80 FR 80776 - Externally-Led Patient-Focused Drug Development Meetings
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 80, Issue 248 (December 28, 2015)
Food and Drug Administration
Federal Register Volume 80, Issue 248 (December 28, 2015)
| Page Range | 80776-80777 | |
| FR Document | 2015-32476 |
[Federal Register Volume 80, Number 248 (Monday, December 28, 2015)] [Notices] [Pages 80776-80777] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2015-32476] ----------------------------------------------------------------------- DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA-2015-N-0001] Externally-Led Patient-Focused Drug Development Meetings AGENCY: Food and Drug Administration, HHS. ACTION: Notice. ----------------------------------------------------------------------- SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing the opportunity for externally-led patient-focused drug development meetings. The Patient-Focused Drug Development (PFDD) initiative is part of FDA's commitments under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). The PFDD initiative aims to more systematically obtain the patient perspective on specific diseases and their treatments. FDA recognizes that there are many more disease areas than can be addressed in the planned FDA meetings under PDUFA V. To help expand the benefits of FDA's PFDD initiative, FDA welcomes patient organizations to identify and organize patient-focused collaborations to generate public input on other disease areas, using the process established through Patient-Focused Drug Development as a model. ADDRESSES: FDA recommends that patient organizations who are interested in conducting an externally-led PFDD meeting initially engage with FDA by submitting a letter of intent (LOI) to [email protected]. Submission details are outlined on FDA's Web site: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm453856.htm. FOR FURTHER INFORMATION CONTACT: Pujita Vaidya, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, rm. 1144, Silver Spring, MD 20993-0002, 301- 796-0684. SUPPLEMENTARY INFORMATION: As part of its commitments under the Prescription Drug User Fee Act reauthorization of 2012, FDA has taken several steps to inform the benefit-risk assessments that inform CDER's regulatory decisions concerning new drugs. Among these efforts is the PFDD initiative that aims to more systematically obtain the patient perspective on specific diseases and their treatments. FDA has committed to obtaining the patient perspective on at least 20 disease areas during the course of PDUFA V. PFDD meetings give FDA an important opportunity to hear directly from patients, patient advocates, and caretakers about the symptoms that matter most to them; the impact the disease has on patients' daily lives; and patients' experiences with currently available treatments. The patient perspective is critical in helping FDA understand the context in which regulatory decisions are made for new drugs. This patient input can inform FDA's decisions and oversight both during drug development and during our review of a marketing application. The Agency recognizes that there has been growing external interest in expanding efforts to gather patient input in support of drug development and evaluation. To help expand the benefits of FDA's PFDD initiative, FDA welcomes patient organizations to identify and organize patient-focused collaborations to generate public input on other disease areas, using the process established through Patient-Focused Drug Development as a model. An externally-led PFDD meeting and any resulting products (e.g., surveys or reports) will not be considered FDA-sponsored or FDA-endorsed, and FDA does not guarantee specific involvement in such meetings. However, FDA will be [[Page 80777]] open to participating in a well-designed and well-conducted meeting on a case-by-case basis. Given the expanse of diseases affecting the U.S. patient population and the effort required to conduct a successful PFDD meeting, externally-led PFDD meetings should target disease areas where there is an identified need for patient input on topics related to drug development. FDA will determine its level of participation in these meetings on an individual basis, taking into account a number of factors, including any identified need for a better understanding of patient perspective, recent interactions with patient stakeholders, proposed meeting details, and FDA staff capacity. More information regarding considerations to take into account when deciding to plan an externally-led PFDD meeting can be found on this Web site: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm453856.htm. FDA recommends that patient organizations who are interested in conducting an externally-led PFDD meeting submit an LOI that communicates (1) the value of the proposed meeting in the context of drug development for a particular disease area, and (2) important details regarding the meeting plan. Guidelines for developing a letter of intent are provided here: http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM453857.pdf. Please submit the letter of intent to [email protected]. FDA's CDER Office of Strategic Programs will receive and review the letter. Dated: December 21, 2015. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2015-32476 Filed 12-24-15; 8:45 am] BILLING CODE 4164-01-P
![80776 Federal Register / Vol. 80, No. 248 / Monday, December 28, 2015 / Notices
Therapeutic Equivalence Evaluations,’’ evaluated relevant literature and data areas, using the process established
which is known generally as the for possible postmarketing adverse through Patient-Focused Drug
‘‘Orange Book.’’ Under FDA regulations, events. We have reviewed the available Development as a model.
