Saturday, March 29, 2014

South-East Asia Region: Certified Polio Free

Dear All,

Today, on 27th 
South-East Asia 
 is officially declared 
 This is an opportunity to look at progress the region has made advancing children’s health, highlight the power of vaccines, and encourage continued political and financial support for polio eradication.
 1.8 billion
 people across 
11 countries
 are polio-free thanks to unprecedented commitment from governments, exceptional program quality, and the dedication of millions of community health workers and volunteers.

o In 2007, there were approximately 
 polio cases 
in SEAR – 
 of all polio cases worldwide. The region has not reported a case since 2011.

o Between 1995 and 2013, the polio program conducted at least 
189 nationwide polio campaigns
and administered more than 
13 billion OPV doses
 across the region.

o India, once deemed the most difficult place to end polio, recorded its 
last case in January 2011 
– a remarkable triumph that opened the door for the entire region to be certified polio-free.

More than 
3 million front-line workers
 across South Asia (India, Nepal, Bhutan, Maldives, Sri Lanka, Bangladesh) provided oral polio vaccine to children under 5.

 Ending polio in these countries—some of which have been polio-free for more than 15 years—forged strong systems that are now being used to advance other health priorities.

o In several countries, 
high-performing polio surveillance systems
 have been expanded to track other vaccine-preventable diseases such as measles, neonatal tetanus, and Japanese encephalitis.

o In Bangladesh, immunization coverage for essential vaccines (diphtheria-tetanus-pertussis) rose from
82% to 96% 
between 2000 and 2012, in a period of intense polio eradication activity; in Nepal, the rate went from 
74% to 90%
In order to protect gains against polio, we must remain committed to 
improving routine immunization and maintaining sensitive surveillance

Recent outbreaks in the Middle East and the Horn of Africa—both linked to virus from endemic countries—are proof that as long as polio exists anywhere, it is a threat everywhere.

Dr. Naveen Thacker

Patient Cracks Her Own Mysterious Dual Diagnosis - using genetics !

Ron Zimmerman
March 18, 2014
SAN DIEGO — Kim Goodsell started noticing physical ailments about 20 years ago, when she was in her 30s and training for an Ironman triathlon. She said she felt "a strange instability" related to the adrenaline of training and had to drop out of the competition.
After finally being diagnosed with 2 rare diseases, "I started looking for what I called the unifying field theory to explain why I had both these conditions," Goodsell said here at the Future of Genomic Medicine VII.

