Pilot and feasibility studies US Medical PG Practice Questions and MCQs
Practice US Medical PG questions for Pilot and feasibility studies. These multiple choice questions (MCQs) cover important concepts and help you prepare for your exams.
Pilot and feasibility studies US Medical PG Question 1: A 21-year-old man presents to the office for a follow-up visit. He was recently diagnosed with type 1 diabetes mellitus after being hospitalized for diabetic ketoacidosis following a respiratory infection. He is here today to discuss treatment options available for his condition. The doctor mentions a recent study in which researchers have developed a new version of the insulin pump that appears efficacious in type 1 diabetics. They are currently comparing it to insulin injection therapy. This new pump is not yet available, but it looks very promising. At what stage of clinical trials is this current treatment most likely at?
- A. Phase 0
- B. Phase 2
- C. Phase 3 (Correct Answer)
- D. Phase 1
- E. Phase 4
Pilot and feasibility studies Explanation: ***Phase 3***
- **Phase 3 trials** involve large-scale studies comparing the new treatment to standard therapy or placebo, often across multiple centers.
- The scenario describes a "new version of the insulin pump" being compared to "insulin injection therapy," indicating a definitive comparison for efficacy and safety against existing treatments.
*Phase 0*
- **Phase 0 trials** are exploratory, small-scale studies (10-15 subjects) using micro-doses to gather preliminary data on pharmacodynamics and pharmacokinetics, not efficacy comparisons.
- They are typically conducted very early in drug development, examining if the drug behaves as expected in humans.
*Phase 2*
- **Phase 2 trials** evaluate the efficacy and further assess safety of a new treatment in a larger group of patients (tens to hundreds).
- While they assess efficacy, they usually don't involve direct comparison with an established standard therapy on the scale implied by the question, which is typically reserved for Phase 3.
*Phase 1*
- **Phase 1 trials** primarily focus on safety, dosage, and side effects in a small group of healthy volunteers or patients with the condition (20-100 subjects).
- These trials are not designed to assess a treatment's efficacy against an existing therapy.
*Phase 4*
- **Phase 4 trials** occur after a drug or device has been approved and marketed, focusing on long-term safety, effectiveness in diverse populations, and new indications.
- The described pump "is not yet available," indicating it has not reached the market and thus is not in Phase 4.
Pilot and feasibility studies US Medical PG Question 2: A physician attempts to study cirrhosis in his state. Using a registry of admitted patients over the last 10 years at the local hospital, he isolates all patients who have been diagnosed with cirrhosis. Subsequently, he contacts this group of patients, asking them to complete a survey assessing their prior exposure to alcohol use, intravenous drug abuse, blood transfusions, personal history of cancer, and other medical comorbidities. An identical survey is given to an equal number of patients in the registry who do not carry a prior diagnosis of cirrhosis. Which of the following is the study design utilized by this physician?
- A. Randomized controlled trial
- B. Case-control study (Correct Answer)
- C. Cross-sectional study
- D. Cohort study
- E. Meta-analysis
Pilot and feasibility studies Explanation: ***Case-control study***
- This study design **identifies subjects based on their outcome (cases with cirrhosis, controls without cirrhosis)** and then retrospectively investigates their past exposures.
- The physician selected patients with cirrhosis (cases) and patients without cirrhosis (controls), then assessed their prior exposures to risk factors like alcohol use and intravenous drug abuse.
*Randomized controlled trial*
- This design involves randomly assigning participants to an **intervention group** or a **control group** to assess the effect of an intervention.
- There is no intervention being tested or randomization occurring in this study; it is observational.
*Cross-sectional study*
- A cross-sectional study measures the **prevalence of disease and exposure at a single point in time** in a defined population.
- This study collects retrospective exposure data and compares two distinct groups (cases and controls), rather than assessing prevalence at one time point.
*Cohort study*
- A cohort study **follows a group of individuals over time** to see if their exposure to a risk factor is associated with the development of a disease.
- This study starts with the outcome (cirrhosis) and looks backward at exposures, which is the opposite direction of a cohort study.
*Meta-analysis*
- A meta-analysis is a statistical method that **combines the results of multiple independent studies** to produce a single, more powerful estimate of treatment effect or association.
- This is an original research study collecting new data, not a systematic review or synthesis of existing studies.
