of at least 1
million U.S. participants from diverse social and ethnic backgrounds,
all ages and all health conditions. However, just months before the
President announced his plans for the PMI, the government was forced
to shut down the National Children’s Study (NCS) before enrolling a
single child despite spending 14 years and $1.3 billion on planning
and pilot testing. As the NIH is now preparing to begin enrolling
volunteers for the PMI research cohort in early 2016, some remain
skeptical of the feasibility of large-scale population studies.
Problems and Solutions
The NCS intended to study environmental influences on
child health and development by tracking 100,000 U.S. children from
before birth until the age of 21. By collecting biological specimens,
environmental samples from homes and data from the families,
scientists hoped to gain insight into the impact of these factors on
the development of disorders such as asthma and autism. While there
is no simple explanation for why the NCS study failed, there were a
number of significant problems with the study that NIH officials are
hoping to avoid with the PMI.
1. The PMI must move
quickly
One of the major issues with the NCS was that it simply
moved far too slowly. The NIH spent 7 years and $54 million on
planning alone, as debates and internal arguments over study design as
well as how to recruit and enroll participants dragged on for years.
Pilot studies, which accounted for the majority of the $1.3 billion
spent, took more than 9 years to even begin. These delays crippled
the study, as NIH director Francis Collins told the website
STAT, “Science changed drastically during that time, and so the design
that had been put together didn’t really fit the scientific
opportunities by the time it was ready to get started.”
Enrollment Strategies
To address this issue the PMI will be utilizing
different enrollment strategies, both allowing individual Americans to
volunteer directly as well as collaborating with large health care
provider organizations such as the Geisinger Health System, Veteran’s
Affairs and Kaiser Permanente who serve diverse populations and
already have electronic health records and stored biosamples for
millions of people.
Aided by the advent of electronic health records, this
strategy should be significantly faster and more cost-efficient than
the door-to-door recruitment strategy employed during the NCS. The
NIH’s PMI working group estimates that the recruitment goal of 1
million participants could be reached in 4 years with this
methodology.
2. The PMI must have consistent and
empowered leadership
Throughout the course of the study, the NCS went
through multiple directors and was continuously undermined by
inconsistent and ineffective leadership. Researchers and
administrators went back and forth on fundamental aspects of the study
design and implementation, culminating in a letter written to the NIH
by 5 lead investigators involved in the study in which they argued
“the present direction and conduct of the study places it at high risk
of scientific failure”.
The PMI Working Group specifically addressed the need
for consistent and empowered leadership in their report to director
Collins, recommending that the NIH appoint a director with
“institutional authority, professional expertise and structural
support to lead the effort”.
3. PMI must avoid
funding issues
In
the case of the NCS, funding for the full study was delayed for years
and the initial funding had to be reauthorized by Congress every
year. Cost projections began to increase significantly beyond the
initial total budget and the NIH eventually dramatically cut funding.
The NIH hopes these funding issues can be avoided with the PMI due to
strong political support from both Republicans and Democrats. The PMI
already has a 2016 budget of $215 million, $130 million of which is
designated for the development of the research cohort. In addition,
by learning from the mistakes of the NCS, the NIH believes they can
utilize more cost-effective recruitment strategies to keep expenses
under control.
Links to related articles:
1.
https://tinyurl.com/j8bq45c
2.
https://tinyurl.com/ktoka69
3.
https://tinyurl.com/zxxao7q ■
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