Without clinical trials, we would make no progress in the treatment of Idiopathic Pulmonary Fibrosis. Only through properly conducted scientifically designed clinical trials can we advance the care of IPF patients. After 30 years of negative studies, clinical trials have finally yielded two treatments that are FDA approved to treat IPF. Esbriet (pirfenidone) and OFEV (Nintedanib) are the first treatments that improve outcomes in IPF. They both slow the decline in lung function that accompanies IPF.
Why participate in clinical trials?
Patients benefit from clinical trials in several ways. First, when participating in a clinical trial, a research team monitors patients particularly carefully and comprehensively. Second, patients benefit from potential early access to the newest therapies. Third, even when not receiving the active treatment arm (patients that are randomized to placebo) patients generally do better than when followed outside of research. This may relate to a placebo effect or be related to the meticulous attention from the research team.
What are the risks of participating in clinical research?
The risks depend on the study type. There are several different types of research studies.
1. Placebo controlled double blind studies.
In this type of study, patients are randomized to either active therapy with the study drug or a placebo (sugar pill or sham medication). Neither the patient nor the study team knows which patients are receiving which intervention. This rigorous design prevents patients and their study team from influencing the outcomes of the study (using scientific terms, this removes the possibility of bias).
Risks: a new medication of unproven efficacy and safety is under study. Patients may experience adverse events from the medication or from the study procedures.
Advantages: Patients get early access to potentially effective medications years before the medication would be approved. For a progressive and fatal disease this can be life-prolonging or life saving.
2. Open label studies.
In this type of study, patients and their research team know that the patient is receiving the active intervention. Generally open label studies follow a placebo controlled double blind study as an extension period. Certain types of interventions may also be studied using an open label design.
Risks: if the medication is not yet approved, then patients face all of the risks of exposure to a medication or intervention that is of unproven efficacy and safety.
Advantages: patients are often frustrated by the possibility of getting a placebo (not being randomized to the active treatment arm). There is no placebo group so all patients get the active intervention.
3. Registry studies.
This type of study gathers information about patients. There is no specific intervention. Patients are followed forward in time and the study team contacts patients at regular intervals and collects information relevant to the their disease process. For example, in IPF, the registry will gather pulmonary function test results, symptoms and information related to any hospitalizations or new symptoms.
Risks: there are no major risks to participating in a registry study. Patients spend their time periodically either coming into the office or talking on the phone with the study team. No additional procedures or medications are used that would not be used under regular clinical practice.
Why have a placebo group?
Imagine a research study for a new medication — let’s call the new drug BigPromise. The drug company that is developing BigPromise wants to do a study as an open label study without a placebo group and without blinding (patient and doctor know their assignments). The primary outcome of the study is how patients feel about their breathing and how far they walk. 100 patients enroll. Baseline measurement of breathing symptoms and walk distance are measured and then again at the end of three months. 60% (60 patients) feel better and walk an average of 25 yards further on their walk test. BigPromise is hailed as a huge success. However, the FDA requires a second study that is randomized and double blinded. A second study of 100 patients is undertaken. This time 50% of patients receive a placebo and 50% receive the study drug BigPromise. The same endpoints are used (breathing symptoms and walk distance). After three months, patients in the BigPromise active treatment arm show the same benefit as they did in the earlier open label study. The placebo group however, also shows improvement in their breathing symptoms and a 25 yard increase in their walk test. With the same test results, it is clear that BigPromise failed to deliver.
How do I find a clinical trial for pulmonary fibrosis?
There are several approaches.
1. Ask your pulmonologist.
2. Look on the National Institutes of Health Web site Clinicaltrials.gov. This web site allows you to search by disease, medication and many other parameters.
3. Attend a support group and talk to other patients.
4. Search on the web for clinical trials. The pulmonary fibrosis foundation and Coalition for Pulmonary Fibrosis are good resources.
Beware of fake science and scammers.
1. All bona fide research studies will be registered with clinicaltrials.gov.
2. You will never have to pay to participate in a research study!! If you are being asked to pay, then you are being scammed.
3. If it sounds too good to be true, then it’s a scam
4. All clinical research studies have the same process. Patients are given an informed consent that details the risks of participating. There is an independent organization called an IRB (Institutional Review Board) that ensures patient safety is the first priority. If these processes are not followed, then you are not participating in a true research study.