NIH Small Biz Hangout: Finding the Right Regulatory Consultant


-GOOD AFTERNOON, AND WELCOME TO
TODAY’S NHLBI SMALL BIZ HANGOUT. I’M CHRIS SASIELA,
A REGULATORY SPECIALIST AT THE NATIONAL HEART, LUNG,
AND BLOOD INSTITUTE. I’LL BE TODAY’S MODERATOR, BUT BEFORE WE GET INTO THE BULK
OF OUR PRESENTATION TODAY, WHAT I’D LIKE TO DO IS TELL YOU
A LITTLE BIT ABOUT MY OFFICE. THE OFFICE OF TRANSLATIONAL
ALLIANCES AND COORDINATION HAS A NUMBER OF RESOURCES THAT ARE OF POTENTIALLY
SIGNIFICANT VALUE TO YOU, OUR SMALL-BUSINESS
AWARDEES AND APPLICANTS, AS WELL AS OTHER AWARDEES INNOVATING IN THE HEART, LUNG,
BLOOD, OR SLEEP SPACE. AS ONE OF THOSE RESOURCES
MYSELF, I’M A REGULATORY SPECIALIST, AND I WORK WITH OUR APPLICANTS
AND AWARDEES TO HELP THEM UNDERSTAND A REGULATORY PATH
FORWARD FOR THEIR BIOMEDICAL INNOVATION
DEVELOPMENT. AND THAT TAKES MANY FORMS. IT CAN BE VIA E-MAIL OR
ONE-ON-ONE PHONE CONVERSATION. IT CAN BE THROUGH RESOURCES
SUCH AS TODAY’S HANGOUT. WE ARE HERE AS AN OFFICE
AND I, AS A RESOURCE, AS WELL AS SOME OF
MY OTHER COLLEAGUES — AN ENTREPRENEUR IN RESIDENCE,
INVESTOR IN RESIDENCE, A BUSINESS-DEVELOPMENT
SPECIALIST, AND AN INTELLECTUAL-PROPERTY
SPECIALIST. WE’RE AVAILABLE TO YOU
TO HELP YOU NAVIGATE SOME OF THOSE KEY
KNOWLEDGE AREAS THAT, AS NEW BIOMEDICAL
INNOVATORS, YOU MAY NOT HAVE THE RESOURCES
TO SUPPORT WITHIN YOUR BUDGETS AND YOUR COMPANIES. SO, THE SMALL BIZ HANGOUT SERIES
IS ONE OF THE WAYS THAT WE HAVE DEVELOPED
TO HELP COMMUNICATE TO YOU THOSE KNOWLEDGE-GAP AREAS
IN A REALLY BASIC AND JARGON-MINIMALIZED MANNER TO SUPPLEMENT
YOUR SCIENTIFIC TRAINING. TODAY WE’LL BE TALKING ABOUT
HOW TO IDENTIFY AND EXECUTE A REGULATORY STRATEGY TO SUPPORT
TECHNOLOGY DEVELOPMENT. SMALL BUSINESSES, SUCH AS THOSE
THAT GET FEDERAL FUNDING TO SUPPORT THEIR R&D EFFORTS, MAY GET REGULATORY ADVICE
FROM DIFFERENT SOURCES THROUGHOUT THEIR TECHNOLOGY
DEVELOPMENT CYCLE. THEY NEED TO UNDERSTAND
HOW TO IDENTIFY AND RATE POSSIBLE REGULATORY
COLLABORATORS, WHETHER THOSE ARE C.R.O.s,
CONSULTANTS, STRATEGIC PARTNERS, MEDICAL ADVISORY BOARD MEMBERS,
ET CETERA. MANY TIMES, SMALL BUSINESSES… WONDER ABOUT
THE APPROPRIATE STAGE IN THEIR TECHNOLOGY DEVELOPMENT
TO BEGIN LEARNING ABOUT AND DEVELOPING A REGULATORY PLAN AND THOSE REGULATORY REQUIRED
SYSTEMS AND DOCUMENTATION THAT WILL BE NEEDED FOR
LICENSING THEIR TECHNOLOGIES. IF YOUR EXIT STRATEGY IS
TO OUT-LICENSE YOUR TECHNOLOGY, WHY DO YOU REALLY HAVE TO
BE CONCERNED AT EARLY STAGES IN GENERATING
THAT TYPE OF DOCUMENTATION? ALSO, WHAT TYPES OF REGULATORY
KNOWLEDGE ARE APPROPRIATE TO BUILD IN HOUSE AND WHAT’S
BEST LEFT TO THE EXPERTS? THE TOPICS THAT WE COVER
IN OUR SMALL BIZ HANGOUTS ARE THOSE TOPICS
THAT ARE CRITICAL FOR BIOMEDICAL-INNOVATOR
SUCCESS. THEY COVER
INTELLECTUAL PROPERTY, COMMERCIALIZATION ACTIVITIES,
REGULATORY CONSIDERATIONS, AND THE LIST CONTINUES TO GROW. WE HOPE THAT BY HAVING
THIS ONGOING DIALOGUE WITH YOU, OUR COMMUNITY OF INNOVATORS, THAT YOU’LL GIVE US
THAT FEEDBACK, WHICH WE’LL BE ASKING FOR
AT THE END OF THIS PRESENTATION. WE HOPE THAT THE QUESTIONS
THAT YOU HAVE YOU’LL FEEL FREE TO COMMUNICATE
WITH US THROUGHOUT THIS HANGOUT. YOU CAN ASK QUESTIONS
IN A NUMBER OF WAYS. WE HAVE A TWITTER ACCOUNT,
WHICH IS… AND YOU CAN DIRECT-MESSAGE
US THERE. OR YOU CAN TWEET
USING THE #SBIRChat. OR IF YOU PREFER TO KEEP
YOUR QUESTIONS ANONYMOUS, YOU CAN SUBMIT THEM TO US
VIA E-MAIL AT… AND WE’LL PICK
THOSE QUESTIONS UP AND FEED THEM INTO
OUR DIALOGUE TODAY. THE PRIMARY GROUND RULE
FOR QUESTIONS IS THAT THEY SHOULDN’T APPLY
TO ONLY ONE SPECIFIC TECHNOLOGY. THESE HANGOUTS ARE INTENDED
TO BE A GENERAL CONVERSATION, AND IN ORDER TO PROVIDE THAT
TECHNOLOGY-SPECIFIC FEEDBACK YOU MIGHT BE LOOKING FOR, YOU PROBABLY NEED TO GIVE US
MORE INFORMATION ABOUT YOUR INNOVATION THAN IS
APPROPRIATE IN A PUBLIC SETTING. SO EVERY NOW AND THEN
THROUGHOUT OUR LIVE EVENT, WE’RE GONNA SHARE THIS SLIDE
SO THAT YOU CAN GET THE TWITTER HANDLE
AND THE HASHTAG RIGHT, AND THROUGHOUT THE PRESENTATION
YOU’LL SEE THE E-MAIL ADDRESS HOVERING ABOVE MY HEAD
AS I HAVE A HALO OF A GLOW OF E-MAIL SURROUNDING ME. TODAY I’M EXCITED TO BE JOINED
IN OUR HANGOUT BY TWO GRANTEES WHO HAVE RECEIVED SMALL BUSINESS
INNOVATIVE RESEARCH, OR SBIR, AWARDS FROM NHLBI. OUR FIRST PANELIST
IS MR. BRIAN LIPFORD, AN ENGINEER AND A FOUNDING
PARTNER OF KEY TECHNOLOGIES, COMMONLY KNOWN AS KEY TECH,
WHERE HE IS CURRENTLY THE VICE PRESIDENT
OF STRATEGIC DEVELOPMENT. IN THIS ROLE,
HE LEADS PROGRAMS TO FIND, DEVELOP, AND COMMERCIALIZE
NEW MEDICAL PRODUCTS. KEY TECH IS
AN ISO 13485-REGISTERED PRODUCT DEVELOPMENT COMPANY
WITH 35 ENGINEERS AND DESIGNERS, DEVELOPING AND SUPPORTING NEW
MEDICAL DEVICES AND INSTRUMENTS FOR THE PAST 16 YEARS. KEY TECH HAS ALSO DEVELOPED
AND SOLD SEVERAL OF ITS OWN MEDICAL
PRODUCTS TO STRATEGIC PARTNERS. OUR SECOND PANELIST
IS DR. JAKE REDER. JAKE CO-FOUNDED CELDARA MEDICAL
IN 2008 WITH DR. MICHAEL FANGER AS WAY TO BRIDGE ACADEMIC
INNOVATION AND THE MARKETPLACE. CELDARA HAS GROWN TO ENCOMPASS
FIVE FUNDED PROGRAMS AND A LARGE PIPELINE, INCLUDING A CLIA-CERTIFIED
DIAGNOSTIC SERVICE, WHICH IS ON THE MARKET
AND REIMBURSED, AND A CELLULAR THERAPY
THAT WAS SOLD IN JANUARY AND ALSO ENTERED THE CLINIC
THIS YEAR. JAKE HAD PREVIOUSLY FOUNDED
THE NEW VENTURES OFFICE AT DARTMOUTH MEDICAL SCHOOL, MANAGED STRATEGIC ALLIANCES
AND INTERNAL STARTUPS AT CABOT CORP AND INVESTED IN
EARLY-STAGE TECHNOLOGY COMPANIES AT PolyTechnos VENTURE-PARTNERS, A MUNICH-BASED
VENTURE-CAPITAL FIRM. JAKE EARNED A B.Sc.
