Draft Policy Recommendations Self-directed Interactive Review
Category 1: Sponsors/Funders of Clinical Trials
Section 1: Regulation
Background:
The National Institutes of Health (NIH) Revitalization Act of 1993
requires applicants for federal research funding to provide a strategy for inclusion of people of
diverse racial and ethnic origin and women into clinical trials. While this requirement does force
investigators to think about issues of disparities in clinical trials and formulate a plan to alleviate
some of these disparities, recent data show that most clinical trials are still falling short of their
target inclusion percentages and that submitted plans are not always being followed after their
initial approval by the Institutes.
Policy Recommendation 1: NIH Revitalization Act of 1993
Because the NIH Revitalization Act is crucial to eliminating disparities in clinical trials, the
EDICT Team recommends that:
(a) - the NIH Revitalization Act provide for more direct instruction as to
what an appropriate inclusion plan should look like in regards to recruitment and
retention, and how success of the plan will be evaluated.
Rationale for this recommendation:
While the NIH Revitalization Act of 1993 mandates appropriate representation of
traditionally underrepresented populations in clinical trials, that mandate has, unfortunately, not
translated into measurable improvements. There are many reasons for this difficulty in
translation, but the ultimate point is that the Revitalization Act needs to be revitalized. The
Revitalization Act could be substantially strengthened with better and more specific guidance
from NIH that would provide detailed analysis and suggestions on how to implement appropriate
inclusion plans.
Moreover, NIH reviewers should more stringently assess submitted plans for their
likelihood of achieving the target inclusion and for the appropriateness of the target percentages
given the location. The EDICT Team noted the possibility of satisfying the mandates of the
Revitalization Act by attaching direct grant scoring to the merits of the inclusion plan.
Incentives should be offered to those proposals which contain superior inclusion plans.
Policy Maker Focus: U.S. Congress
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Policy Recommendation 2: Amend NIH Revitalization Act of 1993
Because the NIH Revitalization Act is crucial to eliminating disparities in clinical trials, the
EDICT Team recommends that:
(b) - The Act should also provide for substantial incentives for investigators to implement
appropriate inclusion plans.
Rationale for this recommendation: same as for previous recommendation
Policy Maker Focus: U.S. Congress
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Policy Recommendation 3: Reinvigoration of the FDA Modernization Act
Because FDA action is integral to eliminating disparities in clinical trials, the EDICT Team
recommends that:
(a) - FDA policy be harmonized with NIH policy to require appropriate racial and
ethnic inclusion in clinical trials
(b) - FDA implement penalties for non-compliance.
(c) - FDA implement incentives for appropriate racial and ethnic inclusion.
Background:
In 2005, the US Food and Drug Administration enacted the guidance, FDA
Modernization Act, to standardize the
collection of race and ethnicity data in public and privately funded clinical trials. However, the
FDA guidance "does not address the level of participation of racial and ethnic groups in clinical
trials." It has been suggested that the lack of requirements for diverse racial and ethnic inclusion
is not consistent with the essential ethical principles of the Belmont Report (i.e., respect for
persons, beneficence, and justice) . Moreover,
due to the fact that the NIH Revitalization Act mandates that NIH address issues
of appropriate inclusion and representation, the FDA has tended to describe the issues
surrounding disparities in clinical trials as largely outside of their jurisdiction or scope.
Rationale behind this recommendation:
A harmonization of FDA policy with NIH policy pursuant to the NIH Revitalization Act
of 1993 could have a significant effect in eliminating disparities in clinical trials. It is possible
that an unintended consequence of the Revitalization Act is the perception that ensuring
appropriate inclusion is solely NIH's responsibility. This is untrue because the FDA's primary
objective is to ensure the safety of the nation's food and drug supply, and there is a growing base
of quality evidence showing that introducing drugs into populations whose demographics and
comorbidities were not appropriately represented in the clinical trials is at least inadvisable, and
at most dangerous. As such, harmonizing FDA and NIH policy on disparities in clinical trials is
crucial to crafting a collaborative, informed regulatory approach on the federal level. In
addition, the NIH Revitalization Act applies only to recipients of federal funds. Given that many
clinical trials are funded by private sponsors, FDA authority is needed to augment buy-in among
stakeholders not subject to the mandates of the Revitalization Act.
