>> DR. COTE, TELL US ABOUT THE OFFICE OF
ORPHAN PRODUCTS DEVELOPMENT.
>> SURE. THE OFFICE OF ORPHAN PRODUCTS DEVELOPMENT IS
THE HOME, AT FDA, FOR PEOPLE WITH RARE DISEASES AND FOR
PEOPLE WHO WANT TO MAKE THERAPIES FOR PEOPLE WITH
RARE DISEASES.
IT WAS ESTABLISHED BY THE 1983 ORPHAN DRUG ACT, WHICH WAS
THE BRAINCHILD OF A WOMAN BY THE NAME OF ABBEY MYERS.
SHE WAS A HOUSEWIFE FROM DANBURY, CONNECTICUT,
AND TURNED INTO A GRASSROOTS POLITICAL MOVER.
SHE FOUNDED AN ORGANIZATION CALLED NORD, THE NATIONAL
ORGANIZATION FOR RARE DISORDERS, WHICH EXISTS TODAY AND IS DOING
GREAT WORK, ALONG WITH OTHER PATIENT ADVOCACY GROUPS.
SHE BASICALLY HAD A COUPLE OF KIDS WHO COULDN'T GET DRUGS
DEVELOPED FOR THEIR RARE DISEASE BECAUSE IT DIDN'T MAKE
SENSE FOR THE DRUG COMPANY -- YOU KNOW, THE MODEL IS A DRUG
COMPANY SELLS PILLS AND THE NUMBER OF PEOPLE WHO BUY
THE PILLS DETERMINE HOW THEY RECOUP THEIR DEVELOPMENT COSTS.
WELL, THAT DOESN'T WORK FOR RARE DISEASES BECAUSE THERE AREN'T
ENOUGH PEOPLE TO BUY THE PILLS.
>> BUT THERE ARE A LOT OF RARE DISEASES?
>> THERE ARE A LOT OF RARE DISEASES.
THERE'S, LIKE, 7,000 OF THEM, AND THEY ACCOUNT FOR
ABOUT 30 MILLION AMERICANS.
SO, INDIVIDUALLY, THEY'RE INFREQUENT, BUT, COLLECTIVELY,
THEY'RE VERY COMMON.
IT'S A MAJOR PUBLIC HEALTH PROBLEM, THESE RARE DISEASES.
>> SO WHAT DOES YOUR OFFICE DO?
>> SO MY OFFICE IMPLEMENTS THE ORPHAN DRUG ACT, WHICH IS
A MEANS BY WHICH NEW THERAPIES FOR PEOPLE WITH RARE DISEASES
CAN GET SPECIAL INCENTIVES.
AND SO WE DECIDE WHETHER OR NOT A CLAIM THAT THIS IS A NEW
THERAPY FOR A PERSON WITH RARE DISEASES IS VALID OR WHETHER
SOMEBODY'S TRYING TO SCAM THE SYSTEM.
AND THERE HAVE BEEN A TREMENDOUS NUMBER OF NEW THERAPIES THAT
HAVE COME FORWARD.
YOU KNOW, IN THE DAYS BEFORE ABBEY GOT THE ORPHAN DRUG ACT
PASSED, VERY FEW RARE DISEASES HAD THERAPIES.
SINCE THAT TIME, SINCE 1983, 357 NEW THERAPIES HAVE GONE TO
FULL MARKETING APPROVAL AND WE'VE HAD A TOTAL OF 2,100
COMPOUNDS THAT ARE PROMISING THAT HAVE BEEN LABELED AS
ORPHAN DRUGS.
THAT'S WHAT WE DO -- WE FIND ORPHAN DRUGS AND WE
CERTIFY THEM AS THAT.
>> AND GIVE US SOME EXAMPLES OF ORPHAN DISEASES.
>> SURE. WELL, YOU KNOW, THEY'RE ALL OVER THE SPECTRUM.
THEY'RE A BUNCH OF DIFFERENT THINGS THAT YOU MIGHT
NOT IMAGINE.
THEY GO FROM THINGS THAT A LOT OF DOCTORS HAVE NEVER HEARD OF,
AND, CERTAINLY, THAT A LOT OF DOCTORS HAVE NEVER SEEN OR
TREATED, LIKE GAUCHER'S AND POMPE'S, MUSCULAR DYSTROPHY,
CYSTIC FIBROSIS -- YOU KNOW, I GOT 7,000 TO PICK
FROM, SO THERE'S QUITE A LOT -- TO SOME THINGS YOU MIGHT NOT
EXPECT, THINGS WHERE THE POPULATION IS ZERO,
LIKE AGENTS OF BIOTERRORISM, WHICH HAVEN'T --
YOU KNOW, FORTUNATELY, THE POPULATION IS STILL FEWER THAN
200,000 AMERICANS, WHICH IS THE RULE FOR WHERE WE MAKE
THE CUTOFF FOR A RARE DISEASE.
OTHER THINGS THAT YOU MIGHT NOT EXPECT ARE DISEASES THAT ARE
COMMON IN THE REST OF THE WORLD, BUT ARE RARE AMONG AMERICANS,
LIKE MALARIA, SCHISTOSOMIASIS, ALL THOSE TROPICAL,
WORMY DISEASES.
SO THERE'S A LARGE RANGE OF DIFFERENT DISEASES
THAT ARE RARE.
>> SO IN MANY WAYS, YOUR OFFICE MEETS THE PUBLIC HEALTH
RESPONSIBILITY OF THE FDA BY BEING A LIFELINE FOR FOLKS WHO
ARE IN DESPERATE NEED.
>> THAT'S RIGHT, AND SOME OF THOSE LIFELINES THAT HAVE BEEN
THROWN HAVE ACTUALLY RESULTED IN SOME MAJOR MIRACLES.