WITH FIRST CLASS HONORS FROM THE UNIVERSITY OF WATERLOO
AND A PhD IN CHEMISTRY AS A BROWN-WETHERILL FELLOW
AT PURDUE UNIVERSITY AND LUDWIG MAXIMILIANS
UNIVERSITAT. I WELCOME YOU, OUR AUDIENCE
AND OUR PANELISTS, TO TODAY’S HANGOUT, AND LET’S
DIVE RIGHT IN TO SOME QUESTIONS. SO, I HAVE ASKED MY PANELISTS — I’VE PROVIDED THEM
WITH SOME QUESTIONS, AND WE’RE GONNA START OUT WITH SOME OF THESE PREPARED
QUESTIONS, BUT, AGAIN, I ENCOURAGE YOU, OUR AUDIENCE,
TO PLEASE POST YOUR QUESTIONS EITHER ON TWITTER
OR THROUGH E-MAIL, AND WE’LL MAKE SURE THAT
THOSE QUESTIONS GET ANSWERED THROUGHOUT OUR PRESENTATION
TODAY. SO, STARTING OUT, I’D LIKE TO
ASK FIRST BRIAN AND THEN JAKE, WHAT DID YOUR SMALL BUSINESS
LOOK FOR IN IDENTIFYING AND ENGAGING
REGULATORY EXPERTISE? SO, BRIAN? -SO, IN TERMS OF THE EXPERTISE,
WE, AS A COMPANY, PRIMARILY DEVELOP
DIAGNOSTIC DEVICES AND THERAPY-DELIVERY DEVICES, SO WE’RE LOOKING FOR EXPERTS
THAT DEAL WITH DEVICES. I GUESS THAT’S THE FIRST THING. A LOT OF TIMES, OUR DEVICES —
YOU KNOW, LATELY, WE’VE BEEN DOING A LOT
OF MOLECULAR DIAGNOSTIC DEVICES, SO, AGAIN, WE’RE GONNA LOOK
FOR PEOPLE THAT HAVE PREPARED REGULATORY SUBMITTALS
IN THOSE AREAS. SO WE’LL ASK OUR REGULATORY
PEOPLE IF THEY — THE EXPERIENCE THAT THEY HAVE ON
THOSE PARTICULAR KIND OF DEVICES AND PARTICULAR MARKET SEGMENTS
OF THE DEVICES. -YEAH, SO, WE DO MORE
THERAPEUTICS, AND SIMILARLY, THE ANSWER’S REALLY THE SAME
IN THAT DOMAIN EXPERTISE IS EVERYTHING. AND SO THE MOST RECENT EXAMPLE
IS THIS CELLULAR THERAPY, AND WHEN WE STARTED SPEAKING
TO PEOPLE ABOUT IT, THAT WAS THE FIRST QUESTION — “HOW MANY CELL THERAPIES
HAVE YOU DONE?” AND YOU GET AN ANSWER. AND THEN YOU ASK, “WELL, HOW
MANY CAR T-CELL THERAPIES HAVE YOU DONE
AND WHAT ARE THE CHALLENGES?” AND YOU GET INTO THE INTERVIEW
FOCUSED ON THE TECHNOLOGY BECAUSE, UNLESS IT’S A SMALL
MOLECULE TO A KNOWN TARGET, WHICH I THINK VERY FEW
SBIR AWARDEES ARE ACTUALLY WORKING ON, IT’S PROBABLY SOMETHING
RELATIVELY NEW, AND IT MIGHT BE SOMETHING
EVEN THE FDA HASN’T HAD AN ENORMOUS AMOUNT
OF PRECEDENT IN. THE FDA IS ALWAYS
VERY SOPHISTICATED AND HAS A LOT OF
GREAT SCIENTISTS, BUT THERE MAY NOT BE A LOT OF
PRECEDENT TO GUIDE THOSE OF US OUTSIDE OF THE AGENCY
AS TO WHAT TO DO, AND SO WE ALWAYS LOOK FOR
THAT DOMAIN EXPERTISE. AND THEN WE TRY TO CROSSCHECK
IT AGAINST A FEW DIFFERENT SOURCES
OF REGULATORY EXPERTISE. -SO, JAKE, MAYBE YOU COULD
GO INTO THAT IN A LITTLE BIT MORE DETAIL. HOW DO YOU, WHEN YOU’RE SORT
OF CROSSCHECKING THESE PEOPLE, DO YOU PERHAPS ASK
FOR REFERENCES OR DO YOU DO
YOUR OWN EVALUATION? HOW MANY REGULATORY OPINIONS
MIGHT YOU GET BEFORE YOU DECIDE TO ENGAGE
WITH ONE PARTICULAR CONSULTANT OR GROUP OR TO FOLLOW
A CERTAIN STRATEGY? -YEAH, SO WE ALWAYS, ALWAYS,
ALWAYS CHECK REFERENCES, AND WE DO EVERYTHING WE CAN
TO IDENTIFY REFERENCES THAT AREN’T NECESSARILY
SUGGESTED BY THE COMPANY. WE TRY TO FIND OTHERS
WHO HAVE WORKED WITH THEM THAT HAVE HAD
UNBIASED EXPERIENCE. BECAUSE, AS YOU KNOW,
ESPECIALLY WITH SMALLER FIRMS — AND THERE ARE SOME EXCELLENT
SMALL CONSULTANCIES OUT THERE, BUT IT CAN BE QUITE DIFFICULT
TO DETERMINE JUST HOW VALID A PARTICULAR REFERENCE IS.
SO WE CERTAINLY START THERE. WE ASK QUITE DETAILED QUESTIONS
AND TRY TO REALLY PROBE INTO THE TYPES OF WORK
AND THE WORKING STYLE OF THAT PARTICULAR
REGULATORY GROUP. WE DO DILIGENCE
ON THEIR BACKGROUNDS, IN TERMS OF THE PRINCIPLES
THAT WE’D BE WORKING WITH. AND THEN, OF COURSE,
THERE’S THE INTERVIEW, FOR LACK OF A BETTER WORD,
OR INITIAL DISCUSSIONS, TO DETERMINE JUST HOW
COMFORTABLE WE ARE WITH THEIR LEVEL
OF DOMAIN EXPERTISE AND HOW MUCH THEY CAN ACTUALLY
BRING TO THE TABLE. LIKE ANYTHING WHERE THE — I FIND IT FASCINATING
THAT PEOPLE WILL GO OUT AND THEY’LL GET THREE QUOTES
FOR A PIECE OF EQUIPMENT — WHICH, YOU KNOW,
IS IMPORTANT TO THE BUSINESS, BUT NOT STRATEGICALLY
CRITICAL — AND THEY WON’T DO THE SAME SORT
OF THING FOR INFORMATION OR ADVICE. AND SO WE CERTAINLY DO THAT. GETTING THAT ADVICE
IN THE REGULATORY SPACE CAN SET A WHOLE PROGRAM BACK,
AND IF YOU’RE A SMALL COMPANY, SETTING A WHOLE PROGRAM BACK
CAN BE THE END OF THE COMPANY. AND SO IT’S SOMETHING WE TAKE
AS EXTREMELY SERIOUS, AND WE DO AS MUCH GROUNDWORK
AS WE CAN IN ADVANCE OF ACTUALLY PULLING THE TRIGGER. -AND, BRIAN, IN TERMS
OF THE REGULATORY PEOPLE THAT YOU MAY HAVE
INTERACTED WITH, HOW IMPORTANT WAS IT TO YOU THAT
THEY HAD A GEOGRAPHIC PROXIMITY AND ABILITY TO MEET WITH YOU
OR YOUR ENGINEERS AND TO REALLY HELP GUIDE
THAT TECHNOLOGY DEVELOPMENT OR UNDERSTAND
THE DATA-DEVELOPMENT PLAN THAT YOU’RE PUTTING TOGETHER, VERSUS THE MATCH IN TERMS
OF BACKGROUND AND EXPERTISE IN A PARTICULAR MEDICAL FIELD,
FOR INSTANCE? -YEAH, CHRIS, SO THE BACKGROUND
AND THE EXPERTISE CERTAINLY TRUMPS LOCATION. I DON’T THINK — SO, WE HAVE A COUPLE OF PEOPLE
THAT WE DO WORK WITH, AND SO ONE OF THEM
IS IN MICHIGAN, ONE OF THEM IS ON
THE WEST COAST, ONE OF THEM IS DOWN SOUTH,
SOUTHWEST, AND IT REALLY IS — IT COMES DOWN TO THEIR EXPERTISE
IN A PARTICULAR AREA, AND THE LOCATION REALLY
IS NOT IMPORTANT TO US. -ARE THERE ANY PARTICULAR
QUESTIONS THAT YOU MIGHT ASK THAT ARE SORT OF COMMON
QUESTIONS YOU ALWAYS ASK FOR A REGULATORY GROUP THAT ARE SORT OF KEY TELLS
FOR YOU ABOUT THEIR APPROPRIATENESS TO PARTNER WITH YOU
AS A COMPANY? -SO, DESIGN CONTROLS
AND RISK MANAGEMENT IS REALLY KIND OF THE FOUNDATION
FOR MEDICAL-DEVICE DEVELOPMENT. SO WE’RE VERY SENSITIVE TO THE REGULATORY PERSON’S
BACKGROUND AND SENSITIVITY
TO DESIGN CONTROLS. SO A LOT OF TIMES WE WILL
ASK THEM SPECIFIC QUESTIONS ABOUT DESIGN CONTROLS TO MAKE