Pilot and feasibility studies US Medical PG Question 3: An epidemiologist is evaluating the efficacy of Noxbinle in preventing HCC deaths at the population level. A clinical trial shows that over 5 years, the mortality rate from HCC was 25% in the control group and 15% in patients treated with Noxbinle 100 mg daily. Based on this data, how many patients need to be treated with Noxbinle 100 mg to prevent, on average, one death from HCC?
- A. 20
- B. 73
- C. 10 (Correct Answer)
- D. 50
- E. 100
Pilot and feasibility studies Explanation: ***10***
- The **number needed to treat (NNT)** is calculated by first finding the **absolute risk reduction (ARR)**.
- **ARR** = Risk in control group - Risk in treatment group = 25% - 15% = **10%** (or 0.10).
- **NNT = 1 / ARR** = 1 / 0.10 = **10 patients**.
- This means that **10 patients must be treated with Noxbinle to prevent one death from HCC** over 5 years.
*20*
- This would result from an ARR of 5% (1/0.05 = 20), which is not supported by the data.
- May arise from miscalculating the risk difference or incorrectly halving the actual ARR.
*73*
- This value does not correspond to any standard calculation of NNT from the given mortality rates.
- May result from confusion with other epidemiological measures or calculation error.
*50*
- This would correspond to an ARR of 2% (1/0.02 = 50), which significantly underestimates the actual risk reduction.
- Could result from incorrectly calculating the difference as a proportion rather than absolute percentage points.
*100*
- This would correspond to an ARR of 1% (1/0.01 = 100), grossly underestimating the treatment benefit.
- May result from confusing ARR with relative risk reduction or other calculation errors.
Pilot and feasibility studies US Medical PG Question 4: A student health coordinator plans on leading a campus-wide HIV screening program that will be free for the entire undergraduate student body. The goal is to capture as many correct HIV diagnoses as possible with the fewest false positives. The coordinator consults with the hospital to see which tests are available to use for this program. Test A has a sensitivity of 0.92 and a specificity of 0.99. Test B has a sensitivity of 0.95 and a specificity of 0.96. Test C has a sensitivity of 0.98 and a specificity of 0.93. Which of the following testing schemes should the coordinator pursue?
- A. Test A on the entire student body followed by Test B on those who are positive
- B. Test A on the entire student body followed by Test C on those who are positive
- C. Test C on the entire student body followed by Test B on those who are positive
- D. Test C on the entire student body followed by Test A on those who are positive (Correct Answer)
- E. Test B on the entire student body followed by Test A on those who are positive
Pilot and feasibility studies Explanation: ***Test C on the entire student body followed by Test A on those who are positive***
- To "capture as many correct HIV diagnoses as possible" (maximize true positives), the initial screening test should have the **highest sensitivity**. Test C has the highest sensitivity (0.98).
- To "capture as few false positives as possible" (maximize true negatives and confirm diagnoses), the confirmatory test should have the **highest specificity**. Test A has the highest specificity (0.99).
*Test A on the entire student body followed by Test B on those who are positive*
- Starting with Test A (sensitivity 0.92) would miss more true positive cases than starting with Test C (sensitivity 0.98), failing the goal of **capturing as many cases as possible**.
- Following with Test B (specificity 0.96) would result in more false positives than following with Test A (specificity 0.99).
*Test A on the entire student body followed by Test C on those who are positive*
- This scheme would miss many true positive cases initially due to Test A's lower sensitivity compared to Test C.
- Following with Test C would introduce more false positives than necessary, as it has a lower specificity (0.93) than Test A (0.99).
*Test C on the entire student body followed by Test B on those who are positive*
- While Test C is a good initial screen for its high sensitivity, following it with Test B (specificity 0.96) is less optimal than Test A (specificity 0.99) for minimizing false positives in the confirmation step.
- This combination would therefore yield more false positives in the confirmatory stage than using Test A.
*Test B on the entire student body followed by Test A on those who are positive*
- Test B has a sensitivity of 0.95, which is lower than Test C's sensitivity of 0.98, meaning it would miss more true positive cases at the initial screening stage.
- While Test A provides excellent specificity for confirmation, the initial screening step is suboptimal for the goal of capturing as many diagnoses as possible.
