Woman: PEDIATRIC CLINICAL TRIALS ARE IMPORTANT
FOR ENABLING A BETTER FUTURE FOR OUR CHILDREN.
IT IS WITH CLINICAL RESEARCH AND CLINICAL TRIALS
THAT WE MAKE SIGNIFICANT ADVANCES IN MEDICINE.
THE CHANGES THAT I'VE SEEN
IN THE TIME THAT I'VE BEEN IN MEDICINE
HAVE BEEN ABSOLUTELY MINDBOGGLING.
CHILDREN WHO, WHEN I STARTED IN PEDIATRICS,
NO ONE WOULD EVER HAVE EXPECTED THEM TO SURVIVE
ARE ROUTINELY SURVIVING AND HEADING OFF TO KINDERGARTEN
AND GRADUATING FROM HIGH SCHOOL.
WE'VE MADE ENORMOUS PROGRESS IMPROVING THE OUTCOMES
FOR PRETERM INFANTS AND FOR TERM INFANTS
WITH A VARIETY OF PROBLEMS.
WE HAVE LOTS OF DISCOVERIES THAT HAVE LED TO, BASICALLY,
THE ERADICATION OF MANY INFECTIOUS DISEASES:
TETANUS, PERTUSSIS, MEASLES, MUMPS, RUBELLA.
ONE AREA THAT WE'VE MADE REALLY HUGE STRIDES IN
IS THE USE OF INHALED NITRIC OXIDE
TO RELAX THE BLOOD VESSELS TO THE LUNGS.
WHEN THAT FIRST CAME OUT, IT WAS REALLY--
I REMEMBER USING IT. IT WAS A MIRACLE.
THE AVERAGE LIFE EXPECTANCY FOR SOMEONE WITH CYSTIC FIBROSIS
WAS 7 YEARS OF AGE.
THESE DAYS, THE AVERAGE LIFE EXPECTANCY IS 37.
THAT'S A REALLY DRAMATIC CHANGE,
AND IT REPRESENTS A WHOLE VARIETY OF STUDIES.
THE ONLY WAY THAT THOSE STUDIES COULD HAVE BEEN DONE,
THOSE TRIALS, WAS EXCELLENT SCIENCE
AND PARENTS WHO ARE WILLING TO SAY--WHO WOULD LISTEN AND SAY,
"OK, I WANT TO GIVE MY BABY A CHANCE."
ALL TOO OFTEN WE HEAR THAT CHILDREN ARE SMALL ADULTS,
AND WE KNOW THAT'S NOT TRUE.
SOME DISEASES OCCUR IN CHILDREN
THAT DON'T OCCUR IN ADULTS AT ALL.
SOME DISEASES OCCUR BOTH IN CHILDREN AND ADULTS--
FOR INSTANCE, ASTHMA--
BUT ACTUALLY BEHAVE SOMEWHAT DIFFERENTLY IN CHILDREN
THAN IN ADULTS.
AND FINALLY, CHILDREN OFTEN METABOLIZE DRUGS
QUITE DIFFERENTLY FROM ADULTS.
AND SO WE REALLY NEED TO KNOW SOMETHING VERY SPECIFIC
ABOUT HOW A GIVEN THERAPY OR A GIVEN SET OF THERAPIES
IS HANDLED BY A CHILD COMPARED TO AN ADULT.
MOST OF THE MEDICATIONS THAT WE USE IN CHILDREN
ARE MEDICATIONS THAT HAVE BEEN APPROVED BY THE FDA
FOR USE IN HUMANS,
BUT MOST OF THEM HAVE NOT BEEN TESTED IN CHILDREN,
BECAUSE HISTORICALLY THAT EITHER WASN'T REQUIRED
OR IT WASN'T FEASIBLE.
AND SO WE BORROW THOSE MEDICATIONS,
AND WE BORROW THE LITERATURE AND THE INFORMATION
THAT HELP US TO CHOOSE A MEDICATION FOR ADULTS,
AND WE JUST DO OUR BEST.
Kaskel: THE ONLY WAY TO EXAMINE THE SCIENTIFIC VALUE
OF A NEW THERAPY IS BY A PEDIATRIC CLINICAL TRIAL.
Davis: WITH A PARENT, WHEN YOU GO TO THEM
WITH A CLINICAL RESEARCH STUDY,
YOU'RE GETTING THEM AT A VERY VULNERABLE TIME.