SURE THAT THEY UNDERSTAND, BASICALLY, THE VALUE
OF THE RISK-MANAGEMENT PLAN AND SOME OF THE DOCUMENTS,
THE BASELINE DOCUMENTS, THAT HAVE TO BE IN PLACE
TO SUPPORT YOUR SUBMISSIONS. SO WE’LL ASK QUESTIONS
LIKE THAT. -AND DO YOU LOOK FOR PEOPLE
TO HAVE A CERTAIN NUMBER OF REGULATORY SUBMISSIONS
ALREADY ACCOMPLISHED BEFORE YOU’RE WILLING
TO ENGAGE WITH THEM? OR DOES HAVING
A PROFESSIONAL CERTIFICATION, SOMETHING OF THAT NATURE,
ADD BENEFIT TO YOU? OR DO YOU SEE — I MEAN, WHERE DO YOUR PUT
YOUR QUALIFIERS IN TERMS OF ASSESSING
THIS PERSON, OUTSIDE OF THE KNOWLEDGE
OF REGULATIONS? LIKE, WHAT TO YOU TAKES
A “GOOD TO GREAT” REGULATORY-CONSULTANT APPROACH? -SO, YOU KNOW,
I’LL TAKE A SHOT AT THAT, AND MAYBE THEN JAKE CAN
CHIME IN. SO, WE HAVE A COUPLE OF THINGS
WE’VE DONE AND WE REGULARLY DO. SO FOR US,
ONE OF THE BIG FIRST STEPS IS THAT REGULATORY PATHWAY
ANALYSIS, WHERE YOU’RE LAYING OUT
YOUR PREDICATES AND YOU’RE LAYING OUT
THE EXTENT OF THE CLINICAL WORK, IF YOU’RE GONNA HAVE TO DO
CLINICAL WORK, HOW MUCH CLINICAL WORK. YOU’RE REALLY LAYING OUT
KIND OF LIKE THE ROADMAP THAT YOU’RE GONNA HAVE
TO FOLLOW. SO ONE OF THE THINGS THAT
WE’VE DONE AND I REGULARLY DO IS A WILL HIRE TWO — YOU KNOW,
I WILL VET THEM LIKE JAKE TALKS, AND YOU KNOW,
WE DO THE REFERENCE CHECKS, WE’LL HAVE SOME CONVERSATIONS, BUT I’VE HIRED
TWO REGULATORY EXPERTS, AND EACH DOESN’T KNOW
ABOUT THE OTHER. AND I’LL ASK THEM EACH
TO PREPARE A PATHWAY ANALYSIS, AND THEN I’LL COMPARE
THE DOCUMENTS TO MAKE SURE THAT — YOU KNOW, I’LL LOOK AT THE PREDICATES
THAT THEY’VE DEFINED AND WHAT KIND OF CLINICAL WORK,
AND BASICALLY, IT’S A LITTLE MORE EXPENSE, BUT IT’S SUCH A CRUCIAL DOCUMENT
TO GET RIGHT TO KNOW WHERE YOU’RE GOING. IT’S WELL WORTH
THE PRICE TO PAY. SO THAT’S ONE OF THE TRICKS
THAT WE WILL DO. -IT’S FUNNY
THAT YOU MENTION THAT, AND THAT’S SOMETHING WE’RE
STARTING TO DO NOW, AS WELL. SO, YOU KNOW, OPTIONALITY
IN ANY IMPORTANT CONTEXT IN YOUR BUSINESS
IS SO CRITICAL TO SUCCESS, AND IF YOU DON’T HAVE
A PLAN “B,” THEN YOU DON’T HAVE ANY CHOICE, AND YOU ARE LOCKED IN TO
A PARTICULAR SITUATION, WHICH IS NEVER COMFORTABLE. AND SO I THINK THAT’S
A BRILLIANT APPROACH TO IT, AND IT’S SOMETHING
WE HADN’T DONE A YEAR AGO, BUT WE’VE JUST STARTED TO DO. AND, YOU KNOW, SO FAR, SO GOOD. I SORT OF LOOK AT IT AS
THERE ARE STRATEGIC QUESTIONS LIKE ROAD-MAPPING, AND THEN THERE ARE BLOCKING
AND TACKLING SORT OF ACTIVITIES. AND AT LEAST IN OUR MINDS, ONCE YOU’RE WORKING WITH
THE CONSULTANTS, IT’S GOOD TO KNOW
WHERE THEIR STRENGTHS ARE. BECAUSE SOME ARE VERY GOOD
AT THE STRATEGIC AND CAN FALL DOWN
ON THE BLOCKING AND TACKLING AND VICE VERSA. AND SO HAVING TWO FIRMS THERE, YOU CAN GET A SENSE FOR WHERE
THEIR REAL STRENGTHS ARE. AND IN REALITY,
IT COSTS A LITTLE BIT MORE, BUT IT CERTAINLY DOESN’T COST
TWICE AS MUCH BECAUSE GENERALLY THEY’RE
BILLING ON A PER-HOUR BASIS OR EVEN ON
A PROJECT-LEVEL BASIS, THAT YOU’RE STILL GONNA HAVE
HALF THE SCOPE, ESSENTIALLY, AND JUST SPLIT THAT
BETWEEN TWO FIRMS. SO I REALLY LIKE THAT IDEA,
AND AS I SAY, WE’RE DOING THAT OURSELVES. -SO, JAKE, YOU AND I ACTUALLY
FIRST MET VIRTUALLY ABOUT, MAYBE, A YEAR AND A HALF,
TWO YEARS AGO, AND I KNOW THAT YOU’VE HAD
AT LEAST ONE PROJECT THAT GOT SORT OF A SIDEWAYS HIT. YOU HAD A REGULATORY PATH
DEFINED, AND THEN ALL OF A SUDDEN, A REQUIREMENT CAME UP
THAT YOU WEREN’T — YOU HADN’T ANTICIPATED
WOULD NECESSARILY ARISE. CAN YOU TALK A LITTLE BIT
ABOUT SOME OF THE RESOURCES THAT YOU POOLED TOGETHER
TO SORT OF ADDRESS THE UNANTICIPATED OBSTACLES
THAT MIGHT COME UP? -SURE. YOU KNOW, IT’S — THERE WEREN’T REALLY
ADDITIONAL RESOURCES NEEDED. IT WAS ANOTHER — SO THERE
ARE MANY REGULATORY BODIES. EVERYONE FOCUSES ON FDA, BUT THERE ARE LOTS OF OTHERS
OUT THERE, INCLUDING IRBs, AND OTHER GROUPS THAT CAN STOP
A TRIAL FROM MOVING FORWARD OR STOP A TRIAL
BEFORE IT STARTS. AND SO UNDERSTANDING
SEQUENCES — AND, YOU KNOW,
A LOT OF THESE GROUPS, THEY WANT TO BE THE LAST ONE
TO SEE SOMETHING. AND FIGURING ALL THAT OUT
AND THE LOGISTICS WITH YOUR CLINICAL CENTER, AS WELL,
WAS CHALLENGING FOR US. BUT IT WASN’T A MATTER OF
BRINGING NEW RESOURCES TO BEAR. IT WAS REALLY A MATTER OF
TAKING THE RESOURCES WE HAD — WE HAD ALL THE KNOWLEDGE
ON THE TEAM. IT WAS JUST A MATTER
OF PERSUADING THE PARTICULAR REGULATORY BODIES
THAT WHAT WE WERE DOING WAS IN FACT A REALLY GOOD THING
FOR PATIENTS. AND THAT TOOK SOME TIME. WE HAD ANTICIPATED A LOT
OF THINGS MOVING IN PARALLEL, AND IN REALITY, THEY ENDED UP
MOVING NOT ALWAYS IN SERIAL, BUT NOT NEARLY AS PARALLEL
AS WE’D HOPED. -SO THAT SORT OF FEEDS INTO
WHAT WE LEARNED WHEN WE WERE BACK IN ACADEMIA, THAT IF YOU PLAN AN EXPERIMENT
TO LAST FOR ONE MONTH, ANTICIPATE NOT GETTING RESULTS
FOR THREE MONTHS. -YEAH, YEAH. -SO, BRIAN, HAVE YOU HAD
ANY EXPERIENCES WHERE PERHAPS THERE’S BEEN
A CHANGE IN REGULATION OR A SIGNIFICANT DESIGN CHANGE
WHERE YOU’VE NEEDED TO MODIFY YOUR REGULATORY STRATEGY? AND HOW HAVE YOU WORKED
BOTH WITHIN YOUR COMPANY AND WITH ANY OUTSIDE REGULATORY
COUNCIL THAT YOU MIGHT HAVE TO ADDRESS THESE SORT OF
EMERGENT THINGS? YOU KNOW, SCIENCE CAN CHANGE, WHICH CHANGES EVERYTHING
AT TIMES. -OOH, THAT’S A GOOD QUESTION. I DON’T KNOW IF WE’D HAD ANY
SPECIFIC THINGS LIKE THAT. WE’VE HAD SOME CASES WHERE WE
WERE PURSUING ONE INDICATION FOR USE, AND WE WERE ON A 510(k)
KIND OF PATHWAY, AND THEN WE THOUGHT WE WERE
DOING A RELATIVELY SMALL CHANGE ON A DIFFERENT INDICATION,
AND WE WERE TOLD — BASICALLY, WE WALKED INTO A PMA, WHICH IS TYPICALLY SOMETHING WE’LL TRY TO AVOID
