Notice of Approval of Product Under Voucher: Rare Pediatric Disease Priority Review Voucher, 19085-19086 [2019-09005]
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Federal Register / Vol. 84, No. 86 / Friday, May 3, 2019 / Notices
benefit-risk assessments and used case
examples, explored PPI methods, and
future research topics to improve the
use of PPI in regulatory decisions.
Based on feedback attained during the
workshop, FDA identified the following
parameters to assist in the identification
of the priority list of patient preferencesensitive areas for medical device
review, where:
• FDA staff are looking to better
understand the full impact of the
disease or condition and treatment
options on patients and/or caregivers;
• Patients may value the benefits and
risks of a technology or treatment
differently from healthcare professionals
and/or caregivers;
• Population-level differences in
patient perspectives are not well
understood, due to differences in:
Æ Demographic characteristics;
Æ Stages of disease; or
Æ Disease phenotype; and
• There is significant public health
impact (such as high mortality or
morbidity rates and high prevalence
rates of the disease, or few treatment
options available such as in rare
diseases).
fda/cdrh-patient-engagement/prioritylist-patient-preference-sensitive-areas.
In addition, FDA is interested in
responses to the following questions:
1. Do any existing topics on the
Priority List of Patient PreferenceSensitive Areas need to be refined to
better represent patient preferencesensitive areas important to regulatory
efforts? And, if so, how? Please provide
an explanation to support any
recommended refinements.
2. Are there other areas not listed on
the FDA website at https://www.fda.gov/
about-fda/cdrh-patient-engagement/
priority-list-patient-preference-sensitiveareas that FDA should consider as
priority patient preference-sensitive
areas? If there are additional areas for
consideration, please identify and
provide an explanation for each
additional area using the parameters
outlined in Section I: Background.
3. Are there ongoing studies or
published studies that adequately
address any of these patient preferencesensitive areas in a regulatory context?
If so, please provide information or
references regarding the studies.
II. Patient Preference-Sensitive Priority
Areas
IV. References
Based on the above parameters, FDA
generated a list of priority preferencesensitive areas, and organized the areas
into the following categories:
• Patient values in diagnosis and
treatment;
• Relevant clinical endpoints for
specific patient populations;
• Patient benefit-risk trade-offs for
treatment options or diagnostic
approaches; and
• Impact of uncertainty in the benefitrisk tradeoffs.
The current collated list of identified
patient preference-sensitive areas can be
found on the FDA website at https://
www.fda.gov/about-fda/cdrh-patientengagement/priority-list-patientpreference-sensitive-areas. The
priorities listed on the web page may be
broadly applicable to many diagnostic/
therapeutic areas, while others are
specific to a disease/condition or
technology. This is not an exhaustive
list of all patient preference-sensitive
areas, and the prioritization of these
areas may shift over time as health
technologies and patient preference
methodologies advance.
The following references are on
display at the Dockets Management Staff
(see ADDRESSES) and are available for
viewing by interested persons between
9 a.m. and 4 p.m., Monday through
Friday; they are also available
electronically at https://
www.regulations.gov. FDA has verified
the website addresses, as of the date this
document publishes in the Federal
Register, but websites are subject to
change over time.
III. Other Issues for Consideration
FDA is soliciting public input from
interested persons on the identified
priority list of patient preferencesensitive topics captured on the FDA
website at https://www.fda.gov/about-
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1. Patient Preference Information—Voluntary
Submission, Review in Premarket
Approval Applications, Humanitarian
Device Exemption Applications, and De
Novo Requests, and Inclusion in
Decision Summaries and Device
Labeling—Guidance for Industry, Food
and Drug Administration Staff, and
Other Stakeholders, available at https://
www.fda.gov/regulatory-information/
search-fda-guidance-documents/patientpreference-information-voluntarysubmission-review-premarket-approvalapplications.
2. MDUFA Performance Goals and
Procedures, Fiscal Years 2019 through
2022, available at https://www.fda.gov/
media/102699/download.
Advancing Use of Patient Preference
Information as Scientific Evidence in
Medical Product Evaluation Workshop at
https://www.fda.gov/science-research/
advancing-regulatory-science/advancinguse-patient-preference-informationscientific-evidence-medical-productevaluation.
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19085
Dated: April 29, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019–09051 Filed 5–2–19; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2018–N–1262]
Notice of Approval of Product Under
Voucher: Rare Pediatric Disease
Priority Review Voucher
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice.
The Food and Drug
Administration (FDA) is announcing the
issuance of approval of a product
redeeming a priority review voucher.
