Use this as a reference to check your answers based on the videos part of the Patient Advocate Program.

Patient-Centered Outcomes Research Answer Key

1. How is patient-centered outcomes research different from other kinds of health research?

It focuses on questions that the patient community cares about. It compares health options. The process itself involves patients as partners.

2. What are the three main areas of patient involvement?

Planning, Implementation, and Dissemination.

3. For each of these areas, describe one example of how patients can make a difference.

Planning Research

  • Developing the research question and relevant outcomes to be studied
  • Defining the characteristics of study participants
  • Drafting or revising study materials and protocols 

Conducting Research

  • Drafting or revising study materials and protocols
  • Participating in recruitment of study participants
  • Participating in data collection and data analysis
  • Participating in the evaluation of patient and stakeholder engagement
  • Serving as a patient representative on a Data Safety Monitoring Board 

Dissemination

  • Identifying partner organizations for dissemination
  • Planning dissemination in the context of shaping study design and protocol
  • Authoring manuscripts and presenting study findings
  • Identifying opportunities to share information about the study, even as it is in progress

4. What is the difference between efficacy trials and effectiveness or pragmatic trials?

Efficacy trials determine if an intervention works in ideal circumstances, while effectiveness trials or pragmatic trials are designed to see if it works in the real-world settings.

5. What three factors are essential when designing a research question?

  1. The people (population of interest)
  2. The options (and the factors to consider when choosing between/among options)
  3. The outcome (what the research is intended to inform)

6. Is there any way a patient voice in research might not be helpful? What do we need to keep in mind when exercising our voice?

Your thoughts

"Gluten Exposed: What Is It All About?" Answer Key

1. Why might testing the general population for these antibodies be a problem?

The test would often be inaccurate. The positive predictive value of a test depends upon what the population prevalence is. If you do this test, it’s only going to be right 30% of the time. If it’s a high-risk group, such as someone with diarrhea or a family member, it is right about 80% of the time. They’re skewed not to miss anyone.

2. Dr. Green listed some known risk factors for celiac disease. What questions might you ask him to better understand how to evaluate his statements?

Sample Answers: For these risk factors, do we know anything about causation; for example, do we know if people with a propensity toward celiac disease have more cesarean sections because of something about celiac disease? Or is it that people who have cesareans somehow become more susceptible to developing celiac disease because of something about the cesarean?

3. About what percent of the population is at risk for celiac disease?

Forty (40) percent of us have the genes and 99% or more of the population are eating gluten.

4. Which of the following are risk factors for developing celiac disease?

  1. Being born in the summer in the northern hemisphere
  2. Lack of breastfeeding
  3. Introducing gluten to children between 4 and 6 months
  4. Use of iron supplements in pregnancy
  5. Delaying the ingestion of gluten in children until after 12 months
  6. Being born by cesarean section

1, 4, 6

5. About 1% of the U.S. population has celiac disease. What percentage of these people has been diagnosed with the disease?

17% have been diagnosed with celiac disease.

6. If you receive one positive test of transglutaminase antibody, what do current guidelines recommend you do next

Repeat the test. The presence of one positive tissue transglutaminase antibody is not enough to put people on a gluten-free diet their entire life.

7: True or False: Studies of world-class athletes from North America, USA, Canada, and Australia have shown that, even for athletes who have not been diagnosed with celiac disease, a gluten-free diet improved performance.

False.

"Celiac Genetic Testing" Answer Key

1. Why is the antibody test only useful if you are ingesting gluten?

The inflammation may be healed and the celiac antibodies may have disappeared if you are not ingesting gluten.  If you do not want to do a gluten challenge, your other option is celiac genetic testing.

2. What does the celiac genetic test detect?

It tests for certain variants of the HLA genes DQA and DQB. These are found on Chromosome 6, which is involved in our immune system and recognizing self and non-self-molecules.

3. Describe what you learned about the newly developed stool test.

There is a test that’s being developed that detects the gluten immunogenic peptides—they’re called GIPs—that are left in stool samples after digesting of gliadin. The test determines the level of these GIPs, so it’s a way to monitor whether gluten is still being eaten.
The test detects very small amounts of gluten and studies have shown that 15% to 30% of patients—celiac patients in the studies on a self-reported celiac gluten-free diet—still have detectable amounts.

True or False: DQA and DQB are HLA DQ genes specific to celiac?

True. There are hundreds of variants of these two genes. Of these hundreds, two variants, DQ2 and DQ8, have alpha and beta chains on the surface of the immune cells that recognize and create an immune-mediated response.

