Medical Heroes Newsletter
A Patient's Story
Patient perspective essential to trial design and post-trial communication
Jean Burns has learned a lot about clinical trial participation during the past decade.
After being diagnosed with Parkinson’s disease in 2003, she signed up for her first clinical trial only 6 months later. However, she did not find out about the trial from any of her many doctors. Instead, as a former website developer, she turned to the tool she knew best: her computer.
Her own research led her to the PRECEPT trial, a Phase II/III investigation to determine whether CEP-1347 was effective in treating newly diagnosed Parkinson’s patients. Despite her upsetting diagnosis, she wanted to learn everything she could about her incurable, progressive disease and she wanted to fight.
Jean joined the double-blind, placebo-controlled trial understanding that she might not get the investigational drug. "I wanted the drug," she says. "Of course I did. I hoped it would help, but for me there’s a greater good to participating in research…Even trials that do not reach their end points aren’t failures necessarily. Scientists are learning things from them, and I truly believe it is my duty to participate. The only way I can help scientists is to participate."
The medication made Jean nauseous and required many tests, but she persisted. She and a handful of other participants kept up by email and they were hopeful progress was being made. Those hopes were dashed in May 2005 when the trial was abruptly halted after interim results showed the medication wasn’t effective. A fellow participant emailed Jean with the news after stumbling across an announcement on the drug company’s web site.
"That was all," she said. "No one knew anything." Patients had no idea whether to stop their medication or how to taper off it, she says. It took weeks before any information filtered down.
Years later, while viewing a Parkinson’s Disease Foundation webinar, Jean learned that numerous scientific papers had been produced based on a follow-up study in which she had participated. Although researchers had shared their findings through scholarly papers, Jean hadn’t been notified.
Jean remains an undaunted advocate for clinical trials and says research team members have repeatedly thanked her for her participation over the years. When investigators approached Jean about participating in a follow-up observational study of Parkinson’s, she readily agreed.
The participant’s perspective should be considered and reflected at every stage, she says. Whether they’re designing a trial or communicating results after the study has ended, researchers need to consider participant’s needs and how study demands may impose hardships for them.
For example, "A lot of people with Parkinson’s are on disability," she says. "That’s not a lot of money… It’s a burden to pay for those expenses up front and then be reimbursed by the research center." Similarly, she says, researchers should factor travel expenses for care partners into their budgets because many people with Parkinson’s can’t travel alone. Participants taking part in local trials likewise may struggle with transportation and parking issues, she says, and researchers need to make it as easy as possible.
While sympathetic to researchers’ need to safeguard the integrity of data in double blind studies and to observe HIPAA requirements protecting patient privacy, Jean’s also adamant that researchers need to do a better job sharing research results with participants.
"There has to be some way we can set things up so that
patients can say, ‘Please contact me at the end of the trial and
New Medications: FDA Approval, Phase IV and Beyond
Reaching Phase IV
In a nutshell, a drug must pass through Phase I, II and III
clinical trials before it is approved by the FDA. Each phase
Getting FDA Approval
Once the first three phases of clinical trials have ended, an application is submitted to the FDA for approval to be mass produced and to become available to the public. Doctors and other medical professionals at the FDA review the results of the clinical trials, and drugs that are deemed safe and effective receive approval. The FDA has up to 10 months to review the new drug applications.
The FDA is responsible for approving both prescription and over the counter medication. Interestingly, the FDA must also approve the way in which the new medication is labeled before it can be distributed.
It is important to note that not all drugs that are approved by the FDA each year are entirely new. In fact, 40% of "new" drugs approved every year are actually pre-existing medicines that have been slightly modified to produce either more effective results or less severe side effects. Regardless of how small the modification, these drugs must follow the same journey that entirely new drugs do.
Phase IV Clinical Trials
Phase IV studies gather data from the general public, resulting in a large amount of data (as opposed to data from around 3,000 or less people in the Phase III trials). A diverse sample of people participate in the first three phases of clinical trials, but because drugs in Phase IV trials reach a greater number of people, the variety of people that it reaches also greatly increases when compared to the earlier phases. New data from Phase IV trials can reveal that the drug affects different groups of people differently, something that the data from the earlier phases may not show. Having data from a much larger pool of people allows researchers to reevaluate the safeness and effectiveness of the drug. If data from Phase IV trials suggest that the drug is actually not as beneficial as previously thought, this may result in the drug being taken off of the market.
The journey that medicine takes, from discovery to the marketplace
and beyond, is very long and challenging. Along the