15 Questions - Spotlight on Clinical Trials

1. My concern with some of the trials out there is the requirement that the participant quit taking their current medications and the possibility that they will receive a placebo and they may get sicker. Then there is the question, what if the medication works, but the patient can't keep taking it after the study is over.

These are very important concerns and it’s great that you’re aware of these issues before choosing to participate in a clinical trial. The best way to address your concerns is to gather as much information about the clinical trial and to then discuss that information with your family, trusted friends, physician and nurse. It is important that you raise these specific concerns with the study staff and ask what the investigator’s experience has been to date. Ask very pointedly: have some of the participants gotten sicker when they stopped taking their current medication?

Not all studies involve a placebo. A growing number involve comparing an investigational drug with a standard of care. The principal investigator and study staff can walk you through the study protocol and answer any questions that you have about comparative treatments or a placebo in the study design. Some drug companies also make special provisions to continue to provide the study medication if patients respond very well to the investigational treatment. You can ask the investigator and study staff if the sponsor has a history of supporting these types of ‘compassionate use’ and ‘expanded use’ programs.

2. My question is with regards to the safety. I am interested in participating in a study, but I'm concerned about long-term side effects. I understand that these trials are necessary to advance lupus research, but living with lupus is difficult enough without adding potential complications from the trial drug.

As scientific studies, all clinical trials carry some risk because much is not known about the investigational drug. You raise a very important question that may not have an answer at this time. The principal investigator will have an opinion about potential long-term side effects and he or she may have some information about the results of earlier trials. After you solicit their input, it’s important that you meet with trusted friends, your doctor and your family to discuss whether the potential risks of the clinical trial outweigh the potential benefits.

3. I have never questioned my safety when I a study. Meaning I know there are risks and I may not even be getting the drug being studied, but someone had mentioned “safeguards” and that there were changes put in place by the federal government years ago ensuring that people remain safe in studies. No one ever tells you what those safeguards are. What are they?

The largest safeguard is that study volunteers can withdraw from a clinical trial at any time. If you have any concerns about your safety, you should immediately speak with the principal investigator and the study coordinator to discuss your concerns and to withdraw from the trial if necessary. If you experience any adverse reaction while participating in the study, report it to the investigator and the study coordinator. Doing so raises study staff awareness of your safety and also helps them see any patterns of safety risk among all volunteers. There are many other safeguards: the principal investigator, for example, is required to report specific adverse and serious adverse reactions to the IRB and to the study sponsor who, in turn, must report them to the Food and Drug Administration and must notify other investigators participating in the study. Study monitors also periodically visit each research center to review data collected for the clinical trial. These monitors sometimes identify safety issues. Data Safety Monitoring Boards (DSMBs) are also in place at some institutions and research networks to perform interim assessments of the data. DSMBs uncover safety risks to participants in the clinical trial. There are a number of safeguards and a number of research professionals looking at your safety and that of all volunteers. It’s still important for each study volunteer to be their own self-advocate – if you have any concerns about your safety during the trial, speak up and take action if appropriate.

4. I currently participate in a trial and I feel that the consent forms have done a terrific job of informing me of all study related information including risks as well as the odds of receiving the study medication. I am receiving the medication and I have been in the trial for a number of years now. The results have been well worth the risk. The medication works better than standard treatments and has allowed me to live my life more fully again. I think it's important for people to know that they can drop out of a study at any time if they are unhappy with the results, but by participating they may have a better quality of life and help further badly needed lupus research.

Thanks for sharing your view. I find that as a community, we often tend to focus an inordinate amount of attention on the potential risks of participating in clinical trials. It’s equally important that study volunteers have an informed understanding of the potential benefits of participation.

5. I'm considering participating in a study, but I can't decide if the pros outweigh the cons.

This is a very hard decision and often one that is never completely clear. CISCRP recommends that study participants engage a network of support – your family, trusted friends, advisors and your doctor and nurse – to help discuss and think through this decision. Equally important is making sure that all of your questions are answered by the study staff. You can also ask your doctor or nurse to speak with the study staff on your behalf to provide another perspective and more detail to the potential benefits and risks.

6. I have been in a monoclonal antibody drug trial for a little over a year. I wish I had known that they would stop monitoring the labs that were specific to MY lupus situation (sed rate, CRP, muscle enzymes), and that the focus would be on my joints and mouth sores. Neither of those are a major problem for me.

Have you discussed this with the study staff and with your physician and nurse? Your clinical trial may still be the best option for you – but it is important for you to discuss your concerns with the principal investigator. You may want to explore, with their help, other clinical trials? The potential benefits and risks of a given clinical trial change over time. Study participants need to occasionally revisit their decision – with the study staff and a support network -- to see if the benefits and risks balance has shifted.

7. Where can I participate in lupus clinical trials or find more information about where to participate in my area?

There are a number of valuable resources – some that are easier to use than others. The federal government maintains a vast listing of government and industry-funded clinical research activity (www.clinicaltrials.gov); CenterWatch maintains the largest global listing of industry-funded clinical trials of medical treatments (www.centerwatch.com). CISCRP – a non-profit organization – also helps patients and their families find clinical trials. This is a free service for people who need help doing a search. CISCRP gathers the information and emails or mails it to the patient directly. Go to www.ciscrp.org and look for information about the Search Clinical Trials program.

Note: The LFA's Center for Clinical Trials Education (CCTE) is partnering with the CISCRP to provide you with a search of clinical trials in your geographic area (combining results from both www.clinicaltrials.gov and www.centerwatch.com). Please visit the CCTE Web site for more information.