>> WHAT ARE SOME OF THE FRUSTRATIONS IN YOUR JOB?
>> WELL, IT'S THE GOVERNMENT.
THE GOVERNMENT ALWAYS HAS FRUSTRATIONS, AS YOU WELL KNOW.
YOU WORK FOR THE GOVERNMENT.
YOU KNOW -- OUR ORGANIZATION IS ACTUALLY
GROWING, IT'S EXPANDING VERY RAPIDLY, BUT NOT RAPIDLY ENOUGH.
IF THERE WERE A FRUSTRATION -- I TOLD YOU WE HAVE 350 NEW
THERAPIES FOR PEOPLE WITH RARE DISEASES, BUT I TOLD YOU WE HAVE
7,000 RARE DISEASES, SO WE'RE SCRATCHING THE SURFACE
RIGHT NOW.
THINGS CAN'T EXPAND FAST ENOUGH FOR THE PEOPLE WHO REALLY NEED
THEM, SO THAT'S THE MAJOR, SINGLE MOST IMPORTANT THING,
IS THAT WE HAVE TO HURRY BECAUSE PEOPLE HAVE CHILDREN WHO NEED
THERAPIES RIGHT NOW.
>> WHAT ARE SOME OF THE THINGS ABOUT YOUR OFFICE THAT
YOU THINK THE PUBLIC DOESN'T KNOW ABOUT AND SHOULD?
>> WELL, WHEN YOU THINK "RARE DISEASES," YOU SHOULD KNOW THAT
THERE IS A PLACE, A HOME, FOR THEM AT THE FDA.
WE WORK AS AN OMBUDSMAN FOR PATIENTS, PATIENT GROUPS,
INDUSTRY, EVERYBODY.
IT'S A CONCERTED EFFORT.
IT'S GETTING BIG, NOW.
YOU KNOW, WE ACTUALLY -- ABOUT A THIRD OF ALL OF THE NEW
THERAPIES THAT FDA APPROVES NOW ARE ORPHAN PRODUCTS,
SO WE'VE COME A LONG WAY FROM THOSE DAYS WHEN ABBEY SET UP THE
ORPHAN DRUG ACT AND THINGS HAVE GONE FORWARD TREMENDOUSLY.
WE'RE EXPECTING A LOT AS PERSONALIZED MEDICINE
GOES FORWARD.
WE ARE VERY HUMAN, OKAY?
PEOPLE COME TO US AND THEY'RE -- FDA SUFFERS FROM A LITTLE BIT OF
AN OPACITY PROBLEM.
IT'S SORT OF A BLACK BOX -- PEOPLE DON'T KNOW WHAT GOES ON
INSIDE THERE.
AND OUR CRITERIA, THE WAYS THAT WE WORK AND THAT WE HANDLE
THINGS ACCORDING TO REGULATIONS, ARE PRETTY STRAIGHTFORWARD,
PRETTY SIMPLE.
FOR EXAMPLE, A GOOD ORPHAN STATUS DESIGNATION APPLICATION
COULD BE DONE IN ABOUT EIGHT OR NINE PAGES, AND I HAVE RECEIVED
APPLICATIONS THAT SIT IN VOLUMES THAT ARE TALLER THAN YOU ARE.
SO THOSE ARE THE KINDS OF PROBLEMS.
PEOPLE ARE A LITTLE BIT -- THEY DON'T KNOW WHAT TO EXPECT
OF THE FDA, BUT IT'S MOSTLY JUST PEOPLE HELPING AMERICANS.
>> AND, WHILE YOU'RE A LIFELINE TO PATIENTS, YOU'RE
ALSO A PROMPT FOR INDUSTRY AS WELL, BECAUSE INDUSTRY WOULD
LIKE TO MAKE THESE PRODUCTS AS WELL AND YOU GIVE THEM
A WAY TO DO IT.
>> THE TRUTH IS THAT THE FDA DOESN'T MAKE DRUGS.
WE DON'T MAKE MIRACLES.
MIRACLES ARE BEING MADE AND WE'RE OVERSEEING THEIR SAFETY
AND THEIR EFFICACY AND THAT THEY'RE APPROPRIATE FOR
POPULATIONS, BUT WE, OURSELVES, DON'T ACTUALLY MAKE DRUGS.
WE'RE REGULATORS.
SO, YEAH, THEY ARE A VERY IMPORTANT PART OF THE PROCESS,
AND SO WE NEED TO -- OUR MISSION IS TO SERVE THE
AMERICAN PEOPLE, AND INDUSTRY IS A TOOL THROUGH WHICH WE DO THAT.
>> DR. COTE, I NOTICE THAT YOU'RE WEARING A UNIFORM.
WHAT'S THAT ALL ABOUT?
>> SURE. THIS UNIFORM IS THE UNIFORM OF
THE U.S. PUBLIC HEALTH SERVICE.
THE PUBLIC HEALTH SERVICE HAS EXISTED FOR A LONG TIME --
IT GOES BACK TO 1798 -- AND WE ARE THE NATION'S RESPONSE
TO PUBLIC HEALTH NEEDS.
THE NATION'S ALWAYS NEEDED TO HAVE DOCTORS TO SEND IN TO
OUTBREAKS AND EPIDEMICS AND DOCTORS TO SEND TO KATRINA AND
OTHER EMERGENCIES.
THESE DAYS, MOST OF US WORK AT DIFFERENT AGENCIES --
AT FDA, CDC, NIH -- AND AT THOSE AGENCIES,
THERE ARE PEOPLE IN UNIFORM AND OUTSIDE OF UNIFORM.