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We support and applaud the goal of the National Plan -- to
prevent and effectively treat Alzheimer's Disease by 2025.
- We recommend that interim milestones be set, through development
of a clear roadmap of research and treatment discovery priorities and
timelines, to assure continuing and successful progress toward achievement of
this goal. For example, we recommend that an interim milestone be set to make
available to the public by 2020 significant disease-modifying or
substantially-enhanced symptom-mitigating behavioral or pharmacologic
interventions.
- While these goals of making new remedies for AD available by 2020
and 2025 are ambitious, they should not be interpreted as favoring
translational drug development over basic discovery. New investment in basic
research and drug discovery must reflect a critical balance between long-term
investment and the urgency of immediate progress to our nation's public and
fiscal health.
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There is an urgent need for annual federal research funding to be
increased to the level needed to fund a strategic research plan and to achieve
the breakthroughs required to meet the 2025 goal. Initial estimates of that
level are $2 billion per year but may be more. That investment would be applied
to Alzheimer's research initiatives spanning basic, translational and clinical
research.
- The Administration, working with the research and business
communities, should develop an overall budget needed to achieve the 2025 goal,
and should propose to Congress and support a rapid ramp up to a minimum $2
billion in Alzheimer's research at NIH. The optimum levels of annual funding
needed to achieve the 2025 goal should be determined in connection with the
preparation of the President's budget, and should be reviewed and adjusted each
year based on progress and new developments.
- As part of the strategic research plan mentioned in the National
Alzheimer's Plan, we recommend that NIH develop a system of accountability to
monitor progress toward the 2025 goal.
- We recommend that NIH coordinate with other federal agencies to
ensure that overall federal Alzheimer's funding complements the NIH's
investments and enhances progress towards the goal of preventing and
effectively treating Alzheimer's by 2025. We also recommend that the strategic
research plan identify and monitor not only existing resources within the
Federal government, but also new resources outside the Federal government,
including new private-public partnerships, incentives for increased private
investment, State-based research funding, and mobilization of global
investments.
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We recommend that HHS develop, execute and regularly update a
strategic research plan and priorities to accelerate breakthroughs in AD
research.
- The process of developing that scientific research plan and
accompanying priorities should be viewed as shared project of NIH, FDA, and
other relevant government agencies; the academic and corporate research
community; industry; and NGO's.
- Given the global scope of the Alzheimer's challenge and the
international character of the research enterprise, we recommend that the
strategic research plan be coordinated with the research efforts of other
nations and that stakeholders from other countries with Alzheimer's plans in
place or in process be included in the planning process.
- The structure of the scientific research plan should be framed
with the National Alzheimer's Plan updating process in mind so that issues can
be addressed not only annually, but also in synch with the plan updates and so
that progress can be tracked using potential convening partners for different
action or convening 'streams'.
- The Director of NIH should monitor the Alzheimer's research
portfolio across all Institutes and Centers of the NIH.
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We recommend that the Administration designate specific Offices
and officials within the White House and the Office of the Secretary of Health
and Human Services with responsibility and accountability for effective
implementation of, and timely, transparent reporting on, all aspects of the
implementation of this National Alzheimer's Plan, including responsibility for
issuing statutorily required reports to Congress on behalf of the Secretary,
reports to the Advisory Council, and other reports as warranted.
- The designated Office within the White House should be
responsible for adequate monitoring across agencies and the designated Office
within the Office of the Secretary of HHS should be responsible for monitoring
within the departments of HHS.
- We believe it important to develop a system of accountability for
the achievement of the 2025 goal based on quantifiable metrics and milestones
with respect to the action steps and strategies in the national plan.
- We recommend that the Secretary, as part of her annual report to
Congress and the Advisory Council, report on progress over the prior year in
meeting the annual objectives, strategies and actions enumerated in the
National Alzheimer's Plan, as well as providing a comprehensive, multi-year
perspective, and mid-course corrective action steps, that are needed in order
to meet the 2025 goal of this Plan.
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We recommend that HHS, in partnership with the research community
and industry, take steps to accelerate public access to new therapeutic
interventions by compressing the current average time in the process of
identification of therapeutic targets, validation of those targets, development
of behavioral and pharmacologic interventions, testing of efficacy and safety,
and regulatory review, including the following:
- Convening expert advisory panels/conferences to identify genetic,
family history, medical co-morbidities, biomarkers, and clinical features in
asymptomatic persons that are risk or protective factors for AD
neuro-pathological physiology and ultimately AD clinical symptoms.
- Cataloguing existing Alzheimer's biological and behavioral marker
initiatives including their current development and review, and identify gaps
and a plan for addressing them.
- Issuing, upon endpoint approval, of unambiguous guidance on the
use of behavioral and biological markers to industry on their usage.
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We recommend that the Secretary, in consultation with academic
researchers, not-for-profit Alzheimer's organizations, and the private sector,
including sponsors of investigational diagnostic and therapy trials, by
year-end 2012 identify and prioritize the action steps needed to reduce the
time for moving therapies from target identification and validation through
clinical development, regulatory review, market approval, and reimbursement
determinations.
