New treatments for people with spinal cord injuries can take years to develop. Approving a drug or a treatment before it has been tested extensively can have horrific, even deadly, consequences. To protect patients and participants in research studies, the United States closely regulates the development of drugs and treatment devices and requires that they undergo extensive research before they can be used therapeutically.
Clinical Trials are a series of research studies that are required before a new drug or treatment can be marketed to the public. The clinical trials are a costly, time consuming, and rigorous process that follow specific protocols to ensure the safety of the participants. Studies that are conducted without this extensive research and review can prove to be detrimental to the participants in the study. Before a clinical trial receives approval to begin, researchers must provide the Food and Drug Administration (FDA) and the Institutional Review Boards (IRB) with specific research data to support the need for the clinical trials. It can take several years before the idea for a new drug or treatment reaches the first phase of a clinical trial and can be tested on humans, then several more years before it can be approved and regulated for sale to the public. There is no exact way to predict how long a particular clinical trial will last. There are several reasons for the lengthy process of clinical trials, especially those for spinal cord injury. Three of the top reasons are funding, recruitment of participants, and classification of participants in the studies.
- Funding: Clinical trials are generally funded by the Government, Foundations, and private donations, all have which have been adversely affected by the economy. Pharmaceutical companies also fund studies, but often their motives are based on profitability. Because the population of people with spinal cord injuries is relatively small, they often look to fund studies that will benefit both the SCI population and other populations with nerve damage/disease (i.e. ALS or Lou Gehrig’s Disease).
- Recruitment of Participants: It can be difficult to recruit participants for studies as the population of people in the US with spinal cord injuries is relatively small when compared to other disabilities or diseases such as Parkinson’s disease and cancer.
- Classification of Participants in the Studies: The classification of spinal cord injuries is complex- they can be described by the type of injury, the location, sensory and motor involvement, and complete vs. incomplete injuries. Other factors that come into play when assigning participants to a particular research project can include age at onset of injury, the time that has passed since the injury, and other medical conditions and secondary complications that may hinder recovery.
Each clinical trial is conducted in individual steps, or phases. Each phase is designed to answer a specific research question. When each phase is completed, researchers analyze the collected data and formulate a new research question, or hypotheses, for the next phase. In order for the clinical trial to continue into the next phase, approval must be received from the FDA. As additional information is collected during each phase, the benefits and risks of the study are re-evaluated. The IRB, also called Ethical Review Boards, monitors and approves each step of the clinical trial to protect the rights and welfare of the study participants.
The Clinical Trials are generally conducted in 4 phases:
Phase I/Pilot Study: The safety, effectiveness, and side effects of a drug or device are evaluated. Usually this phase involves less than 100 participants and takes a few months to several years to complete.
Phase II: The drug or devices are further evaluated on a larger group of participants. More information is collected about the effectiveness of the treatment. Often comparative studies are completed in this phase. In this type of study, ½ of the participants receive the treatment and the other ½ does not. The two groups are compared to see if there is a different outcome in the group that received the treatment versus the group that did not. There may be several “branches” in this phase, as researchers want to compare similar groups. For example, one group may have complete injuries, another may have incomplete injuries, a third group may have paraplegia, and a fourth group may have quadriplegia. This phase, often called a Therapeutic Exploratory Phase, can last for years.
Phase III: More participants are recruited as the treatment is further evaluated to confirm its effectiveness. Thousands of participants can be involved in this phase of the study, and it can be difficult to recruit this many people with spinal cord injuries to participate. More information about the effectiveness, side effects, risks and benefits can be collected from the large group of participants. This phase can also last for years.
Phase IV: Drugs or devices that were evaluated successfully in the previous phases can be marketed for clinical use. Post-marketing studies continue to evaluate the effectiveness of the drug or device as compared to other available treatments.
If you are considering participating in a research study, it is important that you understand the risks and benefits associated with the study and that you participate only in studies that are stringently monitored. For more information about clinical studies, please refer to http://clinicaltrials.gov/ct2/info/understand and for specific information about clinical studies involving people with spinal cord injuries please visit:
If you are unable to participate in a study but want to support spinal cord injury research, you can lobby for increased funding from the government to allow new studies to begin and to support the studies that are already in progress.