Posts Tagged ‘clinical trials’

Hi! and welcome to What Works Wednesdays where historically a success story from clinical files is shared. With all the buzz about the latest “research” on getting the flu while pregnant and the supposed link to autism, it seems logical to help readers better understand research so they can interpret findings themselves. If readers know how to read research, then they are better able to know if an intervention works (or if the conclusions from a study are flawed or misinterpreted).

What is Research?

  • “work undertaken systematically to increase the stock of knowledge” (Wikipedia.org)
  • “diligent and systematic inquiry or investigation into a subject in order to discover or revise facts, theories, applications, etc.” (dictionary.com)

Most scientists conduct research by utilizing the scientific method. The scientific method requires the development of a hypothesis (which is usually formed from observation or reading other research), conducting the experiment, gathering results, and determining if the results support the original hypothesis.

Different Types of Research

Using the scientific method, scientists design different types of studies. These study types include:

  1. Experiments. In experimental studies, researchers recruit participants and assign them to treatment groups. Researchers can study one or more treatments and participants may receive some treatments or they may receive a placebo or no treatment at all. Usually, researchers measure one or more important variables before the study and they measure the variable(s) again after the study.
  2. Single Subject Experimental Studies. In these studies (most often conducted by behavior analysts), researchers recruit participants who are observed and measured carefully for a period of time before receiving treatment. Researchers then implement treatment while continuing to observe and measure carefully.
  3. Correlational studies. In these studies, researchers use existing data sets (e.g., collected for some other purpose) or they recruit participants. Researchers gather a wide range of information on each participant (e.g., age, SES, education, health history). Participants do not generally receive treatments or interventions of any kind.
  4. Qualitative studies. In qualitative studies, researchers occasional recruit participants but at times they enroll participants with whom they are already familiar. In qualitative studies, researchers study one or more individuals or one or more groups (e.g., one class). Researchers carefully study the participant and take copious notes. Researchers may interview the participants and they may use focus groups to better understand some of the issues. If a treatment is provided, the researcher continues to carefully study the participants to document the participants’ responses to the treatment.

Conclusions Based on Study Type

Researchers must use caution when drawing conclusions about their studies. Researchers who use well-designed experimental designs can draw cause-effect conclusions. For example, a researcher can enroll a bunch of smokers in a study. Some of the smokers receive a behavioral treatment, some of the smokers receive nicotine patches, and other participants receive both. At the end of the study (if the researchers have conducted the study carefully), the researchers will be able to say that one or more methods is successful at helping smokers quit.

Similarly, in a single subject experimental study, researchers can demonstrate if a treatment changes behavior. Again, the study must be carefully designed and conducted but it is possible to draw cause-effect conclusions. For example, a researcher could study 3 smokers. The researcher would observe the smokers and collect data. One smoker could receive treatment. While she is being studied, the other smokers would still be studied. After the first smoker quits successfully, the next smoker would receive treatment. He would continue to be studied as would the non-treated smoker. Finally, when the last smoker receives treatment, researchers continue to observe him. If the researchers successfully help all 3 participants quit smoking (and the study is carefully designed and carried out), they will be able to say that the treatment caused the behavior change.

Correlational versus Causal

Correlational studies are designed to determine if any relationships exist between variables. Researchers could gather data on 1,000 people from an existing data base. They could sort the data into smokers and non-smokers. They could run a simple data analysis to see if smokers have other tendencies (e.g., like to go to race car events, like to drink socially, and so forth). Researchers may not conclude causal relationships from their studies. They are only able to conclude that a relationship exists. Of more importance is the strength of the relationship. For example, if researchers ran an analysis on the relationship between giving birth to a child and gender, they would find a very strong (almost perfect) relationship between giving birth and being a female. If a weak relationship exists between variables it is more likely due to chance.

Go Forth and Read

In these days of social media, spin rooms, and media crazed talk shows, very poorly designed studies are being presented to the public without appropriate interpretation of the study or its results. If you are interested in reading a few examples of this, check previous posts here and here.

In summary, don’t believe everything you read about the “latest scientific study” unless you read the study itself. When you read the actual study, what you find may actually surprise you.