8. I am currently in a clinical trial and while the study was verbally explained to me and the paperwork was daunting, until you actually start the study you have no idea what to ask. I should have asked if I would need someone to drive me. The answer should have been definitely. I was given Benadryl and that knocked me totally out then after my infusion I had a 1.5 hour drive home. I called 8 people on my way home just to stay awake while I drove. Now my dad drives me and I sleep in the passenger’s seat. 

I hear stories like this all the time. Every clinical trial has issues that don’t arise until you’re actually participating in the study. These are the realities of participation and, in some case, they result in the volunteer having to discontinue their participation. Usually a thorough review and discussion of the protocol with your support network (family, trusted friends, doctor and nurse) helps to identify some of these issues. Try to include someone in your support network who is really detail-oriented and practical to help you think through and discuss what to expect during the actual participation experience.

9. I hear a lot about compensation for time. Some of these appointments can last a whole day and I have to miss work. It seems that every study is different in terms of how they support their participants. Why is that?

You’re right. Compensation varies widely depending on many factors including: the number of visits; the length of each visit; the number and type of procedures performed at each visit. Compensation is also tied to the amount of free medical care and investigational drug that the study volunteer will be receiving. The IRB also weighs in and determines whether compensation is warranted and if the amount of compensation is excessive. All of these factors are considered by multiple parties to determine whether to offer compensation and at what amount. And, there are no hard rules about compensation in clinical trials. Usually, healthy volunteers receive compensation for their time since they are not participating with the hopes of improving a specific medical condition. The likelihood of receiving compensation for participating in a phase II or III is much more variable.

10. I am considering participating in a study but I am concerned that my care won’t be as good during the time I am in a study. Someone had said that in actuality, I would probably be getting the most comprehensive care I have ever received. I don’t understand how this is the case if the study doc isn’t my primary doctor?

In many surveys that have been conducted over the past decade, study volunteers rate the quality of care and attention received during clinical trials to be equal to, or better than, that provided by their primary physician or nurse. I believe that this is due in large part to the additional time and attention that study staff spends with each volunteer. Also, more procedures and tests are performed during a clinical trial than during regular primary care visits.

11. I have participated in several studies. Sometimes the physician goes over the consent forms with me and sometimes it is a study nurse. I never really understood why it wouldn’t be the doctor each time? Is there a reason for this? Should I be concerned that the nurse is going over this with me and not the doctor? So far it hasn’t really impacted my experience in the study later on – meaning that the physician was always available when I had questions – but should I be concerned that maybe the nurse isn’t providing the same info the doctor would?

In most cases -- in my experience - it’s more a matter of style at the research center. Interestingly, I’ve heard from some volunteers that they prefer to have a study nurse review the informed consent form because the nurse can relate more to the patient’s day-to-day concerns. Often the study nurse has spent more time each day managing the protocol and study volunteers. But your point is well taken – if you have any concerns that the information you receive from the study nurse is incomplete or different – you should ask to also meet with the principal investigator. You need to trust and be comfortable with the entire study staff.

12. While in the clinical trial will I be able to talk to others in the study? Am I allowed to provide information to my family or friends about the study?

Usually when you’re participating in a clinical trial, you’re not permitted to speak about your experience in the study with other volunteers and with family and friends. Doing so will compromise the integrity of the scientific study because it may bias your perceptions and experiences. I encourage all study volunteers to be ready to speak with family and friends during participation in a clinical trial if any concerns arise about your safety and the risks of participation. Your support network can help you think through and recalibrate your sense of the potential risks and benefits of continuing your participation.

13. I’m worried about managing the costs related to my lupus care. Do I need to let my health insurance provider know that I am in a clinical trial? Will this impact my health or life insurance benefits?

You really need to contact your health insurance provider to let them know that you’re in a trial and to discuss what impact your participation might have on your coverage. Health insurers don’t usually have standard policies for handling bills for clinical trial activities and for handling complications or side effects caused by participation. Instead, they consider inquiries and requests on a case-by-case basis. Some states have laws requiring health insurers to cover certain clinical trial-related costs. When you call, be sure to have a copy of the protocol summary and the informed consent form handy. Also, ask the study staff for their advice. They have often assisted other study volunteers dealing with these same issues.

14. If my lupus improves because of the investigational medication, can I keep using it after the study? If so, who pays for the treatments after the study is completed?

Usually study volunteers cannot continue to take the investigational treatment after the clinical trial has ended. However, there are special cases where the study sponsor will make the investigational treatment available to all or some participants depending on the safety and efficacy of the investigational treatment and the availability of other treatments. In special cases, sponsors have offered the investigational drug to participants under a discounted and subsidized level or at a market price. Even patients who were unable to participate in a clinical trial can gain access to a very promising investigational treatment. As I mentioned earlier, speak with the study staff to find out about a given sponsor company’s history with regards to compassionate use, expanded use and even per patient access to treatments that have not yet been approved by the FDA.

15. The clinical trial that I am currently in will be ending at the end of the year. What will happen to the information that has been collected on me during the trial? Who will be able to see this information?

The informed consent form typically describes how your data will be used and who will be able to see it. The principal investigator and study coordinator have access to your data; the study monitor and the sponsor are also conducting reviews and analysis of your data and that of other volunteers. The IRB may also conduct periodic reviews of study volunteer data. And, in some cases, regulatory agencies may conduct similar reviews. Just as you expect your health care provider to respect your confidentiality and privacy; you can expect the same from your study staff and other research professionals.