drugs are removed from the list if the evidence and determined that the ADDRESSES: FDA recommends that
Agency withdraws or suspends products were not withdrawn from sale patient organizations who are interested
approval of the drug’s NDA or ANDA for reasons of safety or effectiveness. in conducting an externally-led PFDD
for reasons of safety or effectiveness or Accordingly, the Agency will meeting initially engage with FDA by
if FDA determines that the listed drug continue to list KYTRIL (granisteron submitting a letter of intent (LOI) to
was withdrawn from sale for reasons of hydrochloride) tablets, EQ 1 mg and 2 patientfocused@fda.hhs.gov.
safety or effectiveness (21 CFR 314.162). mg base, in the ‘‘Discontinued Drug Submission details are outlined on
A person may petition the Agency to Product List’’ section of the Orange FDA’s Web site: http://www.fda.gov/
determine, or the Agency may Book. The ‘‘Discontinued Drug Product ForIndustry/UserFees/
determine on its own initiative, whether List’’ delineates, among other items, PrescriptionDrugUserFee/
a listed drug was withdrawn from sale drug products that have been ucm453856.htm.
for reasons of safety or effectiveness. discontinued from marketing for reasons FOR FURTHER INFORMATION CONTACT:
This determination may be made at any other than safety or effectiveness. Pujita Vaidya, Center for Drug
time after the drug has been withdrawn ANDAs that refer to KYTRIL Evaluation and Research, Food and
from sale, but must be made prior to (granisteron hydrochloride) tablets, EQ Drug Administration, 10903 New
approving an ANDA that refers to the 1 mg and 2 mg base, may be approved Hampshire Ave., Bldg. 51, rm. 1144,
listed drug (§ 314.161 (21 CFR 314.161)). by the Agency as long as they meet all Silver Spring, MD 20993–0002, 301–
FDA may not approve an ANDA that other legal and regulatory requirements 796–0684.
does not refer to a listed drug. for the approval of ANDAs. If FDA
KYTRIL (granisetron hydrochloride) SUPPLEMENTARY INFORMATION: As part of
determines that labeling for this drug its commitments under the Prescription
tablets, EQ 1 mg and 2 mg base, are the product should be revised to meet
subject of NDA 020305, held by Drug User Fee Act reauthorization of
current standards, the Agency will 2012, FDA has taken several steps to
Hoffmann-La Roche, Inc., and initially advise ANDA applicants to submit such
approved on March 16, 1995. KYTRIL is inform the benefit-risk assessments that
labeling. inform CDER’s regulatory decisions
indicated for the prevention of nausea
and/or vomiting associated with initial Dated: December 21, 2015. concerning new drugs. Among these
and repeat courses of emetogenic cancer Leslie Kux, efforts is the PFDD initiative that aims
therapy, including high-dose cisplatin, Associate Commissioner for Policy. to more systematically obtain the
and for the prevention and treatment of [FR Doc. 2015–32496 Filed 12–24–15; 8:45 am] patient perspective on specific diseases
postoperative nausea and vomiting in BILLING CODE 4164–01–P
and their treatments. FDA has
adults. committed to obtaining the patient
On April 30, 2012, Hoffman-La Roche perspective on at least 20 disease areas
notified FDA that KYTRIL (granisteron DEPARTMENT OF HEALTH AND during the course of PDUFA V. PFDD
hydrochloride) tablets, EQ 1 mg and 2 HUMAN SERVICES meetings give FDA an important
mg base, were being discontinued, and opportunity to hear directly from
FDA moved the drug products to the Food and Drug Administration patients, patient advocates, and
‘‘Discontinued Drug Product List’’ caretakers about the symptoms that
[Docket No. FDA–2015–N–0001]
section of the Orange Book. matter most to them; the impact the
Kurt R. Karst, on behalf of Hyman, Externally-Led Patient-Focused Drug disease has on patients’ daily lives; and
Phelps & McNamara, P.C., submitted a Development Meetings patients’ experiences with currently
citizen petition dated May 27, 2015 available treatments. The patient
(Docket No. FDA–2015–P–1898), under AGENCY: Food and Drug Administration, perspective is critical in helping FDA
21 CFR 10.30, requesting that the HHS. understand the context in which
Agency determine whether KYTRIL ACTION: Notice. regulatory decisions are made for new
(granisteron hydrochloride) tablets, EQ drugs. This patient input can inform
1 mg and 2 mg base, were withdrawn SUMMARY: The Food and Drug FDA’s decisions and oversight both
from sale for reasons of safety or Administration (FDA or Agency) is during drug development and during
effectiveness. announcing the opportunity for our review of a marketing application.