LIKE THE PLAGUE. SO WE HAD TO PIVOT BACK
TO OUR FIRST INDICATION, WHICH WE DEALT WITH, BUT IT
WAS ONE OF THOSE THINGS WHERE, IN HINDSIGHT, WHEN WE DECIDED TO
CHANGE THAT INDICATION FOR USE, WE SHOULD’VE A LITTLE MORE
SENSITIVE TO THE IMPLICATIONS OF THE REGULATORY PATH. -YEAH, DEFINITELY
YOUR LABEL CLAIM IS ALWAYS WHAT DEFINES YOUR REGULATORY
PATH, SO YOU NEED TO VERY AWARE
WHEN YOU CONSIDER MAKING ANY CHANGES TO THAT. SO, LET’S TALK A LITTLE BIT
LARGER THAN JUST THE U.S. IF I’M CURRENTLY WORKING
ON DEVELOPING MY TECHNOLOGY, WHATEVER MY TECHNOLOGY IS —
AND WE’LL START WITH BRIAN, SO I’M GONNA BE DEVELOPING
A MEDICAL DEVICE OR DIAGNOSTIC, AND I HAVE FUNDING THROUGH
THE SBIR PROGRAM, WHICH SUPPORTS U.S. DEVELOPMENT
OF THAT TECHNOLOGY. SHOULD I STILL BE CONCERNED
ABOUT MY REGULATORY COUNCIL, WHETHER IT’S INTERNAL
OR EXTERNAL, HAVING EXPERIENCE
OUTSIDE OF THE U.S.? OR SINCE MY FUNDING
IS ONLY GOING TO SUPPORT U.S. DEVELOPMENT, SHOULD I FOCUS ENTIRELY ON
THE U.S. AT THIS POINT? AND WOULD THAT ANSWER PERHAPS
CHANGE IF IT WERE A DEVICE VERSUS A DIAGNOSTIC? -I DON’T KNOW IF I CAN ADD
TOO MUCH TO THAT OTHER THAN, IN TERMS OF THE DEVELOPMENT
WORLD OF THE DEVICE THAT IS WHERE WE TYPICALLY LIVE, WE’LL HAVE A SET OF REQUIREMENTS
THAT WILL FIT THE U.S. SO WE’LL BE DESIGNING
TO 60601 OR 61010. THOSE ARE IEC STANDARDS. BUT WE’LL ALSO PUT IN THE MDD,
THE MEDICAL DEVICE DIRECTIVE, STANDARD REQUIREMENTS FOR
TAKING THE PRODUCT OVERSEAS. A LOT OF THE PRODUCTS
ARE INTERNATIONAL PRODUCTS. SO FROM A PRODUCT-REQUIREMENT
STANDPOINT, WHICH IS WHAT WE’RE SENSITIVE
TO, WE’LL FOCUS ON THAT. I GUESS I DON’T KNOW TOO MUCH
ABOUT THE REGULATORY ENVIRONMENT ON THOSE TOPICS, SO CAN’T
ADD MUCH MORE THAN THAT. -BUT IN TERMS
OF YOUR DEVELOPMENT PLAN, YOU DO INCORPORATE
THE REQUIREMENTS FOR BOTH MARKETS
OR FOR MULTIPLE MARKETS AS YOU’RE GOING THROUGH
YOUR DEVELOPMENT. AND, JAKE, FROM YOUR EXPERIENCE,
HAVE YOU — YOU KNOW, CLINICAL TRIALS,
THE LARGER ONES, NOT NECESSARILY ONES
THAT SMALL BUSINESSES RUN, BUT THEY DO TEND TO BE
INTERNATIONAL IN SCOPE THESE DAYS. ARE YOU LOOKING TO
E.U. DIRECTIVES, AS WELL AS U.S. DIRECTIVES, FOR CLINICAL-TRIAL MANAGEMENT
AND DESIGN? SOMETIMES THE REQUIREMENTS
CAN BE A BIT DIFFERENT BETWEEN ECONOMIC ZONES. -YEAH, YEAH. SO YOU KNOW, REGULATORY STRATEGY IS A SUBSET
OF THE OVERALL COMPANY STRATEGY, AND SO IT’S A QUESTION OF WHAT
IT IS THAT YOU REALLY WANT TO DO WITH THAT PARTICULAR — IN
OUR CASE, GENERALLY THERAPEUTIC. AND I GUESS I COULD IMAGINE
A SITUATION WHERE YOU COULD BE A COMPANY THAT WANTED TO MARCH
THROUGH ALL THE TRIALS AND GET TO MARKET
IN THE U.S. ONLY, BUT I REALLY HAVE TO THINK HARD
TO IMAGINE THAT. YOU KNOW, IT’S A GLOBAL BUSINESS
FOR THERAPEUTICS NOW, AND MOST BIOTECHS END UP GETTING
EITHER INTERNATIONAL INVESTORS OR MULTINATIONAL PARTNERS
AT SOME POINT. AND SO YOU REALLY HAVE TO, IF YOU’RE NOT AN EXPERT
IN THOSE AREAS — AND EVEN IF YOU DON’T PLAN ON
RUNNING TRIALS YOURSELF IN THE OTHER AREAS,
IT’S, I THINK, ONLY GOOD BUSINESS TO HAVE
SOMEONE WITH ENOUGH EXPERTISE TO LOOK OVER YOUR PLAN
AND TO SAY, “OH, WAIT —
HERE’S A FEW MODIFICATIONS THAT WILL AVOID A HASSLE
FOR A FUTURE PARTNER OR FOR YOU IN THE FUTURE IN ASIA
OR IN THE E.U. OR ELSEWHERE. -AND HOW HARD DO YOU LOOK
TO GET THAT KIND OF ADVICE? ONCE YOU’VE DONE
YOUR FIRST VETTING OF THE PRIMARY CONSULTANT
THAT YOU’RE GOING TO USE, DO YOU PERHAPS MAYBE LOOK
FOR SOMEONE WHO’S AN EXPERT IN INTERNATIONAL REGULATORY
AFFAIRS OR IN LATIN AMERICA OR WHEREVER YOUR NEXT MARKET
MIGHT BE TO FEED INTO THAT PLAN? OR DO YOU TRUST THAT
YOUR CONSULTANT AND SORT OF… DO YOU GIVE THEM
THE RESPONSIBILITY FOR SEEKING THOSE INPUTS
FROM THEIR COLLEAGUES? -YEAH, SO THE — I CAN’T THINK OF
A SINGLE REGULATORY CONSULTANCY THAT WE’VE SPOKEN WITH
THAT DOESN’T EITHER HAVE SIGNIFICANT OPERATIONS THAT
ARE WHOLLY OWNED OVERSEAS OR ELSE A VERY STRONG NETWORK
OF A SIMILAR SIZE, SIMILAR PHILOSOPHY CONSULTANCIES
OVERSEAS. AND SO I THINK THE DEMAND
FOR REGULATORY SERVICES HAS DEFINED WHAT
THE OFFERINGS ARE. AND SO ALMOST EVERYBODY
WE’VE DEALT WITH — EVERYONE I CAN THINK OF,
IN FACT — HAS THAT INTERNATIONAL
PERSPECTIVE. ‘CAUSE IT’S REALLY — THAT’S THE NATURE
OF THE THERAPEUTICS BUSINESS. -OKAY. I’D LIKE TO REMIND
OUR AUDIENCE THAT YOU CAN CERTAINLY SUBMIT
YOUR QUESTIONS THROUGH TWITTER OR BY E-MAIL. THE E-MAIL ADDRESS IS HOVERING
OVER MY HEAD AT THE MOMENT. AND I’D LIKE TO START BY — I NOTICE WE HAVE
ONE QUESTION ALREADY. I’D LIKE TO START WITH JAKE
FOR THIS ONE. “HOW IMPORTANT IS IT
FOR YOUR COMPANY TO UNDERSTAND THEIR REGULATORY PATH
ON THEIR OWN BEFORE GETTING THE CONSULTANT
OR VETTING THEIR HELP?” SO HOW MUCH PRE-KNOWLEDGE
DO YOU NEED ABOUT A REGULATORY STRATEGY
BEFORE YOU GO TALK TO SOMEONE? -YEAH,
THAT’S A FANTASTIC QUESTION. AND I THINK THIS IS TRUE FOR
ANY CONSULTING INTERACTION — IF YOU DON’T KNOW, YOU’RE GONNA
SPEND A LOT OF MONEY AND MAYBE END UP IN A BAD PLACE. AND SO I THINK CONSULTANTS
ARE EXACTLY THAT — THEY’RE VERY GOOD AT PROVIDING
ANSWERS TO QUESTIONS AND TO HELPING AID YOU, BUT THE
IDEA THAT YOU CAN WRITE A CHECK, NO MATTER HOW LARGE,
TO ANOTHER GROUP AND JUST SORT OF CLEAN
YOUR HANDS OF IT AND HOPE THAT THEY’LL GET THINGS
RIGHT IS A FALLACY. THAT JUST WON’T HAPPEN. AND SO YOU NEED TO
EDUCATE YOURSELF ENOUGH SO THAT YOU KNOW WHEN
THINGS SMELL BAD. THE STRATEGY THAT YOU HEARD
EARLIER FROM BRIAN ABOUT HAVING TWO CONSULTANCIES
IS ALSO A NICE THING. SO WHEN SOMETHING SOUNDS
A LITTLE STRANGE TO YOU, GIVEN THAT YOU ARE NOT AN EXPERT
IN REGULATORY AFFAIRS, BUT YOU’RE AT LEAST
A JOURNEYMAN, YOU CAN GO TO ANOTHER EXPERT