The Federal Food, Drug, and Cosmetic
Act (FD&C Act), as amended by the
Food and Drug Administration Safety
and Innovation Act (FDASIA),
authorizes FDA to award priority review
vouchers to sponsors of approved rare
pediatric disease product applications
that meet certain criteria. FDA is
required to publish notice of the
issuance of vouchers as well as the
approval of products redeeming a
voucher. FDA has determined that
ULTOMIRIS (ravulizumab-cwvz)
approved December 21, 2018, meets the
redemption criteria.
FOR FURTHER INFORMATION CONTACT:
Althea Cuff, Center for Drug Evaluation
and Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Silver Spring, MD 20993–0002,
301–796–4061, Fax: 301–796–9858,
email: althea.cuff@fda.hhs.gov.
SUPPLEMENTARY INFORMATION: Under
section 529 of the FD&C Act (21 U.S.C.
360ff), which was added by FDASIA,
FDA will report the issuance of rare
pediatric disease priority review
vouchers and the approval of products
for which a voucher was redeemed.
FDA has determined that ULTOMIRIS
(ravulizumab-cwvz) approved December
21, 2018, meets the redemption criteria.
For further information about the Rare
Pediatric Disease Priority Review
Voucher Program and for a link to the
full text of section 529 of the FD&C Act,
go to https://www.fda.gov/ForIndustry/
DevelopingProductsforRareDiseases
Conditions/RarePediatricDiseasePriority
VoucherProgram/default.htm. For
further information about ULTOMIRIS
(ravulizumab-cwvz) go to the ‘‘Drugs@
FDA’’ website at https://
SUMMARY:
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Federal Register / Vol. 84, No. 86 / Friday, May 3, 2019 / Notices
www.accessdata.fda.gov/scripts/cder/
daf/.
Dated: April 29, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019–09005 Filed 5–2–19; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Request for Information (RFI):
Developing an STD Federal Action
Plan
Office of HIV/AIDS and
Infectious Disease Policy, Office of the
Assistant Secretary for Health, Office of
the Secretary, Department of Health and
Human Services.
ACTION: Notice.
AGENCY:
To help inform the
development of the Sexually
Transmitted Diseases (STD) Federal
Action Plan, HHS seeks input from
stakeholders on what strategies can be
implemented by federal agencies to
improve the efficiency, effectiveness,
coordination, accountability, and
impact of our national response to
increasing rates of STDs.
DATES: To be assured consideration,
comments must be received at the
address provided below, no later than
5:00 p.m. ET on June 3, 2019.
ADDRESSES: Electronic responses are
strongly preferred and may be addressed
to STDPlan@hhs.gov. Written responses
should be addressed to: U.S.
Department of Health and Human
Services, 330 C Street SW, Room L001,
Washington, DC 20024; Attention STD
RFI.
FOR FURTHER INFORMATION CONTACT:
Melissa Habel, MPH in the HHS Office
of HIV/AIDS and Infectious Disease
Policy, (202) 795–7697.
SUPPLEMENTARY INFORMATION: Rates of
sexually transmitted diseases (STDs) in
2017 reached an all-time high among
males and females and all racial and
ethnic groups. Since 2013, reported
chlamydia rates have increased 22%,
gonorrhea rates 67%, syphilis rates
76%, and congenital syphilis rates
154%; the combined number of cases
was 2.3 million up from 1.8 million in
2013.1 These infections can lead to longterm health consequences such as
infertility and can facilitate HIV
transmission. While gonorrhea,
chlamydia and syphilis infections have
grown considerably over the past four
years, human papillomavirus (HPV)
remains the most commonly sexually
transmitted infection in the U.S.,
amozie on DSK9F9SC42PROD with NOTICES
SUMMARY:
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affecting close to half of adults of
reproductive age. HPV infections result
in approximately 33,700 cases of certain
types of cancer each year in the U.S.2
Most of these cancers are preventable
through the use of the HPV vaccination
series. These numbers represent real
people and expose hidden fragile
populations who are not getting the
preventive services and health care they
need. While STDs affect all groups of
the U.S. population, they
disproportionately affect certain
vulnerable groups such as pregnant
women, youth ages 15–24 years, men
who have sex with men, and racial and
ethnic minorities. Beyond the impact on
an individual’s health, in 2013 it was
estimated that STDs cost the U.S. health
care system more than $16 billion
annually, and STDs have increased
dramatically since then.3
To respond and address the STD
public health epidemic, OHAIDP in
collaboration with other federal partners
is leading and coordinating
development of a STD Federal Action
Plan. The development process for the
action plan will seek input from subject
matter experts, nonfederal partners and
stakeholders including health care
providers and systems, state, tribal, and
local health departments, communitybased and faith-based organizations,
national professional organizations,
researchers, advocates, and persons
whose lives have been affected by these
infections. The action plan is expected
to address prevention, diagnosis, care
and treatment, as well as coordination
of efforts, policies, and programs
throughout the federal government. It
will also address stigma, discrimination,
co-infections (e.g., HIV and viral
hepatitis), and social determinants of
health.