Woman: YOU NEVER EXPECT THAT YOU'RE GONNA HAVE A SICK BABY.
McEvoy: ALL OF THESE SITUATIONS ARE VERY STRESSFUL.
AND I THINK IN TERMS OF BEING SUCCESSFUL
IN GETTING THE PARENTS AND THE FAMILIES
TO PARTICIPATE IN RESEARCH,
IT TAKES A LOT OF ONE-ON-ONE COMMUNICATION.
ONE OF THE COMMONEST CONCERNS THAT PARENTS HAVE
IS WHEN THEY HEAR THE WORD "CLINICAL TRIALS",
THAT IS EQUATED WITH EXPERIMENTATION.
IT EVOKES A SENSE OF FEAR, AND THAT'S NATURAL.
NO PARENT REALLY WANTS THEIR CHILD TO BE EXPERIMENTED ON.
THE PARENT WANTS TO BE SURE THAT THEY'RE DOING THE RIGHT THING
FOR THEIR CHILD
AND THAT THEY'RE NOT PUTTING THEIR CHILD IN ANY DANGER,
PUTTING THEIR CHILD IN ANY INCREASED RISK.
McEvoy: SOMETIMES PARENTS MIGHT THINK THAT THE INVESTIGATOR
IS MORE WORRIED ABOUT THEIR STUDY THAN THEIR CHILD.
THEY WANT TO KNOW, OK, SO, WHAT'S THE BENEFIT.
IS THE BENEFIT GONNA BE IMMEDIATE TO THEM,
OR IS THE BENEFIT IN GENERAL GONNA BE APPLIED
TO A GROUP THAT THEY'RE PART OF?
WHAT HAPPENS SOMETIMES IS
PATIENTS ARE RELUCTANT TO PARTICIPATE BECAUSE
THEY THINK THE STANDARD OF CARE IS PERFECTLY FINE.
AND THEN YOU HAVE TO EXPLAIN TO THEM THAT, FOR US,
THE STANDARD OF CARE HASN'T BEEN TESTED THAT WELL.
WE NEED TO UNDERSTAND
WHETHER THERE ARE MORE EFFECTIVE THERAPIES
AND THEY MAY BENEFIT THEM IN THEIR OVERALL HEALTH.
IT'S A VERY HARD DECISION FOR PARENTS TO MAKE,
TO ENROLL A CHILD THAT SMALL AND THAT VULNERABLE
IN A CLINICAL TRIAL.
BUT WE CAN'T IMPROVE THEIR THERAPIES
UNLESS WE ACTUALLY STUDY THE THERAPIES
THAT WE'RE USING FOR THEM.
SO WE SEE IT AS A PARTICULARLY STRONG RESPONSIBILITY
TO FULLY ENGAGE THE FAMILIES IN THESE CONVERSATIONS
SO THAT THEY UNDERSTAND WHAT WE'RE DOING.
Kaskel: WE MEET WITH THEM
TO GO OVER THE WHOLE IDEA OF THE TRIAL,
THE GOALS, THE OUTCOMES, THE PROCESSES,
IN A NON-BINDING MECHANISM.
THEY'RE GIVEN INFORMATION IN ENGLISH AND SPANISH
OR OTHER LANGUAGES, IF NEED BE.
FOR THE PARENT WHO IS UNCERTAIN,
WE DON'T WANT TO PRESSURE THEM IN ANY WAY.
WE UNDERSTAND. I MEAN, THIS IS THEIR BABY.
AND WE WANT THEM TO BE VERY THOUGHTFUL ABOUT THE PROCESS.
IF THEY DON'T WANT TO DO IT,
IT'S NOT GONNA CHANGE OUR CARE FOR THAT BABY AT ALL.
Shurin: PEOPLE WHO GO INTO CLINICAL RESEARCH IN PEDIATRICS
ARE A VERY SPECIAL BREED OF PEOPLE.
THEY HAVE A VERY, VERY POWERFUL COMMITMENT TO THE CHILDREN
WHO ARE IN THEIR STUDIES
AND WITH WHOM THEY'RE DEALING.
I THINK MOST PEDIATRIC INVESTIGATORS
REALLY CONSIDER THE CHILDREN AND THEIR PARENTS AND FAMILIES
TO BE CO-INVESTIGATORS IN THE STUDIES.
MOST OF US ARE PARENTS, AND SO WE ASK OURSELVES,
"WOULD I ENROLL MY CHILD INTO THIS PARTICULAR STUDY?"