While calls for increased penalties should be allocated judiciously, the EDICT Team
reached consensus that incentives alone are unlikely to generate buy-in from the relevant
stakeholders sufficient to substantially reduce disparities in clinical trials. Accordingly, penalties
may be needed to change the policy climate regarding federally mandated inclusion in clinical
trials. Such penalties, along with positive incentives for compliance, could conceivably go a
long way to reducing the complexity and the ambiguity of two different standards of reducing
disparities in clinical trials.
Policy Maker Focus: U.S. Congress and the FDA
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Section 2: Allocation Strategy
Background:
Diseases that are high incidence, high mortality, and feature high disparities ought to take
the highest priority for public sector funding of clinical research (see chart). Nevertheless,
there is evidence that diseases (e.g., liver cancer) which feature high disparities and high case
fatality rates are significantly underfunded. (see article)
Part of the problem is that the public and private sectors are essentially investigating the
same disease areas. It appears that economic considerations dictate policy in the private sector
and political considerations dictate policy in the public sector. Moreover, the influence of such
considerations in their respective sectors are unchecked by any defined, accepted standards for
allocations.
This approach to resource allocation leaves many populations underrepresented in
clinical trials. This is all the more problematic because many underrepresented populations bear
a disproportionate burden of disease.
Policy Recommendation 1: Federal Research Funding
All federal agencies that fund clinical research give the highest priority for their clinical research
investments to diseases with large disparities and high case fatality rates in racial and ethnic
minorities, socially and economically disadvantaged populations, and others bearing the greatest
burdens of disease.
Rationale behind this recommendation:
Private firms have little incentive to allocate significant funds to research on low
incidence/high case fatality/high disparity diseases. This is because such a disease simply does
not sicken enough people to enable a pharmaceutical, medical device, or biotechnology company
to earn a return on its capital investment.
Absent public sector funding, populations suffering from such diseases will not benefit
from clinical trials. Accordingly, allocating public monies to the conduct of clinical research is
crucial to ensuring that those who suffer from such diseases receive the benefits from clinical
research.
Policy Maker Focus: Federal agencies that sponsor clinical research and clinical trials and U.S.
Congress that allocates funds for those agencies.
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Policy Recommendation 2: Institute of Medicine Study
As a first step in assessing the extent of duplication of research resources, the U.S. Congress
should request that the Institute of Medicine (IOM):
a) Conduct a study to investigate the duplication of funding in the government, private and
non-profit sectors; and
b) Recommend strategies for reducing/eliminating duplication and promoting coordination.
Rationale behind this recommendation:
The priority status high incidence/high case fatality diseases enjoy in terms of clinical
research funding has an unintended consequence: funding for specific research projects is often
duplicated among the public, private, and non-profit sectors. The efforts of public advocacy
organizations also contribute to this problem, as the most active and influential advocacy groups
are often effective in ensuring increased or sustained funding for research consistent with their
respective missions.
To minimize such duplication, the Team recommends that the Institute of Medicine
undertake a partnership with public sector funders to (1) identify areas of duplicative research
and (2) to recommend strategies for eliminating duplication and promoting coordination.
The IOM is particularly well-positioned to participate in such a partnership because they
released a report in 1998 entitled "Improving Priority Setting and Public Input at the National
Institutes of Health." This report surveyed the informal processes through which NIH allocated
funds for biomedical research. As one of the few associations that has produced a detailed report
on priority-setting for research, the IOM possesses sufficient background to identify and
eliminate duplicative allocations. Moreover, because the IOM is well-respected by all manner of
stakeholders and is considered an important voice in health policy in general, its participation in
such a partnership would increase the level of success.