5. What do DQ2 and DQ8 recognize that triggers celiac disease?

Gliadin from gluten.

6. True or False: It is possible to inherit celiac disease from only one parent?

True. Which gene variant? DQ2.

7. What proportion of the general population carries DQ2 or DQ8? How many of them will develop celiac disease?

30% and 3%.

8. True or False: Among people with celiac disease, more than 90% carry DQ2; approximately 2% to 10% carry DQ8, and the remainder carry only half of the DQ2 heterodimer.

True. HLA variants relevant to celiac include DQ2 with both DQA1 05:01 or 05:05 AND DQB1 0201 or 0202. DQ8 is created if you have DQB1 03:02 AND DQA1 03.

9. Is having both DQ2 and DQ8 a greater risk factor for developing celiac disease than having only one?

Yes.

10. Does a negative HLA genetic test rule out celiac disease?

Yes, it is 99.9% accurate and only needed once in a lifetime.

11. If you have a positive HLA genetic test, but do not show symptoms of celiac disease, should you get regular antibody screening?

Yes. Celiac disease is very complex and can develop at various life stages.

"Introduction to Drug Development" Answer Key

1. How many clinical phases are there in therapeutics development? Four. What does each phase test?

Phase 1: Safety and tolerability, dose escalation
Phase 2: Safety and Efficacy in the target population, proof of principle
Phase 3: Pivotal, registrational studies that confirm efficacy and proven treatment effect, safety in large numbers
Phase 4: Post-marketing studies, safety surveillance through long-term safety assessments for large numbers of patients, use drug and disease-specific patient registries

2. What types of studies are considered the “gold standard?” Why?

Randomized, placebo controlled studies because they provide the highest standard of evidence.

3. What is Epidemiology?

A clinical research method that characterizes the rates, incidence, and prevalence of the disease.

4. An Efficacy Trial compares what?

Baseline characteristics to outcomes in a sample of patients.

Dr. Adelman argues that the bar is getting higher for the development of therapeutics. Why does he believe that?

Drugs not only need to treat the disease, they need to be proven to be better than existing therapies already on the market. Some believe the bar is too high, while others believe it is too low.

6. List as many reasons as you can for why the drug process is slow. Which of these reasons protect the interests of patients?

Your thoughts

7. If patients are fully engaged in a research project might they offer input that could make the research process less expensive and/or more efficient? Is this possible without jeopardizing patient safety?

Your thoughts

"Gluten-Free Diet" Answer Key

1. What does the term gluten-free mean?

It means the product is less than 20-parts per million of gluten.

2. When the U.S. Food and Drug Administration surveyed food products labeled “gluten free,” what percentage met that definition?

Almost 99% of the products on the market indeed fell below 20-parts per million of gluten.

3. What do additional specific certifications ensure?

Not much. Currently, research has found no significant difference in the gluten-free nature of products, whether they were just labeled gluten-free or they had a specific certification. If you are looking for less than 20-parts per million – such as less than 5-parts per million – then special certification might be useful.

4. Generally speaking, which of the following foods are safe for celiac disease patients?

  • Malt: No
  • Yeast extract: Yes
  • Distilled vinegar: Yes
  • Maltose: Yes
  • Malt extract: No
  • Alcohol – Distilled liquor and wine: yes; beer or cider only if these contain gluten-free ingredients; alcoholic beverages that say processed to remove gluten: No
  • Maltodextrin: Yes
  • Caramel colors: Yes
  • Malt syrup: No
  • Natural flavors: Yes
  • Dextrin: Yes
  • Malt flavor: No

5. When a label warns that a product was made in a facility that also processes gluten, what does this mean?

Not much. This is a voluntarily label – the presence of such a statement does not mean it contains allergen, and the absence of a statement does not mean it is in a dedicated facility. The FDA found that products that contain oats, and some other products such as cereals, were actually more likely to be contaminated if they had this warning.

6. About how much gluten does it take to damage the villi of most people with celiac disease?

About an eighth of a teaspoon of wheat flour contains enough gluten to damage the villi of most people with celiac disease. There are a portion of people with celiac who cannot even tolerate this much, but the vast majority that can tolerate this amount is how the 20-parts per million was determined.

7. What is one question you can ask at a restaurant that will provide you with a lot of great information?

“How do you prevent cross-contact with your gluten-containing menu items?”

8. Can you absorb gluten through your skin?

No, you cannot absorb it through your skin.