Pilot and feasibility studies US Medical PG Question 5: In a randomized controlled trial studying a new treatment, the primary endpoint (mortality) occurred in 14.4% of the treatment group and 16.7% of the control group. Which of the following represents the number of patients needed to treat to save one life, based on the primary endpoint?
- A. 1/(0.144 - 0.167)
- B. 1/(0.167 - 0.144) (Correct Answer)
- C. 1/(0.300 - 0.267)
- D. 1/(0.267 - 0.300)
- E. 1/(0.136 - 0.118)
Pilot and feasibility studies Explanation: ***1/(0.167 - 0.144)***
- The **Number Needed to Treat (NNT)** is calculated as **1 / Absolute Risk Reduction (ARR)**.
- The **Absolute Risk Reduction (ARR)** is the difference between the event rate in the control group (16.7%) and the event rate in the treatment group (14.4%), which is **0.167 - 0.144**.
*1/(0.144 - 0.167)*
- This calculation represents 1 divided by the **Absolute Risk Increase**, which would be relevant if the treatment increased mortality.
- The **NNT should always be a positive value**, indicating the number of patients to treat to prevent one adverse event.
*1/(0.300 - 0.267)*
- This option uses arbitrary numbers (0.300 and 0.267) that do not correspond to the given **mortality rates** in the problem.
- It does not reflect the correct calculation for **absolute risk reduction** based on the provided data.
*1/(0.267 - 0.300)*
- This option also uses arbitrary numbers not derived from the problem's data, and it would result in a **negative value** for the denominator.
- The difference between event rates of 0.267 and 0.300 is not present in the given information for this study.
*1/(0.136 - 0.118)*
- This calculation uses arbitrary numbers (0.136 and 0.118) that are not consistent with the reported **mortality rates** of 14.4% and 16.7%.
- These values do not represent the **Absolute Risk Reduction** required for calculating NNT in this specific scenario.
Pilot and feasibility studies US Medical PG Question 6: A 28-year-old male presents to his primary care physician with complaints of intermittent abdominal pain and alternating bouts of constipation and diarrhea. His medical chart is not significant for any past medical problems or prior surgeries. He is not prescribed any current medications. Which of the following questions would be the most useful next question in eliciting further history from this patient?
- A. "Does the diarrhea typically precede the constipation, or vice-versa?"
- B. "Is the diarrhea foul-smelling?"
- C. "Please rate your abdominal pain on a scale of 1-10, with 10 being the worst pain of your life"
- D. "Are the symptoms worse in the morning or at night?"
- E. "Can you tell me more about the symptoms you have been experiencing?" (Correct Answer)
Pilot and feasibility studies Explanation: ***Can you tell me more about the symptoms you have been experiencing?***
- This **open-ended question** encourages the patient to provide a **comprehensive narrative** of their symptoms, including details about onset, frequency, duration, alleviating/aggravating factors, and associated symptoms, which is crucial for diagnosis.
- In a patient presenting with vague, intermittent symptoms like alternating constipation and diarrhea, allowing them to elaborate freely can reveal important clues that might not be captured by more targeted questions.
*Does the diarrhea typically precede the constipation, or vice-versa?*
- While knowing the sequence of symptoms can be helpful in understanding the **pattern of bowel dysfunction**, it is a very specific question that might overlook other important aspects of the patient's experience.
- It prematurely narrows the focus without first obtaining a broad understanding of the patient's overall symptomatic picture.
*Is the diarrhea foul-smelling?*
- Foul-smelling diarrhea can indicate **malabsorption** or **bacterial overgrowth**, which are important to consider in some gastrointestinal conditions.
- However, this is a **specific symptom inquiry** that should follow a more general exploration of the patient's symptoms, as it may not be relevant if other crucial details are missed.
*Please rate your abdominal pain on a scale of 1-10, with 10 being the worst pain of your life*
- Quantifying pain intensity is useful for assessing the **severity of discomfort** and monitoring changes over time.
- However, for a patient with intermittent rather than acute, severe pain, understanding the **character, location, and triggers** of the pain is often more diagnostically valuable than just a numerical rating initially.
*Are the symptoms worse in the morning or at night?*
- Diurnal variation can be relevant in certain conditions, such as inflammatory bowel diseases where nocturnal symptoms might be more concerning, or functional disorders whose symptoms might be stress-related.
- This is another **specific question** that should come after gathering a more complete initial picture of the patient's symptoms to ensure no key information is overlooked.