WHO’S ALREADY FAMILIAR WITH WHAT IT IS THAT YOU’RE
TRYING TO DO AND ENSURE THAT THEY AGREE WITH
THE ADVICE THAT YOU RECEIVE. SO THERE’S NO SORT OF EASY WAY. YOU HAVE TO GET UP THE LEARNING
CURVE AT LEAST PART OF THE WAY SO THAT YOU CAN INTERACT WITH
YOUR CONSULTANTS ON A MEANINGFUL LEVEL. -AND IF TODAY’S HANGOUT IS THE
FIRST ONE THAT YOU’RE ATTENDING AS ONE OF OUR AUDIENCE MEMBERS, I’D LIKE TO POINT OUT
THAT THIS IS A SERIES, AND THE VERY FIRST HANGOUT
THAT WAS DELIVERED WAS ON NAVIGATING
THE FDA WEBSITE. AND IN THAT HANGOUT,
I DESCRIBED HOW TO IDENTIFY SOME OF THE FREEDOM
OF INFORMATION ACT’S REDACTED, PUBLICLY AVAILABLE DOCUMENTATION
FOR 510(k) SUMMARIES AND APPROVAL DOCUMENTATION
FOR BOTH PMA DEVICES AND FOR NEW DRUGS AND BIOLOGICS. AND YOU CAN DO SOME RESEARCH BY
LOOKING AT SIMILAR TECHNOLOGIES, WHETHER THEY’RE DEVICES,
DIAGNOSTICS, BIOLOGICS, OR SMALL MOLECULES, AND SORT OF SEE HOW
THE AGENCY IS THINKING ABOUT THAT TYPE OF TECHNOLOGY, WHAT SORT OF BURDEN OF PROOF
HAS AN INNOVATOR NEEDED TO SHOW IN ORDER TO GET MARKET CLEARANCE
OR APPROVAL FOR THAT TYPE OF TECHNOLOGY. SO THE RESOURCES DO EXIST FOR
YOU TO START UNDERSTANDING THIS, IN ADDITION TO THE REQUIRING OR REQUESTING MULTIPLE INPUTS
FROM MULTIPLE CONSULTANTS OR POTENTIAL ADVISORS. BRIAN, IN ADDITION
TO YOUR EARLIER “ASK FOR MULTIPLE OPINIONS” AND
THEN JAKE’S COMMENTS JUST NOW, DO YOU HAVE
ANY OTHER PERSPECTIVES ON HOW TO MAKE SURE
YOU’RE GETTING — MAYBE TWO PEOPLE BOTH CAME UP
WITH THE SAME WRONG PLAN. [ LAUGHS ] YOU KNOW, HOW MUCH KNOWLEDGE
DO YOU DEVELOP IN-HOUSE BEFORE ASKING SOMEONE TO PROVIDE
THE MORE IN-DEPTH GUIDANCE? -YEAH, SO JAKE IS, I THINK,
SPOT-ON, AND I DON’T HAVE A LOT MORE
TO ADD IN THAT YOU REALLY — IT HAS TO COME FROM, LIKE,
I THINK, THE C-LEVEL OF THE COMPANY,
FROM MANAGEMENT OF YOUR COMPANY, TO REALLY GET YOUR ARMS AROUND
THE REGULATORY MATERIAL. ‘CAUSE YOU REALLY CAN
GET YOURSELF IN TROUBLE IF YOU LET SOMEONE ELSE —
IF YOU JUST TRY TO PUSH IT AWAY AND KEEP IT AT ARM’S LENGTH, IT’S JUST NOT GONNA
WORK FOR YOU. ON THE COMMENT THAT YOU JUST
UTTERED ABOUT THE FDA’s WEBSITE, THAT IS REALLY A GREAT WEBSITE THAT PEOPLE SHOULD
TAKE ADVANTAGE OF. YOU REALLY SHOULD GO
AND TRY TO FIND YOUR PREDICATES AND READ UP ON YOUR PREDICATES. THERE’S JUST A WEALTH
OF INFORMATION THERE ON THE FDA’s WEBSITE, AND IT’S
GREAT SEARCHABLE MATERIAL. ONE OF THE THINGS THAT WE DID
THAT I THINK — YOU KNOW, IT’S ANOTHER LITTLE TRICK, BUT I THINK IT CAN ADD VALUE
TO PEOPLE, IS WHEN YOU FIND
SOME OF YOUR PREDICATES, YOU CAN ACTUALLY DO
A LITTLE RESEARCH. YOU CAN DO A LITTLE DIGGING
ON THOSE PREDICATES. ON ONE OF THEM — I MEAN, THESE ARE THE PEOPLE
THAT ARE INTIMATELY FAMILIAR WITH WRITING THE REGULATORY
SUBMITTALS ABOUT OUR PRODUCT. IT’S VERY SIMILAR TO YOURS.
-MM-HMM. -WE DID SOME RESEARCH,
AND WE FOUND THE AUTHOR ON ONE OF THE PAPERS —
ON ONE OF THE SUBMISSIONS THAT WAS A PRODUCT VERY SIMILAR
TO OURS, AND WE REACHED OUT
TO THAT PERSON AND HIRED THEM
FOR OUR SUBMITTAL. SO IT’S KIND OF LIKE GOING
AND TARGETING AND FINDING YOUR OWN EXPERT
THAT YOU KNOW IS AN EXPERT ON THAT PRODUCT. -EXCELLENT. SO, AS A SMALL BUSINESS, AND PARTICULARLY AS
A MEDICAL-DEVICE BUSINESS, BRIAN, YOU KNOW, WHAT WERE
SORT OF THE FIRST THINGS THAT YOU NEEDED TO BE AWARE OF
AND BUILD IN TO YOUR COMPANY TO ENSURE THAT YOUR PRODUCTS
WOULD BE ACCEPTABLE TO FDA AND OTHER REGULATING BODIES? -SO IT REALLY DOES START
WITH THE VERY BORING TOPIC OF DESIGN CONTROLS, I THINK. A LOT OF PEOPLE,
ESPECIALLY STARTUPS, THEY REALLY DON’T KNOW WHAT
THEY MIGHT BE GETTING INTO. THEY’RE INCLINED TO RUN OFF
AND BUILD A PROTOTYPE AS QUICKLY AS THEY CAN
AND START TO DO PRECLINICAL OR MAYBE BUILD THAT — YOU KNOW,
TRY TO ANSWER SOME QUESTIONS. AND THAT’S GREAT.
THAT’S THAT R&D STAGE. IT’S REALLY FUN. BUT AS IT STARTS TO BECOME REAL
AND YOU START TO KNOW WHAT THE ARCHITECT IS
OF YOUR DEVICE AND YOU KNOW IT’S SENSITIVITIES
AND YOU’RE GETTING TO THE POINT WHERE YOU’RE READY TO MAKE, I’LL
SAY, A MORE COMPLETE DEVICE, THEN YOU REALLY GOT TO START
THINKING ABOUT DESIGN CONTROLS AND HOW THE DESIGN CONTROLS
ARE GONNA BUILD IN AND TIE IN TO MANUFACTURING AND TIE IN TO
THE CLINICAL TESTING THAT YOU’RE GONNA HAVE TO DO WITH PRODUCTION-QUALITY
PRODUCTS. AND SO YOU ALMOST — THE DESIGN CONTROLS
DON’T COME INTO THE PICTURE RIGHT OFF THE BAT, BUT YOU
REALLY DO NEED TO UNDERSTAND HOW THE WHOLE PROCESS
WORKS TOGETHER BEFORE YOU START RUNNING DOWN
THE ROAD. -SO, I’M AN ACADEMIC ENGINEER, AND I HAVE THIS GREAT IDEA,
AND I START DEVELOPING IT. I’M IN THAT EXCITING R&D STAGE. I DON’T EVEN KNOW WHAT
DESIGN CONTROLS ARE. DO I GOT TO TAKE THE CLASS
IN IT? DO I READ GUIDANCE DOCUMENTS? HOW DO I START TO DEVELOP
THAT KNOWLEDGE? WHERE CAN I FIND THE RESOURCES I
NEED TO TELL ME HOW TO DO THAT? -SO, I KNOW THAT THERE
ARE LOTS OF WORKSHOPS AND CONFERENCES THAT COVER
THINGS LIKE THIS. I MEAN, AT A VERY FUNDAMENTAL —
I’M SURE YOU KNOW, BUT IT’S JUST ALL THE INPUTS
AND THE OUTPUTS THAT YOU’RE DOCUMENTING. AND SO I THINK THE FDA HAS SOME
REALLY GOOD DOCUMENTS ON IT. I REMEMBER SEEING SOME WEBINARS
THAT THEY HAVE AVAILABLE. SO ALL OF THESE THINGS
YOUR AUDIENCE HERE SHOULD TAKE ADVANTAGE OF. AND SOME OF THESE DOCUMENTS,
AGAIN, SOME OF THEM ARE LONG
AND TEDIOUS, BUT THERE ARE SOME VERY GOOD
SHORT DOCUMENTS YOU CAN FIND THAT’LL GIVE YOU THE OVERVIEW
OF WHAT DESIGN CONTROLS ARE ALL ABOUT. -MM-HMM. AND I’M SURE IF SOMEONE WERE
TO DO A GOOGLE SEARCH, THERE ARE TONS OF WHITE PAPERS THAT TAKE THOSE RATHER LONG FDA
DOCUMENTS AND DISTILL THEM. -YES.
