8 steps to succeed at a risky medical “first”

James Lock, MD, Cardiologist-in-Chief, Children's Hospital Boston

Ever since second grade, when he was suspended from school, Jim Lock has challenged the status quo — in sixth grade pulling off the much harder feat of being expelled. Today, the Cardiologist-in-Chief at Children’s Hospital Boston is credited with a long list of pioneering cardiac procedures going back to 1979, many of them in children with congenital heart defects. More recently, these have included fetal interventions, like reversal of hypoplastic left heart syndrome and implanting a stent in a fetal heart. Nearly all are now established procedures in pediatric cardiology.

These procedural innovations didn’t arise from a rare flash of inspiration, Lock argues, but from perception of a medical need followed by methodical vetting and sometimes years of due diligence.

“Innovation is not a completely random event,” Lock told the Innovators’ Forum at Children’s last week, sponsored by the Innovation Acceleration Program.  “It’s a process.”

Here’s Lock’s instruction set for safe and successful clinical firsts:

#1  Review the literature thoroughly. Some of the most valuable insights come from the oldest papers, and from papers not directly related to the topic. “Unless you’ve looked at 50 papers, you haven’t really scratched the surface,” says Lock.  When he and his colleagues did the first transcatheter closure of a ventricular septal defect in 1987, using a double umbrella to close a hole in the heart, they were informed by a decades-old report about catheter closure of a patent ductus arteriosus. A 1953 paper from Mexico City on a pulmonary valve procedure helped Lock’s team avoid rupture after placement of a stent in the right ventricle–to–pulmonary artery conduit.

#2  Create animal models. This can be difficult, but it’s very important for a proper test of the procedure. The right disease model will result in a superior device. In developing a multibasket catheter to map electrical activity in the atrial septum, Lock and colleagues figured out a way to create atrial arrhythmia in an animal model purely by accident – then went back and showed that the method was reliably reproducible. Animal models should take into account size, anatomy and physiology (certain animals, for example, develop blood clots much more readily than humans).

#3  Consult widely. Tap multiple colleagues. As a pediatric interventional cardiologist, Lock routinely consults with high-risk obstetricians, surgeons, echocardiographers and anesthesiologists. Go find the experts. “Have a high level of tolerance for ideas that aren’t your own,” Lock advises.

#4  Make a list and choose. List all possible approaches to your problem, then vet them. In developing the fetal procedure for correcting hypoplastic left heart syndrome, involving perforation of the atrial septum, Lock’s team considered different options for catheter access and entry to the fetal heart, different imaging methods, different kinds of catheters, whether or not to open the mother’s abdomen, whether the mother should be awake, and different techniques for rotating the baby in utero. “It is a mistake to come in with only one option,” Lock says.

#5  Pick your first patient carefully. Jesse Gelsinger, whose infamous 1999 death in a gene therapy trial jeopardized an entire field, exemplifies poor patient selection: His genetic disease was largely controlled with treatment, whereas the ideal patient has a very bad medical problem with few if any treatment options. Family and colleagues should strongly support trying the procedure. (Some of Lock’s catheterization “firsts” were in patients who wanted to avoid surgery.) The patient’s procedure should be as technically straightforward as possible, and use existing tools – if you succeed, tools can be modified later. “You will not get device companies to make something for you if you’re doing it for the first time,” Lock says.

#6  Use the peer review process to get regulatory approval to try the new technique or device. “Otherwise, you aren’t going to be able to do it a second, third or fourth time,” says Lock. In a trial of transcatheter closure of high-risk holes in the heart with the clamshell/Cardioseal device, an uninvolved cardiologist and surgeon agreed that each procedure was in the patient’s best interest.

#7  Conduct thorough mock drills. This will help you prevent complications and be ready to deal with any that might arise. What if a clot forms? What if a vessel ruptures? “Spend the week before thinking of all the mistakes you could make,” Lock says.

#8  Succeed.  All eyes will be on you. There’s an enormous premium on your first procedure being successful. Don’t publish case reports – wait until you have a string of successes before writing them up.  And be sure to share credit with your colleagues.

A question from the audience: What if you work in a clinical department that’s not innovative? “Seek your own experts,” counsels Lock. “There will be champions.”

If these steps are rigorously followed, first-time procedures can be safe and successful – and should be tried if they will solve a medical problem. “In the current era, being afraid of change is a catastrophe,” Lock says. “We’ve tried to create an environment where a good idea is supported until it’s proven to fail.”