In Practice: Pros and Cons of Clinical Trials

Hundreds of major clinical research trials are currently in full swing across the United States. These studies are serving to investigate treatments and even cures for many illnesses and diseases, including major killers like cancer (402 ongoing studies, a 50 percent increase over the past seven years), heart disease and stroke (123 ongoing studies), and HIV/AIDS (83 ongoing studies).

Some clinical trials address less well-known killers. This past March, Children’s Hospital in Boston began the first-ever clinical trial for a treatment for Progeria, a fatal condition that currently affects only 42 children throughout the world - children who, around the onset of puberty, usually die of heart attacks typically brought on by advanced age.

Regardless of the size of the trial and the illness or disease it is targeting, these research studies have served to advance medical care for decades, and many have led to cures to formerly fatal conditions. Many private practices are choosing to participate in clinical trials, a move that can benefit scientific research and the lives of many patients as well as enhance your business’s bottom line. But committing to long-term involvement in clinical trials requires specialized medical knowledge, perseverance, monetary investment, and an aptitude for business. Do you have what it takes?

Clinical trials 101

Pharmaceutical companies, the federal government, academic medical centers, hospitals, foundations, volunteer groups, individual physicians, and even private citizens can sponsor clinical trials.

Clinical trials comprise the final testing a potential new drug must undergo before the drug’s sponsor submits it for approval by the FDA. Only five in 10,000 new drugs receive approval for testing on human beings in clinical trials.

There are three phases to clinical trials. In Phase I, medical scientists study the effects of the drug on the human body and explore administration techniques and dosage levels. In Phase II, the drug’s treatment efficacy on its targeted disease or illness is evaluated, as are any side effects. In Phase III, researchers compare the potential new treatment to standard treatments currently used to treat the same disease the experimental drug is targeting. If the drug’s sponsor has evidence at this point that the new treatment is viable and safe enough to be made available to the general public, the drug is submitted to the FDA for review and ultimate approval. Very, very few potential drugs make it to Phase III, given the rigor of the previous phases.

Finally, in what is sometimes referred to as “Phase IV” in clinical trials, sponsors are required to observe the effects of long-term usage of the new drug post-FDA approval. Sometimes a drug is pulled off the market after unanticipated side effects make patients seriously ill.

Most physician practices that participate in clinical trials test the drugs at Phase II or Phase III.

Trial tribulations

“There’s an appeal to doing research,” says Bill Henderson, practice administrator for Upstate Neurology Consultants in Albany, N.Y., and a speaker on clinical trials. The practice has a fully developed, separate business within it called Upstate Clinical Research. But clinical trials are undoubtedly complicated, Henderson adds, and it’s not for the instant gratification set. Have commercial drug developers told you that you’ll double or even triple your income by conducting clinical research? That’s not true, says Henderson. Why? Because it’s difficult both to get and stay involved in such a complex endeavor.

Consider these figures that Henderson has collected: In 2004, 21,735 investigators conducted clinical trials in the United States. Of these, roughly 2,200 (or 10 percent) constituted a “veteran” core group. Only 65 percent of 2004’s investigators had conducted trials the year before. The remainder are newbies, indicating a huge annual turnover.

Many physicians are searching for ways to add revenue to their practices quickly. When this doesn’t happen or if unanticipated hassles arise, Henderson says most practices abandon the initiative. The main reason for low physician re-enlistment is failure to plan for a long-term commitment, he says. The clinical trials business is anything but a get-rich-quick scheme. To realize a profit, you need to be in it for the long haul.

And as with any business startup, there’s risk. It typically requires a sizeable financial investment. You must set up space to provide the new service, stock it with appropriate supplies, and hire qualified staff. That investment may not be recouped for years. And don’t count on timely payment for your services - Henderson says the typical turnaround time on clinical-trial related invoices is six to eight months. “This means you will have to float salaries, etc., until you get rolling,” Henderson emphasizes. “Even if you are committed, it’ll take three years to make a profit. Many doctors don’t have the patience to wait it out.”

Daunted yet? There are other variables that can also lay the ground for your success or failure in clinical trials. Do you practice in a desirable geographic location in relation to the specific trials you will be conducting? Do you have a solid relationship with your lending institution? And how will you let people know about your new offerings? Do you have the marketing dollars?

If you have done your homework and think your practice is a good candidate for the execution of clinical trials, there’s also the matter of convincing a sponsor to utilize your services. Breaking into the field is rather Catch-22-ish, as sponsoring organizations prefer to use experienced investigators and sites that have performed well in the past. “You don’t just open the door and [the opportunities] start pouring in,” says William Huggins, a gynecologist from Birmingham-based Alabama Women’s Specialists, which is currently taking part in a clinical trial testing a drug formulated to stimulate female sexual arousal. (One factor that may help you break into the business: having an unusual or very specific subspecialty.)

Finally, count on the government to make things as complicated as possible. “There are huge regulatory hoops, vast amounts of paperwork,” says Huggins. “The government oversees these things in great detail.”

And you’re not the only one taking risks. What about your patients?