-[ COUGHS ] EXCUSE ME. SO, JAKE, SIMILAR QUESTION
FOR YOU — AS YOU’RE STARTING TO DEVELOP A NEW THERAPEUTIC TYPE
OF TECHNOLOGY, DO YOU START BY JUST DOING
THE BASIC RESEARCH AND NOT WORRYING ABOUT
EVENTUALLY HAVING TO DO THIS THING CALLED GMPs AND GLPs? OR DO YOU START OUT WITH
A MORE STRUCTURED APPROACH? WHEN DO YOU TRANSITION THERE? AND IS THAT SOMETHING THAT
YOU BRING IN EXPERTS TO DO, OR IS THAT SOMETHING YOU GROW
ORGANICALLY WITHIN YOUR COMPANY? HOW DOES THAT FEED INTO
THE REGULATORY DEVELOPMENT OF YOUR NOVEL TECHNOLOGIES? -SO IT’S SOMETHING THAT’S, I THINK, HIGHLY, HIGHLY
REGIMENTED AND WELL-DEFINED. IT’S LIKE BIG PHARMA. AND SO THERE ARE MANY STEPS, AND THERE’S SORT OF
A STAGE-GATE-LIKE PROCESS. YOU NEED TO PASS
CERTAIN REQUIREMENTS FOR A CERTAIN MOLECULE
TO GET TO THE NEXT STAGE, AND THEN THE AMOUNT
OF FUNDING GOES UP, AND THE AMOUNT OF ATTENTION
AND FDEs GOES UP. IN A SMALL BUSINESS, I DON’T THINK THAT’S
VERY PRACTICAL, HONESTLY. AND IT’S MORE OF A CONTINUUM WHERE YOU’RE RATCHETING UP
THE RIGOR AS YOU MOVE ALONG. CERTAINLY, THERE ARE SOME
STEP FUNCTIONS IN THERE WHEN YOU CHOOSE
YOUR LEAD COMPOUND AND YOU KNOW THAT THIS IS
WHAT IS ACTUALLY GOING TO BE ADMINISTERED
TO A HUMAN. THAT’S A HUGE STEP FORWARD,
AND THEN FROM THAT POINT ON, YOU WANT TO REALLY INCREASE
THE RIGOR AND DOCUMENTATION AND DO, IF IT’S NOT GLP, THEN THINGS THAT ARE GLP-LIKE
WITHIN THE GUIDANCES THAT, AGAIN, FDA WILL PROVIDE
OR HAS PROVIDED. AND SO THERE ARE CERTAIN
REALLY KEY POINTS IN THE DEVELOPMENT CYCLE,
BUT THE REALITY OF IT IS, FOR MOST OF THE SBIR COMPANIES
OUT THERE, YOU’RE PROBABLY
AT THAT INTERFACE BETWEEN RESEARCH
AND DEVELOPMENT. A PROFESSOR OR SOMEONE INSIDE
YOUR LABS HAS INVENTED SOMETHING OR DISCOVERED SOMETHING
REALLY EXCITING, AND THERE’S STILL A LITTLE MORE
RESEARCH LEFT TO DO TO DETERMINE IS THIS
REALLY REAL, IS THE EFFICACY THAT WE SEE
IN THIS ONE MODEL ABLE TO BE REPRODUCED
IN ANOTHER MODEL, ET CETERA? SO THERE’S STILL MORE RESEARCH
QUESTIONS TO BE ANSWERED, BUT ONCE YOU’VE REALLY LOCKED
DOWN AND YOU SAY, “THIS IS WHAT THE PRODUCT’S
GOING TO LOOK LIKE — THIS IS HOW WE’RE GOING TO TREAT
PATIENTS,” THEN AT THAT POINT, YOU REALLY NEED TO INCREASE
THE AMOUNT OF EFFORT AND TIME AND SOPHISTICATION AROUND
HOW YOU’RE DOING YOUR PRECLINICAL TESTING. ‘CAUSE, OTHERWISE,
YOU’LL BE DOING IT ALL AGAIN, AND THAT WOULD BE
VERY FRUSTRATING, I’M SURE. -AND SO THAT STAGE GATE WHERE YOU’VE LOCKED DOWN
YOUR LEAD MOLECULE AND MAYBE YOU’VE DONE
THE FORMULATION WORK AROUND IT TO GET APPROPRIATE SOLUBILITY
AND, YOU KNOW, HALF-LIFE AND THINGS
OF THAT NATURE — AT THAT POINT, IS THAT WHERE YOU WOULD PERHAPS
ASK FDA FOR PRE-I.N.D. MEETING, OR DO YOU WAIT UNTIL YOU HAVE
A LARGER BODY OF DATA? WHERE DO YOU CHOOSE
THAT INTERACTION POINT? DO YOU GO BEFORE YOU DO YOUR
PIVOTAL PRECLINICAL STUDIES, OR DO YOU GO RIGHT BEFORE
YOU SUBMIT YOUR I.N.D.? -SO I HAVE MY OWN OPINION
ON THIS. I DON’T KNOW HOW GENERALIZABLE
THIS IS, BUT FDA HAS NOW THEIR PRE-I.N.D. AND THEY HAVE
THEIR PRE-PRE-I.N.D. AND SO IF THIS IS SOMETHING — AND WE SEE A LOT OF DIFFERENT
TYPES OF THERAPEUTICS. AND IF THIS IS SOMETHING
WHERE THERE ISN’T MUCH PRECEDENT OUT THERE IN THE WORLD
ON THE REGULATORY SIDE AND MAYBE SOMETHING LIKE THIS
HASN’T ENTERED THE CLINIC YET, THEN WE’LL HAVE
A PRE-PRE-I.N.D. MEETING AS SOON AS THERE’S ENOUGH
TO TALK ABOUT. AS SOON AS THERE’S
A SOLID SET OF DATA, MAYBE SOME SAFETY DATA, AS WELL, WE’LL REQUEST THAT MEETING
WITH THE FDA SO THAT WE CAN BASICALLY PLOT OUT THE REST
OF THE PRECLINICAL DEVELOPMENT AND SAY, “IF WE BRING YOU ALL
THESE DATA, IS THAT SUFFICIENT?” THEN LATER ON
IN THE DEVELOPMENT, ONCE YOU’VE GOT
A CLINICAL PLAN TOGETHER AND THEY CAN OPINE ON THAT — THAT’S SOMETHING THAT
OBVIOUSLY TAKES A LOT OF WORK AND THOUGHT — THEN WE WOULD
REQUEST A PRE-I.N.D. MEETING. WE’VE NEVER DONE IT
RIGHT BEFORE AN I.N.D., BECAUSE THE I.N.D. IS THEN
ANOTHER OPPORTUNITY TO INTERACT SUBSTANTIVELY
WITH THE AGENCY. AND SO WHY PUT THEM
ON TOP OF EACH OTHER? WHY NOT HAVE A LITTLE SPACE
BETWEEN SO THAT WE CAN RESPOND
TO THEIR PREVIOUS COMMENTS? BUT AS I SAY,
THAT’S HOW WE VIEW THINGS, AND I’M SURE THERE ARE OTHER
EQUALLY LEGITIMATE PERSPECTIVES. -AND, BRIAN, IN TERMS OF FOR
MEDICAL DEVICES WORKING WITH CDRH, I KNOW THAT CDRH IN THE PAST
MAYBE THREE TO FIVE YEARS HAS REALLY TRIED TO
TURN THEMSELVES AROUND FROM THE 800-POUND GORILLA
IN THE ROOM TO BEING MUCH MORE INTERACTIVE
AND COMMUNICATIVE, TRANSPARENT WITH INDUSTRY. HAVE YOU FOUND THAT YOU’RE MORE
ABLE TO GET FEEDBACK ON YOUR — IF YOU PUT IN REQUESTS
FOR PRE-MARKET SUBMISSIONS, YOU KNOW, PRE-SUBMISSION
GUIDANCE-TYPES OF INTERACTIONS, ARE YOU ABLE TO GET THAT KIND
OF FEEDBACK IN A TIMEFRAME THAT REALLY HELPS PROGRESS
YOUR TECHNOLOGY DEVELOPMENT APPROPRIATELY AND EFFICIENTLY? -SO I DON’T WANT TO SPEAK
AS SOMEONE WHO HAS A TON OF EXPERIENCE, BUT
FROM THE HANDFUL OF DATA POINTS, IT DOES SEEM MUCH EASIER
TO BASICALLY EVEN PICK UP THE PHONE
AND HAVE A CONVERSATION, AN INFORMAL CONVERSATION,
WHERE YOU CAN SAY, “THESE ARE OUR THOUGHTS,
THESE ARE WHERE WE WERE GOING, YOU KNOW, WHAT DO YOU THINK?”
