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Center for Drug Evaluation and Research (CDER) Small Business Assistance Program The Information Source for Regulated Domestic and International Small Pharmaceutical Business CAPT Brenda Stodart, USPHS Program Director CDER Small Business Assistance Division of Drug Information Office of Communications Center for Drug Evaluation and Research Food and Drug Administration 1

Food and Drug Administration CDRH NCTR CBER ORA Center for Drug Evaluation and Research CTP CFSAN CVM CDER 2

Organizational Structure CDER Office of Communications (OCOMM) Division of Drug Information (DDI) CDER Small Business Assistance 3

Mission Food and Drug Administration (FDA) Protect and advance the public health of the Nation Center for Drug Evaluation and Research (CDER) Promote and protect the health of Americans by assuring that prescription and over-the-counter drugs are safe and effective CDER Small Business Assistance Mission Promote productive interaction with regulated industry by assisting regulated domestic and international small pharmaceutical business who are seeking timely and accurate information relating to development and regulation of human drug products 4

Definition of Small Business The term small business is defined as a business that has no more than 500 employees, including affiliates. An affiliate is a business entity that has a relationship with a second business entity if one business entity controls, or has the power to control, the other business entity, or a third party controls, or has the power to control, both entities. (Section 735(9) of the Food, Drug &Cosmetic Act (FD&C Act)). 5

Resources Website Widget and Online Survey ListServ and Twitter FDA Basics for Industry Educational Outreach Inquiries 6

Website 7

Widget and Online Survey Widget Online survey 8

Twitter, FDA Basics for Industry and ListServ 9

FDA Basics for Industry Contact portal Navigation guide A Z index Frequently Asked Questions Educational Resources 10

Educational Outreach Webinars Workshops CDERLearn Industry meetings Exhibits Presentations 11

Educational Outreach- CDERLearn The Past, Present, and Future of FDA Human Drug Regulation Bringing an Unapproved Drug Into Compliance The FDA Process for Approving Generic Drugs An Introduction to the Improved FDA Prescription Drug Labeling 12

Determination of regulatory status ( is it a drug, device, food) Financial Incentives Pre-IND meetings Identify appropriate review division and facilitate interaction with SB entity and division GMP issues Inquiries Imports Labeling OTC Monographs Medical gases Pediatric exclusivity Repackaging Orphan drug designation Unapproved drugs Registration and listing queries Clinical studies 13

Financial Incentives Prescription Drug User Fee Act (PDUFA) user fee waiver: http://www.fda.gov/forindustry/userfees/prescriptiondruguserfee/defaul t.htm Guidance for Industry User Fee Waivers, Reductions, and Refunds for Drug and Biological products (03/11/11) http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinfo rmation/guidances/ucm079298.pdf Public Health Barrier to Innovation Small Business 14

Financial Incentives - continued Under section 736(d)(1)(D) of the Act, an applicant is eligible for a waiver of the application fee if the applicant is a small business submitting its first human drug application to the Agency for review and does not have another product approved under a human drug application and introduced or delivered for introduction into interstate commerce. An applicant is eligible for a small business waiver when: 15

Financial Incentives continued The applicant employs fewer than 500 employees, including employees of affiliates; The applicant does not have a drug product that has been approved under a human drug application and introduced or delivered for introduction into interstate commerce; and The applicant, including its affiliates, is submitting its first human drug application. To qualify for a small business waiver, an applicant must meet all of these criteria. http://www.fda.gov/drugs/developmentapprovalprocess/smallbusinessassist ance/ucm069943.htm http://www.fda.gov/forindustry/userfees/prescriptiondruguserfee/ucm0933 76.htm 16

Applications Financial Incentives continued Requiring clinical data $1,542,000 Not requiring clinical data $771,000 Establishments $497,200 Product $86,520 17

Pre-IND Meeting Identifying and avoiding unnecessary studies Ensuring that necessary studies are designed to provide useful information Gaining FDA support for a proposed strategy Minimizing potential for clinical hold Providing opportunity for creative exchange of ideas Obtaining regulatory insight Minimizing costs Clearly defining endpoints and goals of the development program Allowing early interactions/negotiations with FDA 18

Pre-IND Meeting Request Listing of specific questions categorized and grouped by discipline, for example, chemistry, manufacturing, and controls (CMC), pharmacology/toxicology, clinical pharmacology and biopharmaceutics, and clinical investigations Quantitative composition (all ingredients by percent composition) of the drug proposed for use in the study to be discussed Dosing regimen, including concentration, amount dosed, and frequency and duration of dosing if known 19

Pre-IND Meeting Packet Provides the historical background information on the chemical development concept Provides information on the active ingredient Provides an initial clinical and preclinical development strategy Provides future development strategy including product scaleup and final formulation, and animal and clinical studies proposed in support of an NDA Provides FDA with a clear and concise overview of the planned development program Allows FDA the opportunity to comment on a proposed program of development www.fda.gov/drugs/developmentapprovalprocess/smallbusinessassistance/ucm069906.htm 20

Audience* Demographics 95% Small Business (other: consultants, large pharma, etc.) 56% of the companies employ <10 people Location Most of clientele is in the U.S. Global reach: India, China, Mexico, Switzerland, Spain, Sri Lanka, Canada, Jordan, Chile. Industry experience 39% have submitted or are in the process of submitting a drug application to FDA 87% do not have an FDA approved drug application * This information was extracted 3/2011 from a survey posted on our SB website, and may not be reflective of our entire audience 21

Accomplishments # of Phone Calls Received by CDER SB Program (2010-2011) Number of Phone Calls Received 120 100 80 60 40 20 0 97 90 79 46 23 24 21 41 10 6 7 5 8 23 30 29 Jan Feb Mar Apr May Jun Jul Aug Sep Oct Nov Dec Jan Feb Mar Apr Month (2010-2011) # of Incoming Phone Calls to DDI from Small Business (2011) * Number of Incoming SB Phone Calls to DDI * 300 250 200 150 100 50 0 205 (9%) 156 (6%) 244 (8%) 228 (8%) Jan Feb Mar April Month (2011) 22

Accomplishments Number of Emails Received in CDER SB Account in 2010-2011 Number of Emails Received 200 150 100 50 0 187 123 65 43 28 28 36 35 47 12 12 12 4 12 16 60 Jan Feb Mar Apr May Jun Jul Aug Sep Oct Nov Dec Jan Feb Mar Apr Month (2010-2011) # of Emails Received in DDI account from Small Business (2011) Number of emails received 400 300 200 100 0 217 (11%) 215 (13%) 344 (19%) Jan Feb Mar Apr Month (2011) * % indicates percentage of total emails received by DDI 244 (21%) 23

Goals Increase inquiries to SB account by 50% in 2011 Increase global audience and presence Webinars one per month Workshops at least 2 per year Develop CDERLearn courses (2 per year) Increase visibility Speaking at Industry conferences Networking within and outside of FDA Holding workshops at the 5 Regional Offices 24

CDER Small Business Contact Telephone 1.866.405.5367 or 301.796.6707 Web-Site - http://www.fda.gov/smallbusinessdrugs Email CDERSmallBusiness@fda.hhs.gov Mail - 10001 New Hampshire Ave. Hillandale Building, 4 th Floor Silver Spring, MD 20993-0002 Sign up for the CDER Small Business Listserv on our website Download our Widget from website 25