After considering the citizen petition externally-led patient-focused drug The Agency recognizes that there has
and reviewing Agency records and development meetings. The Patient- been growing external interest in
based on the information we have at this Focused Drug Development (PFDD) expanding efforts to gather patient input
time, FDA has determined under initiative is part of FDA’s commitments in support of drug development and
§ 314.161 that KYTRIL (granisteron under the fifth authorization of the evaluation. To help expand the benefits
hydrochloride) tablets, EQ 1 mg and 2 Prescription Drug User Fee Act (PDUFA of FDA’s PFDD initiative, FDA
mg base, were not withdrawn for V). The PFDD initiative aims to more welcomes patient organizations to
reasons of safety or effectiveness. The systematically obtain the patient identify and organize patient-focused
petitioner has identified no data or other perspective on specific diseases and collaborations to generate public input
information suggesting that KYTRIL their treatments. FDA recognizes that on other disease areas, using the process
mstockstill on DSK4VPTVN1PROD with NOTICES
(granisteron hydrochloride) tablets, EQ there are many more disease areas than established through Patient-Focused
1 mg and 2 mg base, were withdrawn for can be addressed in the planned FDA Drug Development as a model. An
reasons of safety or effectiveness We meetings under PDUFA V. To help externally-led PFDD meeting and any
have carefully reviewed our files for expand the benefits of FDA’s PFDD resulting products (e.g., surveys or
records concerning the withdrawal of initiative, FDA welcomes patient reports) will not be considered FDA-
KYTRIL (granisteron hydrochloride) organizations to identify and organize sponsored or FDA-endorsed, and FDA
tablets, EQ 1 mg and 2 mg base, from patient-focused collaborations to does not guarantee specific involvement
sale. We have also independently generate public input on other disease in such meetings. However, FDA will be
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![Federal Register / Vol. 80, No. 248 / Monday, December 28, 2015 / Notices 80777
open to participating in a well-designed Paperwork Reduction Act of 1995), the about treating sickle cell disease as well
and well-conducted meeting on a case- Health Resources and Services as increase the number of sickle cell
by-case basis. Given the expanse of Administration (HRSA) announces patients that are seen by providers
diseases affecting the U.S. patient plans to submit an Information knowledgeable about sickle cell disease.
population and the effort required to Collection Request (ICR), described To achieve the goals and objectives of
conduct a successful PFDD meeting, below, to the Office of Management and the program, the SCDTDP uses quality
externally-led PFDD meetings should Budget (OMB). Prior to submitting the improvement (QI) methods in a
target disease areas where there is an ICR to OMB, HRSA seeks comments collective impact model which supports
identified need for patient input on from the public regarding the burden cross-sector collaboration for achieving
topics related to drug development. estimate, below, or any other aspect of measurable effects on major social
FDA will determine its level of the ICR. issues. The collective impact model
participation in these meetings on an DATES: Comments on this Information requires shared measurement which
individual basis, taking into account a Collection Request must be received no facilitates tracking progress in a
number of factors, including any later than February 26, 2016. standardized method in order to
identified need for a better ADDRESSES: Submit your comments to promote learning and enhance
understanding of patient perspective, paperwork@hrsa.gov or mail the HRSA continuous improvement.
recent interactions with patient Information Collection Clearance Need and Proposed Use of the
stakeholders, proposed meeting details, Officer, Room 10C–24, Parklawn Information: The purpose of the
and FDA staff capacity. More Building, 5600 Fishers Lane, Rockville, proposed data collection strategy is to
information regarding considerations to MD 20857. implement a system to monitor the
take into account when deciding to plan FOR FURTHER INFORMATION CONTACT: To progress of MCHB-funded activities in
an externally-led PFDD meeting can be request more information on the improving care and health outcomes for
found on this Web site: http:// proposed project or to obtain a copy of individuals living with sickle cell
www.fda.gov/ForIndustry/UserFees/ the data collection plans and draft disease/trait and meeting the goals of
PrescriptionDrugUserFee/ instruments, email paperwork@hrsa.gov the SCDTDP. Each regional grantee site
ucm453856.htm. or call the HRSA Information Collection will be asked to report on a core set of
FDA recommends that patient Clearance Officer at (301) 443–1984. evidence-based measures related to
organizations who are interested in healthcare utilization among
SUPPLEMENTARY INFORMATION: When
conducting an externally-led PFDD individuals with sickle cell disease and
submitting comments or requesting
meeting submit an LOI that the quality of care of the SCD
information, please include the
communicates (1) the value of the population.