KIND OF THING AND GET SOME REALLY GOOD
FEEDBACK THAT MAYBE WAS HARDER TO GET
BEFORE. SO IN THAT REGARD, I THINK,
YEAH — I THINK THEY HAVE… YOU KNOW,
IT’S EASIER TO REACH THEM. TO ME,
IT’S EASIER TO TALK TO THEM. BUT YOU KNOW, THAT’S
A HALF-DOZEN DATA POINTS. -[ CHUCKLES ]
-CAN I ADD TO THAT? -ABSOLUTELY. -SO, OUR EXPERIENCE WITH
THE FDA — YOU HEAR STORIES, AND EVERYBODY TALKS ABOUT
SOME HORROR STORY WITH THE FDA. WE’VE HAD NOTHING BUT WONDERFUL
EXPERIENCES WITH THE FDA, AND I WOULD SAY THAT
IF I WAS TALKING OVER A BEER ONE-ON-ONE, AS WELL. BUT WHAT I HAVE NOTICED
OVER THAT SAME TIME PERIOD, THE SORT OF FIVE TO SEVEN YEARS,
IS THAT, I THINK, BECAUSE OF THE INADEQUATE
RESOURCES THAT ARE PROVIDED TO THE AGENCY, I THINK THEY WERE
GETTING OVERWHELMED WITH THOSE TYPES OF CALLS. AND SO FIVE, SEVEN YEARS AGO, WE COULD PICK UP THE PHONE
AND FIND OUR WAY TO SOMEBODY AT FDA THAT WOULD SIT
FOR A HALF-HOUR AND TALK ABOUT OUR THOUGHTS
ON THE REGULATORY PATH, WHEREAS NOW WE’VE STOPPED
EVEN TRYING TO DO THAT BECAUSE OF A HALF-DOZEN ATTEMPTS
THAT JUST GOT SENT BACK AND SAID, “HERE, GO THROUGH
THE FORMAL CHANNELS.” AND SO, YOU KNOW,
IT’S UNDERSTANDABLE, BUT, BOY, WAS IT NICE TO BE ABLE
TO JUST PICK UP THE PHONE AND TALK TO SOMEBODY
AT THE AGENCY. YOU KNOW, WHENEVER I HAVE
THE OPPORTUNITY TO TALK TO MY REPRESENTATIVES
IN GOVERNMENT, I TRY TO REMIND THEM THAT
MORE RESOURCES TO CRITICALLY IMPORTANT AGENCIES
LIKE THAT ARE A BENEFIT TO ALL OF US,
CERTAINLY, I THINK, IN THE SMALL-BUSINESS COMMUNITY. -ABSOLUTELY. SO, SOME OF THE MORE FORMAL
PROCESSES FOR INTERACTING WITH THE FDA HAVE COME OUT
AS GUIDANCE DOCUMENTS FOR DRUGS AND BIOLOGICS. THERE’S A RECENTLY RE-RELEASED
FORMAL-MEETINGS GUIDANCE THAT KIND OF SPECIFIES
IN A LITTLE MORE CLARITY, AND WITH SOMEWHAT FIRMER
GUIDELINES, WHAT SHOULD BE INCLUDED
IN A MEETING PACKET AND FOR THE PRE-SUBMISSION
PROGRAM FOR DEVICES AND DIAGNOSTICS. THAT WAS RELEASED, I BELIEVE,
ABOUT A YEAR AGO, MAYBE 18 MONTHS AGO, AND REALLY PROVIDED
A LOT OF CLARITY WITH THE Q-SUBMISSION PROGRAM
NOW, WHERE YOU CAN JUST E-MAIL
A QUESTION, AND THEY’LL TURN AN ANSWER
AROUND. GENERALLY, YOU’LL GET A RESPONSE
WITHIN 24 TO 48 HOURS. YOU’LL GET AN ANSWER
WITHIN A WEEK. AND I THINK THAT THAT’S REALLY
BEEN A BOON TO A LOT OF SMALL BUSINESSES
WHO DIDN’T REALIZE HOW ACCESSIBLE THE AGENCY
REALLY COULD BE. YOU KNOW, A LOT OF PEOPLE
GET VERY INTIMIDATED BY INTERACTING WITH
THE GOVERNMENT. I’M HERE TO SAY WE’RE FRIENDLY. WE’RE THE GOVERNMENT —
WE’RE HERE TO HELP, RIGHT? THAT’S WHAT YOU ALWAYS HEAR,
AND THEN THERE YOU GO — WE THROW A BIG WRENCH
INTO THE WORKS. BUT, YOU KNOW,
HOW DID EACH OF YOU — AND WE CAN START WITH
JAKE PERHAPS. HOW DID YOU OVERCOME
YOUR FIRST INITIAL BARRIER OF, YOU KNOW, PERHAPS BEING
INTIMIDATED AT THE THOUGHT OF INTERACTING WITH THE FDA? AND DO YOU FIND THAT IT’S MORE
PRODUCTIVE, LESS PRODUCTIVE, EQUALLY PRODUCTIVE TO HAVE
FACE-TO-FACE INTERACTIONS VERSUS TELECONFERENCES OR
BY COMMUNICATING ELECTRONICALLY THROUGH E-MAIL
OR JUST SUBMISSIONS? -YEAH, SO, WE HAVE NEVER HAD A FACE-TO-FACE TO MY RECOLLECTION. WE’VE ALWAYS DONE THINGS
BY PHONE. OBVIOUSLY, YOU KNOW, LARGE DATA FILES AND THINGS
NEED TO BE SENT BY E-MAIL. BUT THE PHONE’S BEEN GREAT. AND I THINK YOU GET
A LOT MORE NUANCE AND A LOT MORE QUICKER
TURNAROUND WHEN YOU’RE ACTUALLY SPEAKING
TO SOMEONE ON THE PHONE. THAT’S NOT LIMITED TO FDA,
OF COURSE. I CAN’T SAY THAT — I THINK
ANYBODY WHO’S WATCHING THIS IS PROBABLY AN ENTREPRENEUR, AND SO THE FEAR AND TREPIDATION
ASSOCIATED WITH PICKING UP THE PHONE
AND CALLING FDA, I THINK, IS MINISCULE COMPARED TO LEAVING
YOUR WELL-PAYING, SECURE JOB AND GOING OFF ON YOUR OWN
AND STARTING — -[ LAUGHS ] -I DON’T THINK THAT SHOULD BE
A PROBLEM FOR ANYONE. IF IT WERE SO, I CAN’T REMEMBER
A CONFERENCE THAT DIDN’T HAVE SOMEONE FROM FDA THERE —
THEY’RE REAL HUMAN BEINGS. I HAVE FRIENDS THAT WORK
AT THE FDA. AND YOU KNOW, IT SORT OF
TAKES IT AWAY FROM THIS IDEA THAT IT’S
A BIG GREEK-LOOKING BUILDING, AND IT’S ACTUALLY JUST
A GROUP OF SCIENTISTS THAT ARE CONCERNED
FOR OUR SAFETY. AND SO WE NEVER HAD THAT,
AND AS I SAID, WE USED TO JUST PICK UP
THE PHONE AND ASK QUESTIONS, AND THAT WAS VERY NICE. AND ALL OF THE FORMAL MEETINGS
THAT WE’VE HAD HAVE BEEN EXTREMELY EFFICIENT,
AS WELL. TO HAVE — IF YOU THINK
ABOUT THE I.N.D. PROCESS, THE AGENCY HAS 30 DAYS
TO RESPOND, AND YOU KNOW, WE SENT THEM 40 POUNDS OF PAPER. -IN TRIPLICATE.
-NO, THAT’S ONE COPY. THAT’S ONE COPY FOR YOU.
-RIGHT. LIKE I SAID,
40 POUNDS IN TRIPLICATE. -EXACTLY, YEAH. SO IT’S AN ENORMOUS AMOUNT
OF INFORMATION, AND IF YOU EXPECT THAT YOU COULD
SEND THAT MUCH INFORMATION TO A CONSULTANCY AND,
IN A MONTH, HAVE THEM UP TO SPEED ON WHAT
YOU’RE DOING, THERE’S NO CHANCE. BUT SOMEHOW FDA
NOT ONLY CAN DO IT, AND THEY ACTUALLY GET BACK
TO YOU AND ASK VERY, VERY GOOD
QUESTIONS, GIVE GUIDANCE, AND THEN AT THE END, THEY MAKE A DECISION TO EITHER
ALLOW YOU TO PROCEED OR NOT. SO THOSE INTERACTIONS HAVE BEEN
VERY POSITIVE FOR US, AND IF THERE IS ANYONE
THAT HAS ANY FEAR ABOUT IT, THEY CAN JUST LEAVE IT BEHIND.
