CLINICAL NEWS
NCI Collaborating With Multiple Myeloma Research Foundation
to Collect Genome Data
The National Cancer Institute (NCI) partnered with the Multiple Myeloma Research Foundation (MMRF) to incorporate MMRF’s genomic and clinical data into the NCI’s Genomic Data Commons (GDC), a core component of the
Cancer Moonshot Initiative. MMRF is the first nonprofit organization to donate information to the GDC, which will
include data for more than 30,000 patients with multiple myeloma (MM).
“Data sharing is essential to advancing cancer research, and I cannot overstate the value of the data that MMRF
is providing,” said Doug Lowy, MD, acting director of the NCI.
MMRF’s CoMMpass (Relating Clinical Outcomes in MM to Personal Assessment of Genetic Profile) study,
which has enrolled more than 1,150 patients and is the largest genomic and clinical study of MM, is of particular
interest.
The genomic data from these analyses will be deposited in the GDC, with an anticipated sample size of nearly
1,000 cases by the spring of 2017.
Source: National Cancer Institute press release, September 28, 2016.
ICER Examining
Suggestions from
Industry and
Pharmaceutical
Companies on Drug
Cost-Effectiveness
Analyses
Earlier this year, the Institute for Clinical
and Economic Review (ICER) released
a draft report on treatment options for
MM, focusing on adults with relapsed or
refractory disease, who are not currently
on maintenance therapy, and who are not
considered eligible for hematopoietic cell
transplantation (HCT). Industry and pharmaceutical representatives, including the
American Society of Hematology (ASH),
submitted comments to ICER expressing
concern with the group’s comparative-effectiveness analyses, citing a need for more
transparency on review methods.
Since then, ICER has released more
than 300 pages of comments on its
drug review process from more than 50
pharmaceutical companies and patient
organizations and has announced that it
is meeting with several representatives to
discuss potential changes to the process.
“We want to make sure that the rules
of the road are clear [and] that they
appear valid,” said Steven D. Pearson,
MD, MSc, president of ICER. “For that
to work, we want people to feel like their
voices are being heard.”
In its letter to ICER requesting more
openness in its review methods, ASH
expressed concern with the group’s
comparative-effectiveness analysis, focusing on the difference between clinical
trial data and clinical experience that is
gained following approval.
“Unfortunately, the scope of ICER’s
analysis is far too narrow because it
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does not represent the realities of clinical practice,” ASH President Charles S.
Abrams, MD, and President-Elect Kenneth Anderson, MD, wrote. “As such,
ASH believes that the type of analysis
has only limited value in determining
the just price and utility of novel drugs
and drug combinations in this rapidly
advancing field.”
Despite these concerns, “ASH
strongly encourages other efforts to measure the comparative effectiveness of new
myeloma drugs, such as comparativeeffectiveness trials, or the development of
clinical data registries,” they continued.
ICER plans to revise and publish new
draft policies in December for another
round of public comment.
Sources: Fierce Pharma, October 5, 2016; ASH correspondence, April
20, 2016.
FDA Encourages
Collaborative and
Innovative Approach
to Clinical Trial Designs
The FDA Oncology Center of Excellence
announced a series of efforts to design
more efficient clinical trials. According to
the FDA, “designing more efficient trials
that produce better results can dramatically accelerate how quickly new therapies are brought to patients, and decrease
the cost of doing so.”
Some of the proposed efforts include:
• working with stakeholders across
government and the industry to
modify the eligibility criteria for
clinical trials, which could expand
the number of people who qualify,
open new opportunities for participation, and enhance the generalizability of the outcomes
phase I, II, and III drug development paradigm to a more seamless
approach that could expedite the
regulatory pathway and provide
earlier access to highly effective
therapeutic drugs
• using common control trials to decrease the overall number of patients
needed for recruitment and enrollment, optimize clinical trial resources, and potentially decrease the time
it takes to launch a new study
• encouraging the use of large simple
trials, which generally use easily
measured endpoints that are well
understood, thus optimizing the collection of data for safety or secondary
efficacy endpoints and reducing the
amount of data needed compared
with conventional randomized trials
This effort is part of the Cancer Moonshot Initiative, led by Vice President
Joseph Biden, which seeks to simplify
the process for patients to find and enter
clinical trials as part of the overall program goal. “Right now, less than 5 percent of cancer patients enroll in a clinical
trial, often because patients and doctors
don’t know what trials are available,” said
Vice President Biden.
In addition, the NIH announced that
it is interested in making ClinicalTrials.
gov more user friendly. Investigators
will also be required to publish summaries of trial findings whether or not
they resulted in a drug approval. “This
will enhance transparency within the
clinical trials enterprise, allowing people
to make more informed decisions about
participating in research,” according to a
statement from NIH.
Sources: U.S. Food and Drug Administration news release, September
16, 2016; Reuters, September 16, 2016.
• shifting away from the conventional
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