"As a physician, I've never met a patient like her," Eric Topol, MD, director of the Scripps Translational Science Institute in La Jolla, California, and editor-in-chief of Medscape, said during an interview. "She was able to crack the unifying diagnosis after having been told by the top experts in the country that she simply had bad luck."
Goodsell, who is now 56, said that years after she quit training, she experienced a run of tachycardia and was given her first diagnosis — arrhythmogenic right ventricular cardiomyopathy. After she had a defibrillator implanted, she started experiencing excruciating pain. "I couldn't even hold a fork," she said.
Goodsell's second diagnosis, for the neuropathy, was Charcot–Marie–Tooth (CMT) disease type 2, a progressive degenerative muscle disease. She underwent a full hip replacement for hip dysplasia.
In 2010, Goodsell asked physicians at the Mayo Clinic whether her ailments were related. She was told no. In fact, according to Dr. Topol, the odds of having arrhythmogenic right ventricular cardiomyopathyand CMT are 4 in 10 million, which is "less than the chance of being hit by an asteroid."
But Goodsell wanted answers. "I started looking for what caused my CMT, what genes might be involved," she said. "I came up with theLMNA gene. Mutations in this gene can cause bone disease, cardiac problems, neuropathy, and systemic degeneration. All my symptoms were there."
Goodsell's geneticist tried to dissuade her from private genomic testing, suggesting that the $3000 cost would be of minimal benefit. But Goodsell insisted.
"Lo and behold, the test came back positive," she said.
Then the real research began for Goodsell, who went on to study the molecular pathways of the products of the LMNA gene. "I found there was a convergence with lamin A/C and desmosomal proteins, and that I had a mutation in the LMNA gene that was perturbating the downstream desmosomal proteins. I wrote a whole dissertation on it," she explained.
The results were remarkable, said Dr. Topol. "Not only did she diagnose her disease, she set up her own treatment plan."
She now follows a strict diet. She eats no processed foods containing excitotoxins (free glutamates), and no gluten or solanine, a toxin found in nightshades like peppers, tomatoes, and eggplant, which she previously loved to eat.
Goodsell now has a new outlook on life. "I've dramatically improved. I've not only attenuated the progression of the disease, I've reversed it," she said. "I had lost function in my left hand and couldn't walk without support. I was in so much pain I couldn't see the purpose of living. But I'm back to my old life, traveling the world with my husband having outdoor adventures."
"She's a great inspiration," said Dr. Topol. "Not only to the public, but also to the medical profession. We were not aware that a patient would be capable of doing what she did. Leading physicians in the country were shocked that she could decipher her own problems. We need to give more respect to consumers."
Inspiration for Doctors and Patients
What is significant about Goodsell, according to Dr. Topol, is her single-minded focus and energy. "She said, 'I'm not going to sit with this, I'm going to research this myself'," he explained. "This is a story of someone with no medical background who focused on her multiple genetic diseases and cracked the case."
This is a particularly instructive case because the technology that Goodsell used is very recent. "Five years ago, she wouldn't have been able to do that research," he explained.
This was "a great way to jumpstart the conference," said John Barnard, MD, a pediatrician and medical director of the Research Institute at the Nationwide Children's Hospital in Columbus, Ohio. "That's really what it's all about — insight on human disease," he told Medscape Medical News.
However, Dr. Barnard added, "my second thought was a negative one. The story is compelling, but the woman was obviously an outlier in terms of knowledge, intellect, and energy to be able to do what she did."
Dr. Barnard said he thought about Ohio, "where I'm from, and Appalachia, where there are a lot of poor people who are uneducated and ignorant about genomics. When we take genomics public, how do we take it to Appalachia, where the healthcare needs are so dramatic, and how do we take it to the south side of Chicago, east St. Louis, and east LA," he asked.
Dr. Topol said that Goodsell is probably the first fully informed genomics patient he's seen in his practice, but that doctors should be prepared for this to change. "I think she's an exemplar of the future."
Going forward, Dr. Topol noted, "there will be a lot more patients like Kim. Millions of people will have their genome sequenced. If doctors don't get 'genomified,' their patients will."
Dr. Topol is a shareholder in Cypher Genomics. Dr. Barnard has disclosed no relevant financial relationships.
Future of Genomic Medicine (FoGM) VII. Presented March 6, 2014.

Is There Really a Sports Gene? - Medscape

David Epstein
March 21, 2014
Editor's Note:

At the Future of Genomic Medicine VII conference in March 2014, leading experts in genomic research and clinical application discussed the expanding influence of genomics on the practice of medicine. Medscape asked David Epstein, author of The Sports Gene: Inside the Science of Extraordinary Athletic Performance, to offer some insight on what clinicians need to know about advances in sports and exercise genetics.
Medscape: Are there individuals with a genetic predisposition to excel in sports?
Mr. Epstein: It is a complicated question and, to some degree, is dependent on the sport skill you are talking about. Some things that were previously thought to be genetic characteristics, such as the bullet-fast reactions of Major League Baseball hitters, turned out to be really the result of practice, whereas other things, such as the compulsive drive to train, turn out to have important genetic components. So it really depends on what you are looking at.
Part of what is coming out of sports and exercise genetics is an idea similar to what came out of medical genetics. Medical genetics is showing us that in some cases, no 2 people will respond to, say, acetaminophen the same way because of differences in genes involved in acetaminophen metabolism. Exercise genetics is showing that this is the same for the medicine of athletic training. No 2 people, because of their genes, will respond quite the same way to the same training stimulus. The goal is to find the optimal training and exercise environment, whether it is for a sports performance or for health effects, for each person's individual genome.
Medscape: How should clinicians address these issues in their own practices?
Mr. Epstein: There are a couple of things that clinicians should keep in mind. Some of them now get questions about testing for genes for athleticism, and usually this will include a gene such as the ACTN3gene, which codes for protein found only in fast-twitch muscle fibers. It is sort of sexy to test for this gene now. If you don't have one of the so-called right versions for sprinting, you just won't be in the Olympic 100-meter final. But that only rules out 1 of 7 billion people on earth, so it is incredibly nonspecific.
To make a decision for a child about what sport they should pursue on the basis of this one gene is like looking at one piece of a puzzle that has a thousand pieces and deciding what the puzzle shows. It is incredibly complex. We don't know a lot of the genes, much less the environmental stimuli that go into this.
For the most part, I think genetic testing for sports prowess should just be ignored. You are better off just seeing what the kid is good at physiologically. The best genetic tool you have for sprinting right now is a stopwatch, not the ACTN3 gene.
That being said, I do think there are genes that clinicians should consider talking with patients and parent about -- genes that predispose people to injuries, such as torn tendons and ligaments. These are genes that code for collagen. And I think it is time to have the conversation about the so-calledAPOE-4 gene variant. We have known for a long time that it predisposes people to having Alzheimer disease, but it now looks like it is involved in all manner of recovery from brain injury.
We are having this national furor about brain trauma in football and, to a lesser degree, in soccer, and there is this gene variant out there that might give some risk information.
When I have talked to clinicians, generally their take has been, "No, we don't want to test people for this, because it is just statistical risk information." But telling someone that smoking puts them at an increased risk for heart attack is also just statistical risk information. And the response I get to that is, "Well, they can stop smoking, but they can't change their DNA."
True -- but they absolutely can change their environment by, for example, not playing football. I think that is a discussion that clinicians should be having with patients soon.
Medscape: Where is research in this field headed?
Mr. Epstein: I think the direction that sports and exercise genetics are going toward is individualized training, to a degree. Obviously, it is very difficult, and we have seen some of the difficulties of individualized medicine. But scientists are making some strides in finding genetic markers that predict differences between people and their responsiveness to a particular training plan. Whether that is how much their cardiovascular system strengthens or how much their blood pressure drops, the idea would be to be able to tell someone, "Look, you want this drop in blood pressure, and you have a set of genes predicting that you can get that with this training, or without even training that hard" -- whereas with some other person, you say, "Wow, you would have to train really hard, so you may need to be medicated." We are making strides in that direction.

Friday, March 28, 2014

The new liquid Whole Cell Pertussis Pentavalent vaccine by Novartis (QUINVAXEM) now in India - is it really less painful & worth paying more money?

Here is a discussion going on about the new liquid Whole Cell Pertussis Pentavalent vaccine by Novartis (QUINVAXEM). It costs almost twice as much as the regular whole cell pertussis vaccines, and the company claims that it is having minimal side-effects similar to the acellular pertussis vaccine. 
Here is what a learned doctor had to say
Dear Dr.,
Such claims are not evidence based. When the MR of Novartis claimed such before me i asked for scientific evidence and prove it. He didn't turned up then. More over i also searched the literature including pubmed but could not find and such study. So this is baseless claim , doctors should not get carried out by such claim without evidence. I presume that such false claim may be such to justify their exorbitant cost difference- if i am not wrong 120 times more then DTwP from indigenous manufacturer.
with regards

Dr. Digant D. Shastri
Immediate Past Chairperson IAP Infectious Diseases chapter?

Vice President, IMA Surat 2013-2014
Executive Board Member, Central IAP 2007 ,2008, 2010,2013
Past President IAP Gujarat Branch ( 2007 )

Comment: I would have to agree with the expert opinion here. This vaccine is available in many countries for years now. Nowhere else has this vaccine being promoted as an alternative to DTaP. Also, if it was having such low reactogenicity, why would the western countries having epidemics of Pertussis, possibly related to use of DTaP, not shift to this vaccine?  