information request collection title for
proposed meeting in the context of drug The data collected for the Sickle Cell
reference.
development for a particular disease Information Collection Request Title: Disease Treatment Demonstration
area, and (2) important details regarding Sickle Cell Disease Treatment Program will consist of administrative
the meeting plan. Guidelines for Demonstration Program—Quality medical claims data collected from State
developing a letter of intent are Improvement Data Collection. Medicaid Programs and Medicaid
provided here: http://www.fda.gov/ OMB No. 0915–xxxx–New Managed Care Organizations that
downloads/ForIndustry/UserFees/ Abstract: In response to the growing administer Medicaid on behalf of states.
PrescriptionDrugUserFee/ need for resources devoted to sickle cell The data is collected either for or by
UCM453857.pdf. Please submit the disease and other hemoglobinopathies, State Medicaid offices for delivery of
letter of intent to patientfocused@ the United States Congress, under services subject to Medicaid
fda.hhs.gov. FDA’s CDER Office of Section 712 of the American Jobs reimbursement.
Strategic Programs will receive and Creation Act of 2004 (Pub. L. 108–357) The data collection strategy will
review the letter. (42 U.S.C. 300b–1 note), authorized a provide an effective and efficient
Dated: December 21, 2015. demonstration program for the mechanism to do the following: (1)
Leslie Kux, prevention and treatment of sickle cell Assess the improvements in access to
Associate Commissioner for Policy. disease (SCD) to be administered by the care for sickle cell patients provided by
[FR Doc. 2015–32476 Filed 12–24–15; 8:45 am] Maternal and Child Health Bureau activities in the SCDTDP; (2) collect,
BILLING CODE 4164–01–P
(MCHB) of the Health Resources and coordinate, and distribute data, best
Services Administration (HRSA) in the practices, and findings from regional
U.S. Department of Health and Human grantee sites to drive improvement on
DEPARTMENT OF HEALTH AND Services. The program is known as the quality measures; (3) refine a common
HUMAN SERVICES Sickle Cell Disease Treatment model protocol regarding the prevention
Demonstration Program (SCDTDP). The and treatment of sickle cell disease; (4)
Health Resources and Services SCDTDP is designed to improve access examine/address barriers that
Administration to services for individuals with sickle individuals and families living with
cell disease, improve and expand sickle cell disease face when accessing
Agency Information Collection patient and provider education, and quality health care and health
Activities: Proposed Collection: Public improve and expand the continuity and education; (5) evaluate the grantees’
Comment Request coordination of service delivery for performance in meeting the objectives of
mstockstill on DSK4VPTVN1PROD with NOTICES
AGENCY: Health Resources and Services individuals with sickle cell disease and the SCDTDP; and (6) provide HRSA and
Administration, HHS. sickle cell trait. The specific aims for the Congress with information on the
ACTION: Notice. program are threefold: (1) Increase the overall progress of the program.
number of providers treating persons Likely Respondents: Four regional
SUMMARY: In compliance with the with sickle cell disease, (2) increase the grantee sites funded by HRSA under the
requirement for opportunity for public number of providers prescribing SCDTDP will be the respondents for this
comment on proposed data collection hydroxyurea, and (3) increase the data collection activity and submit
projects (Section 3506(c)(2)(A) of the number of providers knowledgeable responses gathered from State Medicaid
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Document Created: 2018-03-02 09:23:50
Document Modified: 2018-03-02 09:23:50
| Category | Regulatory Information | |
| Collection | Federal Register | |
| sudoc Class | AE 2.7: GS 4.107: AE 2.106: | |
| Publisher | Office of the Federal Register, National Archives and Records Administration | |
| Section | Notices | |
| Action | Notice. | |
| Contact | Pujita Vaidya, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, rm. 1144, Silver Spring, MD 20993-0002, 301- 796-0684. | |
| FR Citation | 80 FR 80776 |
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