- The Secretary, in conjunction with NIH and FDA, should increase
targeted public-private partnerships that bolster innovation and regulatory
science progress.
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We recommend, as part of the initiative to accelerate public
access to new therapeutic interventions, that the Secretary examine and include
as part of her annual report to Congress and the Advisory Council:
- How the HHS uses existing authorities to reduce drug development
barriers and accelerate development of new therapies;
- Immediate steps the HHS will take to address any identified drug
development barriers, including regulatory hurdles; patent, intellectual
property, regulatory science, or clinical trial infrastructure weaknesses; and
plans to advance regulatory science, guidance, and other initiatives under
existing authorities;
- Additional authorities or other legislative action that may be
needed to accelerate development of therapies and diagnostics; and
- Immediate steps to shorten time from market approval to coverage
decision for innovative therapies and diagnostics.
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We recommend that the FDA review and periodically report to the
Advisory Council:
- Recommendations to further accelerate FDA review processes
without compromising current standards of safety and efficacy.
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We recommend that the HHS Secretary develop a continuing process
by which research priorities aimed at accelerating the delivery of effective
treatments would be set, including input from scientific experts.
- In our view, a joint NIH/NIA and Industry Working Group should be
established, which can serve as an opportunity to create a true partnership
between government and industry to inform research priorities.
- In order to accelerate the process of discovery, we recommend
that this Working Group identify strategies for increasing the increased
standardization, disclosure, pooling and analysis of pre-clinical, clinical and
electronic health data.
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To address disparities, we recommend that clinical research
studies and activities aimed at translation of research findings into medical
practice and to the public include specific targets for outreach to specific
populations by racial/ethnic group, sex, and socioeconomic status, as well as
to populations at high risk for AD (e.g., people with Down Syndrome).
- Specific recommendations for recruitment and outreach goals for
diverse populations should, in our view, be integrated into planned AD research
meetings/summits.
- Resources and "formulas for success" of NIH-funded RCMARs, ADRCs,
and R01 awards that have successfully recruited large numbers of ethnic
minorities and socioeconomically diverse people for clinical aging research
can, in our judgment, be leveraged to inform any future recruitment efforts
taken via NAPA initiatives.
- In our view, private and public entities can collaborate to
increase diversity within clinical trial participation through open-architected
prevention registries such as the Alzheimer's Prevention Initiative,
Alzheimer's Association TrialMatch, and NIA-funded RCMARs, producing increased
identification of ethnically and socioeconomically diverse people for
participation in clinical studies of AD.
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We recommend that HHS develop accurate and relevant metrics for
assessing the impact of Alzheimer's on the U.S. economy.
- We believe it important to develop a system of accountability for
the achievement of the 2025 goal including estimates of the impact of
prevention and effective treatment of Alzheimer's Disease on the US economy,
families and costs to Federal health care programs.
- Identify and rectify the shortcomings of the data needed to
assess the prevalence, costs (financial, fiscal and economic), and deaths
relevant to Alzheimer's disease.
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We recommend that HHS commit to an effort to maximize private
investment in the development of treatments and improvements in disease
monitoring technology by identifying policies that would encourage private
industry to invest aggressively in disease-modifying interventions, to support
technologies that improve our ability to detect the disease as early as
possible, and monitor the disease accurately so that the effectiveness of
interventions can be tested.
- As part of the larger NAPA agenda, we recommend that a process or
mechanism for securing sustained industry input on topics such as measures to
spur discovery and streamline regulatory review, tax, and Intellectual Property
be established, with a particular emphasis on diminishing the barriers to
sharing both basic scientific and clinical data), and other incentives.
- We believe a strategic use of SBIR, STTR and other co-investment
initiatives can be used to promote advanced research and support from small
businesses engaged in this work.
- Through a joint public-private process, we believe that we can
advance other related actions included under other recommendations (e.g., the
industry engagement with NIH, research prioritization, behavioral and biomarker
and endpoint validation, etc.) that are already known to be of importance to
industry.
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We recommend that the Administration expand and enhance
meaningful coordination with global partners and move forward to establish a
Global Alzheimer's Action Plan to respond to the global scope of the
problem.
- Moving that objective forward might start with convening a
meeting of all nations or regions with National/Regional Alzheimer's Plans in
place or under development by 2013 in order to compare approaches and identify
mechanisms to foster global coordination and progressively address the global
problem.
- The responsibility for such an initiative would require the
identification of a single high-level U.S. official as the point person for the
National Alzheimer's Plan and appointment of that person to represent the
nation as part of an ongoing dialogue with global counterparts.
- We believe that such a Global Alzheimer's Action Plan can be
built upon existing global collaborative research initiatives and lead to
greater global collaboration and coordination of research funding on a global
level.
- Any Global Alzheimer's Action Plan should foster ongoing
international dialogue and potential coordination on Alzheimer's regulatory
review and related issues.