Thursday, March 27, 2014

Free SMS based Vaccination Reminder Service for Indian children

The world's largest vaccination reminder service

IAP-Immunizeindia is the world's largest vaccination reminder service, and is available free of cost to parents anywhere in India. It is a national non-profit initiative, promoted by Indian Academy of Pediatrics.
IAP-Immunizeindia aims to prevent half a million child deaths and disabilities by 2018.
IAP-Immunizeindia is supported by Vodafone, which is the national telecom sponsor, and IAP-Immunizeindia's national press campaign, promotional posters in hospitals and clinics, promotional pamphlets are funded by an education grant from a leading global corporation.
How to register for the service

Parents opt-in to the service by sending a text message by SMS to the national shortcode 566778 from any mobile phone in India, in the following format*** :

Immunize < Space>   
Example : Immunize Rekha 04-11-2013

The phone will immediately receive a confirmation message.
Text message reminders** will  be sent to the phone for 12 years, following the IAPCOI prescribed immunization schedule.  
3 reminders are sent, at 2 day intervals, for each vaccination that is due. An example of a reminder is - "Rekha is due for a vaccination this week, please do not forget to visit your doctor"**
Reason for the service

Over 2 Million children under the age of 5 die every year in India* and another 1 Million or more are disabled for life. 

A major cause is that parents often forget to vaccinate the child on time, as most parents in India do not maintain a vaccination calendar for their child in a disciplined manner. Other reasons are-
            - parents' misplaced priorities
            - low awareness
            - busy lifestyles
            - forgetfulness
            - social and cultural causes
Vaccination reminder services in several countries have been effective in increasing compliance by 20%. There are over 40 published scientific studies that prove the effectiveness of vaccination reminders.
India has 700 million mobile phones which support Text Message Service or SMS (every family has a phone). A well promoted, national, Text Message reminder service will be the the most cost effective method of  reminding parents that a vaccination is due. 
Contact - For clarifications and support please send an email to 
*     Source- Global Immunization Vision and Strategy (GIVS). 2006-2015, World Health Organization
**    Reminders do not advertise, recommend or promote any vaccine brands or products.
***   Service not available for phones outside India

India Vaccine News - Now Chicken Pox Vaccine running short !

As of March 2014, both the available Chicken Pox vaccines in India, namely Varivax (VHB Pharma) & arilrix (GSK) are running short and presently not available at least anywhere in Chandigarh. The stockist claim that further stocks are likely to become available in the next 10-15 days.
Given the fact that Chicken Pox is generally a mild disease in children, the lack of availability is not a major concern, however the trend of vaccine shortage is a cause of concern, and can play havoc with children's vaccination schedule. This can lead to confusion in parents, and even missed vaccines in children leading to serious illnesses.

Wednesday, March 26, 2014

An open letter to the teachers of my daughter - a letter published in the Times of India regarding educating children

Dear all.
Greetings from
Here is a mail article sent by Our HSG Dr Praveen Mehta which was published in Times of India.
A very sensitive letter from concern father to teacher.
Renowned author name is in bottom of mail- we from our community pay humble regards to him for such teaching letter.
Please go through:-

?An open letter to the teachers of my daughter - The Times of India

My dear teachers,

It appears you had difficulty understanding my daughter, and being her responsible father, who may clearly see her flaws and weaknesses, I am trying to reply to some of the direct and indirect questions you posed to my daughter during the last one year. Before I begin, I would like to remind you that maybe my child is having some problems related to carelessness and she may be less focused compared with your favourite children, but each child is unique and has their own aptitude to bring to the world. Our duty is to help the child bring out those talents and encourage them to develop their own individuality. All the time we don't have to agree with them but making them understand the same with a little care and support may give them more confidence to explore ideas on their own.?