Anyone who enrolls in a clinical trial is necessarily taking on a certain amount of risk - thus the term “trial.” As a conductor of clinical trials, you must realize that any treatment you test may be ineffective or even detrimental to your patients. This could put them behind on what may be crucial treatment timelines with standard treatments. The experimental drug may cause as-yet-unknown side effects - the discovery of which, of course, is one of the primary reasons for running a trial - that may be uncomfortable, painful, or even life-threatening. Or your patients may end up in control groups and be assigned to take placebos, as most trials are double-blind. Finally, patients are personally responsible to keep follow-up visits, self-monitor any changes, and cover any possible travel costs. Of course, all of these risks and contingencies are carefully explained to patients before they voluntarily enroll in a trial, and many of them are more than happy to assume such risks in their hope for a cure or relief from painful symptoms. Nevertheless, they are your patients. Make sure you and your staff can personally deal with any of the above contingencies.

So why do it?

Before you drop the idea entirely, you should know there are positive aspects to conducting clinical trials in your practice. If you are realistic in your expectations and methodical in your execution, clinical trials may greatly enhance your bottom line. Upstate Neurology has been conducting clinical trials since 1988. Its six neurologists and appropriate facilities make clinical trials a lucrative endeavor. Mostellar Medical Clinic in Bayou La Batre, Ala., is a 21-physician practice that has been successfully conducting clinical trials since 2001, says Heidi Beckham, an RN who specializes in coordinating the practice’s trials.

And unlike regular patient-care fee schedules, clinical trials are exempt from such prohibitive federal regulations. This can be good for your bottom line.

And don’t underestimate the personal fulfillment that comes from helping to find cures to devastating illnesses that claim thousands of lives each year. Your patients may be among the first people in the county to receive more efficacious treatment for their illnesses. They’ll also enjoy close physician monitoring, helping them feel more in control of their personal healthcare decisions.

Being current on the latest research and treatments might be enough of a pull for you. “There’s certainly a lot going on and a lot to be learned,” Henderson says. If you stay in the know, so will your patients. Beckham agrees, saying that another major reason her practice started conducting trials is “to try these drugs out to help future patients with these problems.”

Planning is everything

As when you make any significant change or addition to your practice, planning and preparing as thoroughly as possible will help you avoid a disappointing or disastrous outcome. Doing the following will serve you well:

Educate yourself. Cull as much information as possible from reputable sources when establishing clinical research facilities in your practice.

Create a separate entity from your practice that performs the research. The importance of this step cannot be stressed enough. Of course, you’ll want to consult an attorney on the legalities of your new enterprise. And don’t forget that new business necessitates additional office space. Although clinical research facilities essentially resemble typical practice offices, there are crucial differences. For example, you’ll have to designate specific areas in which clinical research associates from sponsoring organizations can audit your collected data.

Hire the right staff. No, you can’t just use your current RN; she already has enough to do. This happened to Beckham when Mostellar Medical Center first kicked off its clinical research component. “I used to have a different title of staff nurse; that was difficult,” she recalls. “I had too much to do. It’s better to have a dedicated person to do the job.”

If one of your nurses expresses interest in switching to the clinical research component of your practice, that’s fine, but you’ll have to hire a replacement for her and then fund her training to become a CCRC, or certified clinical research coordinator. CCRCs typically earn at least $50,000. You may require a significant number of additional staff members, says Henderson, particularly “someone to look after regulatory submissions. Generally, this is not a healthcare person.” Depending on the volume of research you perform, you may also need data, site, and project managers and quality assurance experts.

Advertise. Don’t worry; this isn’t the horrid ambulance-chasing kind. But you do have to fill your research slots, so you’ll want to train your staff to be able to effectively use TV, print, radio, and online media to raise awareness about the specific trials you are conducting.

Fill your research panel slots properly. Because you will not be able to draw solely from your own patient base, at least half of your research subjects will have to come from outside your practice. “The drug companies have lists of what they want in their trials called inclusion and exclusion criteria,” says Henderson. “Just because you have a condition does not mean you’ll qualify for the trial.” And locating appropriate and willing subjects can be challenging. “One-third of all investigations will enroll no subjects due to inclusion or exclusion criteria,” says Henderson. “For one particular condition, we had 700 people respond; 10 people qualified.”

But you won’t be filling one study’s entire patient panel. A sponsoring organization ensures adequate enrollment by distributing the total number of subjects needed for any trial across many research sites. For example, Mostellar Medical Center was just one of roughly 800 sites involved in the diabetes trial it recently completed.

The best motivation

While conducting clinical trials is certainly complicated and fraught with risk, there are solid reasons to explore the possibility of incorporating research into your practice. “When you get further on in your career, and maybe want to back off on how many patients you see, it might be a lucrative thing to get into,” suggests Huggins.

And remember that today’s standard treatments are yesterday’s clinical trials. Research supervisor Kim Henderson at The Children’s Clinic in Jonesboro, Ark., says vaccinating children against diseases to which they were previously vulnerable makes the hard work well worth it. “Children are the future,” she says. “It’s wonderful to be a part of it when we see a new vaccine come out on the market that we helped do the research for.”

Shirley Grace, senior writer for Physicians Practice, holds an MA in nonfiction writing from The Johns Hopkins University. Her articles have appeared in The Washington Post and Notre Dame Business magazine. She can be reached at sgrace@physicianspractice.com.

This article originally appeared in the May 2007 issue of Physicians Practice.