This request for information seeks
public input on how the federal
government should address the rising
rates of STDs and what strategies can be
implemented to improve the efficiency,
effectiveness, coordination,
accountability, and impact of the federal
response to STD prevention, care and
treatment policies, services and
programs. The information received will
inform the STD Federal Action Plan.
Topics of interest include but are not
limited to the following:
1. How should the federal government
address the rising rates of STDs?
2. What strategies can be
implemented by federal agencies to
improve the efficiency, effectiveness,
coordination, accountability, and
impact of our national response to
increasing rates of STDs for all priority
populations?
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3. What are the barriers to people
getting the quality STD health services
they deserve? What strategies can be
implemented by federal agencies to
overcome these barriers?
4. How can federal agencies influence,
design and implement STD-related
policies, services and programs in
innovative and culturally-responsive
ways for priority populations?
5. How can the federal government
help to reduce STD-associated stigma
and discrimination?
Dated: April 11, 2019.
Tammy R. Beckham,
Director, Office of HIV/AIDS and Infectious
Disease Policy.
Footnotes
1. Centers for Disease Control and
Prevention. Sexually Transmitted
Disease Surveillance 2017. Atlanta: U.S:
Department of Health and Human
Services, 2018: Available at https://
www.cdc.gov/std/stats.
2. Eng TR, Butler WT, editors; Institute of
Medicine (US). Summary: The hidden
epidemic: Confronting sexually
transmitted diseases. Washington (DC):
National Academy Press; 1997. p. 43.
3. Owusu-Edusei K Jr, Chesson HW, Gift TL,
et al. The estimated direct medical cost
of selected sexually transmitted
infections in the United States, 2008. Sex
Transm Dis 2013; 40(3):197–201.
DOI:10.1097/OLQ.0b013e318285c6d2.
[FR Doc. 2019–09113 Filed 5–2–19; 8:45 am]
BILLING CODE P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
[OMHA–1901–N]
Medicare Program; Administrative Law
Judge Hearing Program for Medicare
Claim and Entitlement Appeals;
Quarterly Listing of Program
Issuances—January Through March
2019
Office of Medicare Hearings
and Appeals (OMHA), HHS.
ACTION: Notice.
AGENCY:
This quarterly notice lists the
OMHA Case Processing Manual (OCPM)
instructions that were published from
January through March 2019. This
manual standardizes the day-to-day
procedures for carrying out adjudicative
functions, in accordance with
applicable statutes, regulations, and
OMHA directives, and gives OMHA
staff direction for processing appeals at
the OMHA level of adjudication.
FOR FURTHER INFORMATION CONTACT:
Jason Green, by telephone at (571) 777–
2723, or by email at jason.green@
hhs.gov.
SUMMARY:
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]]>Agencies
[Federal Register Volume 84, Number 86 (Friday, May 3, 2019)]
[Notices]
[Pages 19085-19086]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-09005]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2018-N-1262]
Notice of Approval of Product Under Voucher: Rare Pediatric
Disease Priority Review Voucher
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration (FDA) is announcing the
issuance of approval of a product redeeming a priority review voucher.
The Federal Food, Drug, and Cosmetic Act (FD&C Act), as amended by the
Food and Drug Administration Safety and Innovation Act (FDASIA),
authorizes FDA to award priority review vouchers to sponsors of
approved rare pediatric disease product applications that meet certain
criteria. FDA is required to publish notice of the issuance of vouchers
as well as the approval of products redeeming a voucher. FDA has
determined that ULTOMIRIS (ravulizumab-cwvz) approved December 21,
2018, meets the redemption criteria.
FOR FURTHER INFORMATION CONTACT: Althea Cuff, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-4061, Fax: 301-
796-9858, email: [email protected].
SUPPLEMENTARY INFORMATION: Under section 529 of the FD&C Act (21 U.S.C.
360ff), which was added by FDASIA, FDA will report the issuance of rare
pediatric disease priority review vouchers and the approval of products
for which a voucher was redeemed. FDA has determined that ULTOMIRIS
(ravulizumab-cwvz) approved December 21, 2018, meets the redemption
criteria.
For further information about the Rare Pediatric Disease Priority
Review Voucher Program and for a link to the full text of section 529
of the FD&C Act, go to https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/RarePediatricDiseasePriorityVoucherProgram/default.htm. For further
information about ULTOMIRIS (ravulizumab-cwvz) go to the ``[email protected]''
website at https://
[[Page 19086]]
www.accessdata.fda.gov/scripts/cder/daf/.
Dated: April 29, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019-09005 Filed 5-2-19; 8:45 am]
BILLING CODE 4164-01-P