You had a problem why my daughter laughs a lot. Please permit me to mention here that now her unfortunate father is searching for her lost smile for the last few months. I am sorry for the inconvenience (which you may have considered trouble), if any, you had because of my daughter, but it should be understood how much she means to me. I taught her to laugh when she is happy, I taught her to laugh when she is sad, I taught her to laugh when things do not happen as she wanted and I taught her to laugh at herself every time she made a mistake.?
You made a strange discovery that my little girl is having giraffe legs. As she is growing so fast, I sometimes wonder where my little girl is. I taught her that she should stand tall and be confident and proud and never feel any fear; she still has to walk a million miles to achieve all her dreams and goals. You told her that the entire staffroom knows that she would walk up to the Principal if you scold her. A fact you must know is that with God's love in her heart and parents' courage in her soul, and your school's Principal as her ideal, she feels comfortable to open her mind. In fact, I see her as one of millions of kids who will build a strong and happy India. Today we need our young people to speak their mind, not just listen to the leaders, but ask questions, tough questions, so tomorrow they become a source of courage and happiness to others.?

Its seems you always find her a happy-go-lucky girl student with a bad handwriting who also keeps her bag and books carelessly in the classroom. I may agree with you here but cracking jokes about her and asking her best friend how she could tolerate my little child is unbecoming of you as a teacher and such behaviour is unwarranted and uncalled for.?

During the last PTA, you had a complaint that my child is always interested in taking part in the school's extracurricular activities and annual day functions. To the best of my knowledge, students should always be advised to participate in extracurricular activities such as sports, debates and discussions, because these activities would help in all-round personality development. I am also surprised that in this 21st century you still follow the age-old formula as they say in Hindi: "Kheloge kudoge hoge kharab".?

Just a few days ago, during her final exams, you asked the classroom whether anyone had any query regarding the question paper and when my daughter raised her hand, it appears you told her that she herself is a problem thus belittling her in the class and giving the classroom a chance to laugh at her cost, and this was not the first time she faced this humiliation. I am deeply sad to say your unwarranted behaviour shattered her confidence and now she hesitates to ask even basic questions. I am still wondering if the school authorities have bestowed upon you any right to belittle my child in front of her class. All children are God's gift and as a teacher you should have the patience to treat them all alike and to instil in them loads and loads of confidence, but not to shatter it.?

The mental abuse of a child is as bad and dangerous as physical punishment. Because it could put a comma, maybe even a full stop, to her mental growth and development.?

Don't you understand that by your unkind and unreasonable behaviour you are stalling the mental development of a child through your misunderstanding of a corrigible deficiency in a child? I hope you agree that making fun of a student repeatedly in front of her classmates is as bad as mental torture. Such behaviour on the part of a teacher is deplorable and such a person does not deserve to be called a teacher. By such behaviour, you are in fact stalling their growth instead of allowing them to blossom. Perhaps the school and the society can do better without such teachers if they do not mend their ways.?

In conclusion, I would like to say that the teacher instead of insulting and rebuking children in front of their class (and thereby stalling their mental growth) should work for the development of the children. Instead of belittling them, help them understand about the wonderful gift of human life, build strong roots in them about the social, ethical and moral values of life, about their positive participation in the inclusive growth of school life, family, society and the nation. Teachers who cannot participate in building the life of the students have no place in society. The society is in need of teachers who treat their student with an equal eye, love, compassion, understanding and consideration, and not teachers who belittle them in front of their class and thereby destroy their confidence and future.?

A teacher has to be full of love and compassion towards his/her students and treat them as his/her own children and work for their all round personality development. For your relief, I would like to inform you that I am withdrawing my daughter from your school and for my little happiness, my daughter, is responding well to the counsellor and now she has agreed to leave this school with the condition that she will visit all her friends once a month. I am sad to say your sarcasm won once again and another poor child lost, but hopefully after reading this letter you may consider putting a full stop, and not a comma, to incidents like this.?

(Rahul Verma is founder of the grassroots non-profit Uday Foundation, fully dedicated to?children, health and human rights.)

Tuesday, March 25, 2014

DPT causing pain in baby, is painless DPT not available anywhere in India - Pentaxim / Infanrix/ Tripacel?