THERE’S NO REASON FOR IT. -AND, BRIAN, HAVE YOU HAD
THE OPPORTUNITY TO HAVE FACE-TO-FACE MEETINGS
WITH THE FDA? AND IF SO, HAVE YOU NOTICED
A DIFFERENCE BETWEEN A FACE-TO-FACE VERSUS
A TELECONFERENCE OR ELECTRONIC INTERACTION? -SO I THINK MAYBE WE’VE HAD TWO. SO BY FAR —
IT’S LIKE JAKE SAID. BY FAR,
OUR PRIMARY COMMUNICATIONS IS VIA PHONE AND SUBMISSIONS. SO, FOR US, BASICALLY,
IF WE WANT TO DO HUMAN TRIALS, IT’S AN I.D.E. AND WE’LL PREPARE THE SAME KIND
OF MASSIVE AMOUNT OF INFORMATION AND SEND IT OFF, AND THERE’LL BE
QUESTIONS, BUT YOU KNOW, AGAIN, THE PROCESS WORKS VERY WELL. AND I GUESS,
FOR THE ENTREPRENEURS THAT ARE JUST GETTING INTO THIS, THE ONE SUGGESTION I WOULD
HAVE THAT, I THINK, WE’VE PROBABLY ALL MADE
THAT MISTAKE IS YOU REALLY WANT TO DO YOUR BEST,
DO SOME HOMEWORK, AND DEVELOP A PLAN
AND THEN PRESENT THE PLAN WITH SOUND REASONING, AS TO THIS
IS WHAT YOU WANT TO DO AND WHY, VERSUS GOING TO THE FDA
AND SAYING, “I HAVE NO CLUE
WHAT I WANT TO DO. WHY DON’T YOU TELL ME?” ‘CAUSE YOU’RE JUST GONNA OPEN UP
A DOOR YOU DON’T WANT TO OPEN. SO, OTHER THAN THAT, NO,
THEY’RE — AGAIN, IT’S LIKE JAKE SAID. THEY’RE JUST REALLY GOOD PEOPLE,
AND THEY DO WANT TO HELP. AND SO…IT’S ALL GOOD. -OKAY, I’D LIKE TO REMIND OUR
AUDIENCE THAT YOU CAN CONTINUE TO SUBMIT YOUR QUESTIONS
BY E-MAIL OR BY POSTING A TWEET WITH THE #SBIRChat. AND I WOULD LIKE TO NOW BRING UP
ONE OF THE QUESTIONS THAT WE HAVE RECEIVED. AND THE QUESTION IS —
IT’S A TWO-PART QUESTION. “CAN MY REGULATORY CONSULTANT
COMMUNICATE WITH THE FDA ON MY BEHALF? AND DO I NEED TO BE INVOLVED
IN CALLS AND MEETINGS?” SO, BRIAN, PERHAPS YOU
COULD START WITH THIS ONE. -SO, YES, YOU DO WANT TO
BE INVOLVED IN THE MEETINGS. YOU DON’T WANT TO LET
YOUR REGULATORY CONSULTANT BE THERE ON THEIR OWN. AND I DON’T THINK
WE’VE EVER DONE THAT. I HAVE HAD THE REGULATORY
CONSULTANT AT THE TABLE WITH ME, BUT, YES, YOU WANT TO BE ON
ALL THOSE COMMUNICATIONS I GUESS IS THE SHORT ANSWER. -AND HAS
THE REGULATORY CONSULTANT ACTUALLY LED THE INTERACTIONS, OR HAS THE C-TEAM FROM
THE COMPANY LED IT WITH JUST SORT OF PREPPING
AND BRIEFING FROM THE CONSULTANT IN ADVANCE?
-IT’S THE LATTER, GENERALLY. I MEAN, THE CONSULTANT
WILL WORK WITH US TO PREPARE THE MATERIAL. BUT, ALSO,
WHEN WE’RE ON THE PHONE, IT’S KIND OF HARD TO TELL
SOMETIMES EXACTLY, BUT PRIMARILY THE MANAGEMENT
TEAM IS LEADING THE DISCUSSION. -OKAY. AND JAKE? SAME QUESTIONS? -SAME ANSWER. YOU KNOW, IT GETS BACK TO THAT
POINT I MADE AT THE BEGINNING, WHICH IS CONSULTANTS CONSULT
AND THEY PROVIDE ADVICE, BUT THIS ISYOURPRODUCT. AND SO YOU’RE GONNA LIVE WITH IT
FOR THE NEXT, HOPEFULLY, DECADE OR MORE. AND WE’VE NEVER — YOU KNOW, WE TRUST THE PEOPLE
WE WORK WITH, BUT WE’VE NEVER ENTRUSTED
THAT LEVEL OF RESPONSIBILITY TO SOMEBODY OUTSIDE
OF OUR COMPANY. -OKAY. WELL, THANK YOU. I’M GONNA ASK ONE LAST TIME,
IF THERE ARE QUESTIONS, IF YOU COULD PLEASE
POST THEM QUICKLY BECAUSE WE ARE RUNNING LOW
ON TIME. AND I’M NOT SEEING ANY MORE
QUESTIONS COMING UP QUITE YET. SO I THINK, AT THIS POINT, WHAT I’D LIKE TO DO IS
I’D LIKE TO SAY THANK YOU TO BOTH OUR PANELISTS
AND OUR AUDIENCE FOR YOUR VERY ACTIVE
PARTICIPATION IN TODAY’S HANGOUT. WE LOVE HEARING FROM YOU
AND HEARING WHAT WORKS. WE DO RUN THESE HANGOUTS
AS A, HOPEFULLY, VERY VALUABLE SERVICE
TO OUR COMMUNITY, AND WE HOPE THAT YOU WILL TAKE
A FEW MINUTES TO GIVE US SOME FEEDBACK
ON HOW WE’RE DOING. WE HAVE PUT TOGETHER
A SHORT SET OF QUESTIONS THAT YOU CAN ACCESS
AT THE WEBSITE THAT’S CURRENTLY BEING
DISPLAYED. PLEASE DO. IT’S A bit.ly WEBSITE. IT’S 0315 — FOR THE MONTH
OF MARCH IN 2015 — HangoutFeedback, WITH “H”
AND “F” BOTH CAPITALIZED. PLEASE NOTICE THAT
BECAUSE IT WON’T COME UP AS A VALID WEB PAGE
WITHOUT THAT. AND IF YOU’D TAKE JUST A FEW
MOMENTS TO LET US KNOW HOW WE’RE DOING AND WHAT TOPICS
MIGHT BE OF INTEREST TO YOU MOVING FORWARD, WE’D LOVE
TO HEAR THAT, AND WE DO RESPOND. YOU CAN ALSO SIGN UP
FOR KNOWLEDGE ABOUT OUR FUTURE EVENTS. OUR NEXT HANGOUT IS SCHEDULED
TO OCCUR ON APRIL 14th AT 2:00 P.M. EASTERN TIME. AND THE INTELLECTUAL PROPERTY
SPECIALIST IN OUR OFFICE, DR. GAUTAM PRAKASH, WILL BE
PROVIDING AN EXPLANATION OF HOW TO DEVELOP AND PROTECT
YOUR INTELLECTUAL PROPERTY. HE’S CALLING THIS “I.P. BASICS
FOR THE NEW INNOVATOR.” AS WITH TODAY’S PRESENTATION, IT WILL BE AVAILABLE
ON BOTH GOOGLE+ AND YouTube. AND THEN, ON MAY 12th
AT 2:00 P.M., DR. GARY ROBINSON, OUR BUSINESS-DEVELOPMENT
SPECIALIST, WILL BE HOSTING A HANGOUT
FOCUSED ON IDENTIFYING AND CONNECTING
WITH YOUR CUSTOMER. WE CERTAINLY HOPE THAT YOU’LL
JOIN US FOR THESE EVENTS. COMING UP IN JUNE, WE’LL HAVE
ANOTHER REGULATORY ONE, AND WE’RE GONNA CYCLE THESE
THROUGH THE YEAR. BETWEEN NOW AND APRIL 14th, YOU CAN STAY IN TOUCH
WITH OUR OFFICE, THE OFFICE OF TRANSLATIONAL
ALLIANCES AND COORDINATION AT THE NHLBI, BY E-MAIL,
BY PHONE, FOLLOWING US ON TWITTER,
CHECKING OUT OUR WEBSITE, AND SIGNING UP FOR
OUR LISTSERV. WE USE ALL OF THESE RESOURCES
TO HELP KEEP YOU UP TO DATE ON WHAT’S HAPPENING
IN OUR COMMUNITY AND WHERE YOU MIGHT MEET UP
WITH US FACE-TO-FACE. AND IN CLOSING, I’D LIKE TO NOTE
THAT THE SMALL BIZ HANGOUTS ARE ORGANIZED AND PRESENTED BY THE NATIONAL HEART, LUNG,
AND BLOOD INSTITUTE’S OFFICE OF TRANSLATIONAL
ALLIANCES AND COORDINATION. IF YOU ARE DEVELOPING A PRODUCT
OR TECHNOLOGY THAT FALLS WITHIN
THE NHLBI MISSION SPACE, WHICH INCLUDES CARDIOVASCULAR,
PULMONARY, HEMATOLOGIC, AND SLEEP DISORDERS
AND DISEASES, PLEASE CONTACT US THROUGH OUR
WEBSITE, BY PHONE, OR TWITTER FOR ADDITIONAL ASSISTANCE AND
INFORMATION ABOUT THE PROGRAMS AND RESOURCES THAT WE CAN BRING
TO BEAR TO HELP YOU WITH YOUR TECHNOLOGY
DEVELOPMENT. IF YOU ARE DEVELOPING A PRODUCT
OR TECHNOLOGY THAT FALLS OUTSIDE OF OUR MISSION SPACE, WE DO ENCOURAGE YOU TO PLEASE
REACH OUT TO A PROGRAM OFFICIAL IN THE APPROPRIATE INSTITUTE AT
N.I.H. FOR ADDITIONAL ASSISTANCE IN UNDERSTANDING THE SBIR
PROGRAM AND IN UNDERSTANDING THE RESOURCES THAT THEY MAY BE
ABLE TO PROVIDE TO YOU. A LIST OF THE SBIR AND STTR
CONTACTS CAN BE FOUND AT THE WEBSITE LISTED
ON THIS PAGE. AND WITH THAT,
I BELIEVE WE ARE AT THE — I’LL CHECK ONE LAST TIME. AND NO MORE QUESTIONS
HAVE COME IN, SO I’D LIKE TO THANK
DR. REDER AND MR. LIPFORD FOR JOINING US TODAY
AND OUR AUDIENCE. AND WE’LL SEE YOU NEXT MONTH. -THANK YOU, CHRIS.
-THANKS, CHRIS.

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