Sometimes you have to lay out the risks of not innovating to affect change. What are they?

1. Becoming obsolete. Someone will pass you up. It could be a digital health company, another health system or a small mom-and-pop clinic. If it’s a small, irrelevant business line it may not matter but what if you miss out on the next big thing.

2. Accepting status quo. A health system that isn’t looking to innovate or improve will miss out on delivering the best care possible. A culture of innovation and improvement helps to protect you from providing average or below average care.

3. Losing great personnel. Innovative and creative people need freedom to innovate. They need a structure to help them execute. If you don’t have freedom or structure, frustration sets in. When frustration sets in, those personnel with great ideas will get picked off.

Health care systems can innovate and they must in order to maintain relevancy in a rapidly changing and challenging landscape

Megan and her brother Patrick battle Pompe disease, a rare neuromuscular disorder. Her father has spent nearly two decades raising money to fund research for drugs to fight Pompe. A story told in the movie, “Extraordinary Measures.”

The use of an experimental drug isn’t an uncommon practice at most major hospitals, including at Cook Children’s, including for Pompe disease.

For more than a decade, Cook Children’s has been involved with research for Pompe disease. Testing and treatment are available at the medical center. Treatment involves frequent intravenous infusions of enzyme replacement (Lumizyme). Enzyme replacement can slow the progression of the disease, as it has in many Cook Children’s patients.

Alglucosidase alfa was an approved drug indicated for use in patients with Pompe disease and marketed in the United States as Lumizyme and as Myozyme. Myozyme has been shown to improve ventilator-free survival in patients with infantile-onset Pompe disease as compared to an untreated historical control.

The drugs used for treatment for Pompe, like most of experimental medications under research at Cook Children’s, were pursued because our physicians wanted the capability to treat their patients in the best way possible. Most of the time, they weren’t opening a trial and then looking for patients to participate. They sought out the medication that would make a difference for their patients.

What is an experimental drug?

An experimental drug is a substance which may be useful to diagnose or treat disease or preserve or enhance health, but has not yet been approved by the U.S. Food and Drug Administration for the specific purpose of treating a patient.

“The drug might or might not be approved for one or a number of conditions, but it becomes ‘experimental’ when used to systematically investigate treatment for an unapproved condition,” said James Marshall, M.D., Chief Research Officer at Cook Children’s.

The importantance of 'informed consent'

The data obtained from the use of experimental drugs can be useful to future patients as doctors can see how beneficial they are the next time they are treating a patient with a similar condition.

At Cook Children’s, children participate voluntarily in all experimental drug investigations. Patients and their families, particularly in the case of minor children, must volunteer to participate in any experimental investigation through a process called informed consent.

Informed consent for children is regulated heavily by the U.S. and international governments. This type of consent is managed by a local board concerned only for the safety of research subjects, and far more comprehensive than the normal treatment consent process. Informed consent for children has several key elements:

• Education by the investigator and his/her team regarding all aspects of the research.
• Informed consent or actually signing a legal document by a minor child’s parent or legally authorized representative.
• Assent or documenting that a minor child understands and agrees to participation, and a continuous, open and welcome ongoing conversation between the participant/family and the investigational team to assure understanding through all phases of the research.

The initial consent/assent process can take several hours, and investigators never really consider the conversation to be closed. Patients and their families often learn more about their condition during the consent/assent process than they would by receiving treatment only. Patients and families can decide to stop participation even before the experimental drug is administered.

However, many patient families see the importance of participating in these experimental drugs and the benefit it may bring to not only their child, but to patients in the future.

“At Cook Children’s, we believe that all children deserve the best, evidence-based treatment strategies available to manage their illnesses and maintain good health,” Dr. Marshall said. “Cook Children’s patients have the opportunity to contribute to the health of other children through safe and ethical clinical research. We treasure the fact that clinical research is the essence of people voluntarily caring for the future of mankind.”

Related Article:

​Inspiring story sheds light on Pompe disease

More about Clinical Research at Cook Children's

At Cook Children's, we believe that all children deserve the best treatment strategies available to manage their illness and maintain good health. Our research environment applies leading-edge scientific knowledge, technology and evidence-based practice to improve quality care outcomes for children. Click here to learn more.

The 8 year old woke up in the middle of the night with a nosebleed that lasted 20 minutes. After drenching the bed and several towels, Casey, Carter’s mom, became concerned. Carter tried his best to calm his mom by saying, “I’m going to be Ok mom. It’s going to be OK.” During the nosebleed, Carter became progressively weak , seemed very pale and was complaining of vision changes like he was about to pass out.

Casey called 911, worried that something bad might happen before she could get help. Fortunately, while the first responders were on their way, the nosebleed slowed and Carter started to get back to normal but Casey still needed some reassurance that things would truly be OK.

I saw Carter soon after that night. As we talked, it became clear that, while had a history of nosebleeds, things were worsening in both how often they were occurring and how severely his nose was bleeding. We decided to keep a close eye on him and monitor when the nosebleeds were happening. Later in the week, she contacted me to let me know that his nose had bled for over an hour the previous night. During this nose bleed, he didn’t have any of the other more scary symptoms, but we both felt it was time to get him some more help.

Fortunately Natalie Roberge, M.D., one of Cook Children’s Ear Nose and Throat doctors was able to see Carter the next day. Dr. Roberge described what she discovered, “His nose bleeds were unusual because they lasted so long (up to 1-2 hours), and blood would also come out his mouth when he pinched his nose.”

The day following his appointment, Carter was scheduled for a surgery in which she “cauterized a little artery on each side of his nose to seal up the blood vessel and prevent bleeding.” He did very well with his procedure and has had no further nosebleeds since.

Nosebleeds are very common this time of year and it’s very unusual for children to require a procedure to help control nosebleeds. The blood usually comes from dry, cracked skin and fragile vessels in the nose especially in dry seasons. Even minor trauma like nose picking can cause significant bleeding.

Most nosebleeds can be controlled by applying pressure to the bridge of the nose for 5 minutes (and don’t check every 10 seconds). You can prevent nosebleeds by applying some nasal saline or Vaseline to the nose at night time, especially during dry seasons.

Dr. Roberge states that parents should “seek care for nosebleeds lasting more than 10 minutes, when the amount of blood is concerning (more than a quarter cup), more than twice a week or associated with other concerning symptoms.”