Q: Dear Doctors, My 2.5 month old son is due for his second dose of DTP vaccination. We gave him DTwP {Pentavacc} the first time due to non availability of Pentaxim. But he cried a lot and had lot of pain full day. For second dose, we want to give him painless vaccine but Pentaxim is still not available. But hospitals in Mumbai do have Infanrix available. I am not aware of this vaccine, and am not able to find much info on internet either. Can I give my son Infanrix for the second dose_? Pls help me as I am very confused, and we want to give him painless vaccine only. Regards
A: Infanrix is also not available at present. It is otherwise a safe & effective vaccine made by the multinational company GSK. At present no 'painless' DPT vaccine is available in India. You need to wait or continue giving the same vaccine. The next batch may become available by mid april, 2014 as per the company representatives.

Friday, March 14, 2014

A new baby saline spray for babies - easier to use - PHYSIOMER - watch a youtube video

Now India has a European product by the name of PHYSIOMER(Mfg - Laboratoire de la Mer - France, Marketed by Modi Omega in india)  that is a 115 ml saline spray which is safe & effective for babies. It even has a you tube video demonstrating how to use it !

While Nasoclear is the most well known saline brand in the Indian market, I believe that this may be easier to use in some babies, and with the video available online, parents can actually check if they are giving it properly,
The disadvantages would be a higher price - around 300/- rupees for 115 ml bottle, and lesser availability due to recent introduction & higher price.

Monday, March 10, 2014

Can my child get recurrent episodes of Hand Foot Mouth Disease ?

Q: My 4 year old had Hand Foot Mouth Disease (HFMD) around 1 year back. Now he is having the same symptoms of rash in feet, hands and mouth ulcers again, can this disease recur?

Ans: This is certainly an unusual situation. Here is what Dr Greene has to say about this ...
Hand-foot-and-mouth syndrome is a distinct viral illness. It produces blisters in the mouth in 90% of infected children and a characteristic rash primarily on the hands, feet, or buttocks in 64% of these children. Most children are cranky, with a sore throat, decreased appetite, and/or fever. The illness typically clears within a week.
Hand-foot-and-mouth syndrome was first reported in 1956, in Australia. As far as we know, it never occurred before that time. For the next 7 years it was reported, only occasionally, in pockets dotting the globe. By 1963, however, it became a common feature of childhood worldwide.
Hand-foot-and-mouth syndrome is caused by several different viruses, including coxsackieviruses A5, A9, A10, A16, B1, B3, enterovirus 71, foot-and-mouth disease virus, and herpes simplex. The vast majority of cases, however, are caused by coxsackievirus A16.
A child with a healthy immune system will form antibodies to whichever virus caused the infection. If your son is re-exposed to the same virus, he will probably not be re-infected. He is still susceptible, in varying degrees, to the other viruses. Since 1963, most children have had one case of hand-foot-and-mouth syndrome, caused by coxsackievirus A16.
There is one other snag. While most children clear their bodies of the virus within one week, coxsackievirus A16 occasionally succeeds in hiding inside children’s own cells, like herpes. By eluding the cellular immune system, coxsackievirus A16 can cause chronic or recurring skin lesions. Healthy humoral immunity is able to keep these recurrences from being as severe as the initial episode.
Will your son catch hand-foot-and-mouth syndrome again? Probably not, but there are no guarantees. Who knows? In the next century, enterovirus 71 might become the major cause of hand-foot-and-mouth syndrome. Even so, the human immune system has a remarkable history of adapting to the ever-changing microscopic world around us.
So the answer is that your child can get it again, but if we help keep the immunity high then the chances of recurrence are less, and the severity of the disease is likely to be lesser too.

Caution to Pediatricians (& Parents) - Fake Vaccine(s) in India

This letter has been sent by the Indian Academy of Pediatrics to all members (mro9e than 20,000 pediatricians in India) counseling them regarding the availability of fake PENTAXIM (manufacturer - Sanofi Pasteur) in India. Here is the letter reproduced in its entirety

