Questions & Answers to Help You Determine if Clinical Research is Right for You
These terms are often interchangeable. Clinical research is an umbrella term that has become more popular. The one element they all have in common is volunteer participants. There are two general types of clinical research; an interventional study, often referred to as a clinical trial that involves evaluating a drug, medical device or procedure and an observational study such as natural history studies and survey studies where participants are observed or certain outcomes measured, but there is no treatment given.
It’s important to have all your questions answered before you can determine if clinical research is right for you. Consider these aspects of the research:
• The length of the clinical trial
• The investigational therapy
• Potential benefits and risks
As a trial is starting a list of the clinical sites that are participating can be found on www.clinicaltrials.gov or by contacting patient advocacy groups or contacting the company that is sponsoring the research. Talking to your healthcare provider about the study can also provide an additional perspective. It’s important to know that each step in the drug development pathway is strictly governed by regulatory authorities like the FDA and the EMA to ensure the most rigorous medical safeguards are put in place for the ethical well-being of all volunteer participants.
The journey to bring a new therapy to market has phases (or stages) to ensure it is both safe and effective. Even before a therapy makes it to human testing in clinical trials, many years of animal testing and other studies have already been conducted. Traditionally, there are three phases guiding clinical research and each phase is designed to answer a specific question about the treatment. In some cases, there may be a fourth phase of clinical research.
Phase 1: How safe is the therapy by examining possible side effects or other issues?
Phase 2: Is the therapy effective in a small group of patients determining effective dosages of the investigational drug along with possible side effects and risk factors?
Phase 3: How safe and effective is the investigational in a larger number of patients?
Phase 4: What more do we need to know about the therapy over time after the drug is approved?
All volunteers must be eligible in order to participate in clinical research. Age, gender and the severity of disease are typical eligibility criteria. Eligibility criteria are developed to ensure the volunteers’ safety and that the objectives of the research can be properly evaluated. Research in rare diseases has its unique set of challenges including small patient populations that are geographically diverse. To increase participation, the FDA & EMA have worked together with clinical researchers to overcome these obstacles through the development of novel protocol designs and soliciting valuable input from the ‘patient perspective.’
If you are considering participating in clinical research, your healthcare provider is a great resource to help explain and answer your questions. Listed below are several questions to get the conversation started.
1. What are the possible benefits and risks associated with participating in clinical research?
2. Will I be guaranteed that I will be on the investigational drug?
3. When the clinical research trial is completed, can I stay on the therapy?
4. Can you help me identify the nearest study site?
5. What should I anticipate as far as travel and time away from home?
There are many trusted resources to help guide your decision making about participating in clinical research. Clinicaltrials.gov is the largest and most comprehensive clinical trials database provided by the United States National Library of Medicine at the National Institutes of Health (NIH).
Additionally, you might check out the following trusted sources:
• Rare Disease Clinical Research Network (RDCRN) is an initiative of the Office of Rare Diseases Research (ORDR) at the National Institutes of Health’s National Center for Advancing Translational Sciences (NCATS). You can view the network of 23 active consortia or research groups.
• By answering a few short questions, TrialsToday.org can help you find a list of studies that may interest you.
• Orphanet provides information on ongoing and unpublished research projects explicitly focused on a rare disease or on a group of rare diseases.