Policy Maker Focus: U.S. Congress
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Section 3: Requirements for CLAS (Culturally and Linguistically Appropriate
Services) Standards in Clinical Trials
Background:
In 2000, the Department of Health and Human Services (DHHS) Office of Minority
Health (OMH) published 14 National Standards on
Culturally and Linguistically Appropriate Services (CLAS). The CLAS standards are
designed to help health care organizations and individual providers make their practices more
culturally and linguistically accessible. Unfortunately, CLAS Standards are currently not
addressed consistently or systematically, if at all, in clinical trials.
Policy Recommendation 1: Commitment to CLAS Standards
All clinical research policy makers (e.g., accrediting bodies, public and private sector funders, as
well as healthcare agencies, corporations, institutions, and organizations sponsoring or
conducting clinical trials) will formally:
- Acknowledge their commitment to CLAS standards in their written policies and clinical
research guidelines.
Rationale behind this recommendation:
While OMH promulgated the CLAS standards in 2000, little progress has been made in
incorporating these standards into the clinical research enterprise. This is despite the fact that
several of the CLAS standards are mandatory for all recipients of federal funds. The EDICT
Team is aware that the Office of Civil Rights is currently considering whether failure to satisfy
the mandated standards qualifies as a violation of applicable civil rights law. Such penalties may
be necessary to produce compliance with the CLAS standards, but the EDICT Team is also
working with both OMH and the Office of Women's Health on a project designed to find ways
of applying the CLAS standards to clinical trials ("CLAS-ACT").
The goal of the CLAS-ACT project is to familiarize clinical trials investigators and staff
with CLAS standards, and to assess how well CLAS standards are implemented in specific
research studies as well as throughout individual agencies, institutions, and organizations.
Enhanced application of the CLAS standards into the clinical trials process could contribute
significantly to ameliorating disparities in clinical trials by ensuring that interaction and
communications with participants will proceed along culturally and linguistically appropriate
pathways.
Policy Maker Focus: Clinical research policy makers in the public, private and non-profit
sectors, as well as other entities that have influence on clinical trials and investigators.
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Policy Recommendation 2: Incorporation of CLAS Standards
All clinical research policy makers (e.g., accrediting bodies, public and private sector funders, as
well as healthcare agencies, corporations, institutions, and organizations sponsoring or
conducting clinical trials) will formally:
- Incorporate CLAS standards into their formal professional training, whether for
certification or as continuing education.
Rationale behind this recommendation: same as for previous recommendation
Policy Maker Focus: Clinical research policy makers in the public, private and non-profit
sectors, as well as other entities that have influence on clinical trials and investigators.
click here to submit your feedback on this policy recommendation
Policy Recommendation 3: Distribution of CLAS Standards
All clinical research policy makers (e.g., accrediting bodies, public and private sector funders, as
well as healthcare agencies, corporations, institutions, and organizations sponsoring or
conducting clinical trials) will formally:
- Distribute the CLAS standards to all researchers and staff engaged in clinical research
under their auspices.
Rationale behind this recommendation: same as for previous recommendation
Policy Maker Focus: Clinical research policy makers in the public, private and non-profit
sectors, as well as other entities that have influence on clinical trials and investigators.
click here to submit your feedback on this policy recommendation
Policy Recommendation 4: Adherence to CLAS Standards
All clinical research policy makers (e.g., accrediting bodies, public and private sector funders, as
well as healthcare agencies, corporations, institutions, and organizations sponsoring or
conducting clinical trials) will formally:
- Strive for adherence to the CLAS Standards related to
> language access services (Standards 4, 5, 6, and 7).
> diversity of staffing (Standard 2), and
> maintaining a current demographic, cultural, and epidemiological profile of the
community for purposes of recruiting and retaining representative participants in
clinical trials.
Rationale behind this recommendation: same as for previous recommendation
Policy Maker Focus: Clinical research policy makers in the public, private and non-profit
sectors, as well as other entities that have influence on clinical trials and investigators.
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