"Dear Colleagues,We would like to apprise you regarding a serious matter where a renowned vaccine manufacturer, Sanofi Pasteur has identified and come across a suspected counterfeit pentavalent vaccine, Pentaxim available in Cuttack, State of Orissa, in the eastern part of India. The vaccine pack has copied the brand but glaring errors on the pack indicate that the product is spurious. They have immediately informed concerned health authorities as well as enforcement agencies and asked them to investigate the matter and bring the culprit to book.IAP strongly condemns and expresses serious concern on the issue of availability of counterfeit products in the Indian market. We also urge our paediatric community to ensure that products are purchased through authorized distributors only through proper invoices and carefully check the packaging for any visible errors. In case of any questions and concerns, we request you to get in touch with the central IAP and the respective manufacturer to ensure that quick action is taken at the local and central level.Let us come together as a pediatric community and be vigilant to ensure we protect the health of the Indian children by using genuine products.Dr. Vijay YewaleIAP President 2014"

Friday, March 07, 2014

Off-Label Pediatric Drug Prescribing: American Academy of Pediatrics Updates Guidelines

Diedtra Henderson
February 24, 2014
Because more than half of the medications approved for marketing by the US Food and Drug Administration (FDA) lack evidence of safe and effective use in pediatric patients, the practice of medicine will "more than likely" require that practitioners prescribe medicines off-label to appropriately treat pediatric patients, according to the American Academy of Pediatrics (AAP).
The group published an updated policy statement on the topic online February 24 in Pediatrics.
The AAP last issued a statement on off-label use of prescription medicines in 2002 and reaffirmed it in 2005. Since then, more than 500 medicine label revisions have been made by the FDA to reflect information about use in pediatric patients, a trend accelerated by passage of the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA). Acting as complementary federal sticks and carrots, the PREA mandates that almost all new medicines be studied in children if pediatric use of the product is likely, whereas the BPCA opens the door for an additional 6 months of market exclusivity for sponsors that submit completed pediatric studies to the FDA.
Still, the number of pediatric-tested remedies is exceeded by the number of FDA-approved prescription medicines that have not specifically been tested for safe and efficacious use in pediatric patients.
The AAP statement indicates that clinicians' decision making in this instance "should always be guided by the best available evidence and the importance of the benefit for the individual patient. Practitioners are in agreement regarding the importance of practicing evidence-based medicine. However, for the pediatric population, gold standard clinical trials are often not available, so practitioners must rely on either less definitive information, such as expert opinion for the age group that they are treating, or use evidence from a different population to guide practice."
The FDA does not regulate the practice of medicine, according to the statement. "The administration of an approved drug for a use that is not approved by the FDA is not considered research and does not warrant special consent or review if it is deemed to be in the individual patient’s best interest," the policy statement explains.
"To conform to accepted professional standards, the off-label use of a drug should be done in good faith, in the best interest of the patient, and without fraudulent intent," according to Kathleen A. Neville, MD, from the AAP Committee on Drugs, and coauthors.
According to the statement,
  • clinicians are responsible for deciding which medicine at which dose the pediatric patient will receive for which purpose;
  • pediatricians should continue to advocate for incentives and requirements that promote the study of medicines in children;
  • physician researchers should conduct well-designed pediatric drug studies or collaborate in them;
  • journals should publish studies about well-designed trials, irrespective of the results; and
  • institutions and payers should not settle for label revisions as the sole determinant of which medicines to include in formularies.
The statement draws a distinction between individual clinician decision making on the behalf of individual patients and the active promotion of off-label use that is prohibited, whether by the sponsor or by the clinician speaking on behalf of the sponsor.
Truly investigational off-label prescriptions should be done in the context of a well-designed clinical trial, the statement indicates. Patients and their legal guardians should be duly informed, and clinicians should document consent to proceed. In addition, because off-label prescriptions can heighten liability risks for practitioners, the statement counsels clinicians to document their decision-making process in the patient's record.
"Off-label drug use remains an important public health issue, especially for infants, young children, and children with rare diseases," Dr. Neville and the AAP committee coauthors conclude. "Evidence, not label indication, remains the gold standard from which practitioners should draw when making therapeutic decisions for their patients."
The study authors have disclosed no relevant financial relationships.
Pediatrics. Published online February 24, 2014.
COMMENTS: This is an important document, since many drugs, especially cold medications that are used in children in India are also not approved below 2 years age. Pediatricians can thus continue to use some medicines off-label if they feel that the individual benefits are likely to exceed potential side-effects, and no alternatives exist.