AND IF I COULDN'T SAY YES,
AS A PARENT, I PROBABLY WOULDN'T BE DOING THAT STUDY.
WE'RE DOING AS MUCH AS WE CAN TO SAFEGUARD THE CHILDREN
THAT ARE INVOLVED
AND REWARD THE TRUST AND LOYALTY OF OUR PARENTS.
THERE ARE A VERY RIGOROUS SET OF FEDERAL REGULATIONS
THAT GUIDE ENROLLMENT, RECRUITMENT, AND CONDUCT
OF PEDIATRIC RESEARCH.
OUR FIRST RESPONSIBILITY IS TO THE SAFETY OF THE CHILD,
AND THAT SAFETY OVERWEIGHS ANYTHING
THAT MIGHT BE REQUIRED IN THE PROTOCOL.
ALL OF THESE REVIEW PROCESSES ARE DONE TO MAKE SURE
THAT FOR EVERY PARTICIPANT, THE POTENTIAL BENEFIT
FAR EXCEEDS ANY POTENTIAL RISK.
Gipson: THE PARENT AND THE CHILDREN
ACTUALLY ARE VERY INTERESTED IN BEING PART OF THESE STUDIES.
THEY LOOK UP AND SAY, "I HAVE A HEALTH PROBLEM,
AND IF THAT STUDY MIGHT HELP ME, THAT WOULD BE WONDERFUL,"
BUT THERE'S AN AMAZING SENSE OF ALTRUISM.
SO, THEY LOOK BEHIND THEM AND SAY,
WHAT I'D REALLY LIKE TO DO, THOUGH,
IS TO SOLVE THIS PROBLEM
OR MAKE THEIR LIFE JUST A LITTLE BIT EASIER.
FOR CHILDREN AND FAMILIES WHO COME IN BEHIND ME.
THEY ARE REALLY VERY INTERESTED IN FURTHERING MEDICAL KNOWLEDGE
TO HELP OTHER FAMILIES COMING AFTER THEM.
Kamani: WHEN YOU HELP CHILDREN WHO ARE SICK
AND OFTEN HAVE OTHERWISE FATAL DISEASES
AND YOU CAN CURE THEIR DISEASE,
THEY LIVE WONDERFUL, FULL, HEALTHY LIVES.
A DAD SAID TO ME, "DR. WAGNER, YOU HAVE TO SAVE MY BABY."
AND IN THOSE MOMENTS, AT 2:00 IN THE MORNING,
THAT'S WHEN SOMEONE REACHES OUT TO YOU
AND YOU THINK OUTSIDE OF THE BOX.
SO, TO ME, THAT'S MY PASSION.
AND I THINK IT'S A PRIVILEGE AND AN HONOR
TO NOT ONLY TAKE CARE OF BABIES,
BUT IN DOING RESEARCH AND CONDUCTING RESEARCH,
YOU CAN AFFECT THOUSANDS OF CHILDREN
IF YOU DESIGN A STUDY THAT'S INSIGHTFUL
AND IT HAS A HUGE IMPACT.
Pemberton: PARENTS ARE REALLY THE ESSENTIAL PIECE TO THIS WHOLE PUZZLE.
WITHOUT THEM AND CHILDREN,
WE WOULDN'T BE ABLE TO ANSWER A LOT OF THE QUESTIONS
THAT WE NEED TO ANSWER.
THEY REALLY ARE HEROES,
WHEN YOU STOP AND THINK ABOUT IT,
TO OFFER UP THEIR CHILDREN, THEIR FAMILIES, THEIR TIME,
TO RESEARCH AND TO HELP NOT ONLY PERHAPS THEMSELVES
AND THEIR CHILDREN
BUT TO HELP CHILDREN THAT THEY'VE NEVER MET BEFORE.
THERE IS HOPE THAT THERE'S A BETTER DAY OUT THERE,
AND THE WAY THAT WE'RE GONNA GET THERE
IS THROUGH THIS CAREFULLY DESIGNED TRIAL.
WE'LL KNOW AN ANSWER,
AND THAT WILL ALLOW US TO GO TO THE NEXT STEP.
SO, IT'S BUILDING A SERIES OF FOUNDATIONS
THAT WILL TAKE US TO A BETTER TOMORROW.
IF YOU'RE A PARENT OF A SICK CHILD,
THAT'S WHAT YOU HOPE FOR-- IS A BETTER TOMORROW.