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One of the greatest challenges you will face following the diagnosis of your child is determining which treatments to pursue to help your child grow and develop maximally. Many treatments are readily available for treating autism making the treatment selection decisions difficult for families.

To ensure that your child receives treatment that is most effective for him or her, it is essential that you select treatments that have been shown to be effective. Choosing a treatment that is ineffective for your child may cause your child to lose time from effective treatments in order to access the ineffective treatment. Moreover, the ineffective treatment could be detrimental to the effective treatment you are implementing. Even worse, the ineffective treatment could be harmful to your child.

Several factors should be considered prior to starting a new treatment.

  1. The treatment should have a research base to support its effectiveness.
  2. The intervention procedures should be available in a published paper.
  3. The paper describes the participants in the study as well as the methods that were used in the study.
  4. The paper describes the intervention in detail and then presents the results that were obtained from the intervention.

An intervention does not have a solid research base if it only has one published outcome paper. There is one exception to the research base rule. If you are enrolling your child in a clinical trial, the intervention most likely does not have a research base; hence the need for clinical trials. Please note that clinical trials must be approved by boards that have been established to assess the safety of clinical trial for your child. Read all the information you can about the clinical trial, including the consent form. If you have questions, the investigators will make themselves available to you in order to answer your questions.

Once you know that an intervention has been researched, make sure that the research has been published in peer-reviewed journals. Anyone can publish a paper on their personal blog or website. However, the peer-review process helps to ensure that the research is valid. The peer-review process assists researchers in accurately describing their results from the treatment. When a scientist has results that she wants to publish, she submits then to an editor. The editor sends the paper out to several other researchers who are experts in that particular area of stud. The peer-review researchers help ensure that appropriate research methods have been utilized and that adequate experimental control has been employed. If the peer-reviewers agree that the science is sound, then they recommend its publication to the editor. This entire process makes it easier for consumers to know that the research they are reading is valid and accurate.

You will know that the research has been peer-reviewed if it is published in a journal with an editor and an editorial board. Some journals and boards are more prestigious than others. This is determined by an impact factor. Typically, a higher impact factor is indicative of a more prestigious journal.

Once you have determined that the intervention has been published in peer-reviewed journals, check to see that the publications have occurred across a variety of journals. If all the research on one intervention is published in one journal, a greater likelihood exists that the research has limitations or has not been accepted by other scientists. Similarly, it is important to verify that more than one scientist has studied the intervention. This helps to ensure that the intervention outcome can be replicated across people and locations.

In the likely event that you do not have time to critically analyze treatments yourself, you may also turn to various organizations that have devoted time to determining the efficacy of treatments. Each of the entities below review a variety of treatments and treatment methodologies including biomedical intervention, behavioral therapies, medical treatments, and allied therapies.

The National Autism Center developed the National Standards Project, set out to identify the scientific evidence for autism treatments. The report was written and reviewed by a team of scientists who collaborated to reach consensus on the findings. The report may be purchased on their website for $19.95.

The New York State Department of Health established a multidisciplinary team of individuals to critically analyze a variety of treatments for children with autism.  Their findings are available on their website. A booklet of their findings is available at no charge to residents of the state of New York. The information is available for a small fee for non-residents.

Research Autism has created a web page describing the efficacy of a variety of interventions. An independent team of scientists evaluated treatments and posted their findings for the public. The information may be found on their website.

While the aforementioned reviews are comprehensive, the materials are dated and thus, do not include any recent or emerging research. As such, it is important for families to empower themselves with tools for sorting through the science.

Once you have determined that the intervention you are going to implement for you child has been well-researched, then you are ready to select your treatment provider. Carefully select your provider by asking for the degrees and credentials that are available. Ask about training specific to autism and training specific to the intervention that you are going to implement.

In summary, before starting a new therapy ask yourself:

  1. Has the treatment has been researched on children with autism?
  2. Has the research been published in peer-reviewed research journals?
  3. Has the research been replicated by a variety of people in different locations?
  4. Has the research been published in a variety of journals and not just within one publication?
  5. Has the provider had the appropriate training and credentials to provide the treatment to your child?

Good luck sorting through the science!

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