Clinical Trials for Children: Messages for Parents and Caregivers
16
November

By Adem Lewis / in , , , , , , , , , , , , , , , , , /


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.


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