Pilot and feasibility studies US Medical PG Question 7: A 44-year-old woman presents to her primary care physician’s office with episodes of pain in her right hand. She says that the pain is most significant at night and awakens her from sleep numerous times. When she experiences this pain, she immediately puts her hand under warm running water or shakes her hand. She has also experienced episodes of numbness in the affected hand. Driving and extending the right arm also provoke her symptoms. She denies any trauma to the hand or associated weakness. Medical history is notable for hypothyroidism treated with levothyroxine. She works as a secretary for a law firm. On physical exam, when the patient hyperflexes her wrist, pain and paresthesia affect the first 3 digits of the right hand. Which of the following is the confirmatory diagnostic test for this patient?
- A. Magnetic resonance imaging
- B. Needle electromyography
- C. Nerve conduction studies (Correct Answer)
- D. Nerve biopsy
- E. Tinel test
Pilot and feasibility studies Explanation: ***Nerve conduction studies***
- **Nerve conduction studies (NCS)** are the most sensitive and specific diagnostic test for **carpal tunnel syndrome**, definitively confirming median nerve compression.
- They measure the speed and amplitude of electrical signals through the **median nerve** at the wrist, identifying slowed conduction across the carpal tunnel.
*Magnetic resonance imaging*
- While MRI can visualize soft tissues and nerve pathology, it is not typically the **first-line confirmatory test** for carpal tunnel syndrome due to its lower sensitivity compared to NCS.
- MRI is more useful for identifying **structural abnormalities** like tumors or synovitis, which might cause secondary nerve compression.
*Needle electromyography*
- **Electromyography (EMG)** involves inserting a needle into muscles to assess their electrical activity; it helps evaluate for **axonopathy** and muscle denervation.
- While EMG is often performed alongside NCS, it primarily assesses muscle function and nerve damage severity, rather than directly confirming nerve compression itself, which is best done by NCS.
*Nerve biopsy*
- **Nerve biopsy** is an invasive procedure generally reserved for diagnosing demyelinating or infiltrative neuropathies when less invasive tests are inconclusive.
- It carries risks and is **unnecessary** and inappropriate for diagnosing a common compressive neuropathy like carpal tunnel syndrome.
*Tinel test*
- The **Tinel test** is a clinical provocative maneuver where percussion over the median nerve at the wrist elicits tingling or pain.
- It is a **screening tool** and part of the physical exam for carpal tunnel syndrome, but it is not a confirmatory diagnostic test due to its variable sensitivity and specificity.
Pilot and feasibility studies US Medical PG Question 8: A 75-year-old man presents to the physician because of bloody urine, which has occurred several times over the past month. He has no dysuria, flank pain, nausea, or vomiting. He has no history of serious illness and takes no medications. He is a 40-pack-year smoker. The vital signs are within normal limits. Physical exam shows no abnormalities except generalized lung wheezing. The laboratory test results are as follows:
Urine
Blood 3+
RBC > 100/hpf
WBC 1–2/hpf
RBC casts Negative
Bacteria Not seen
Which of the following is the most appropriate diagnostic study at this time?
- A. Intravenous (IV) pyelography
- B. Computed tomography (CT) urogram
- C. Cystoscopy (Correct Answer)
- D. Chest X-ray
- E. Ureteroscopy
Pilot and feasibility studies Explanation: ***Cystoscopy***
- Given the patient's age, history of **40-pack-year smoking**, and **painless gross hematuria** without signs of infection or renal disease, there is a high suspicion for **bladder cancer**.
- **Cystoscopy** is the **most appropriate initial diagnostic study** because it allows **direct visualization of the bladder mucosa** and enables **immediate biopsy** of any suspicious lesions.
- The clinical presentation (painless hematuria + smoking history + absence of upper tract symptoms) strongly suggests a **bladder origin**, making cystoscopy the highest-yield diagnostic test.
- Per **AUA guidelines**, cystoscopy is essential for all patients with gross hematuria and risk factors for urothelial malignancy.
*Intravenous (IV) pyelography*
- This older imaging modality has been **largely replaced by CT urogram** due to lower sensitivity and poorer visualization of both upper and lower urinary tract structures.
- It cannot provide direct mucosal visualization or tissue diagnosis.
*Computed tomography (CT) urogram*
- A **CT urogram** is excellent for evaluating the **upper urinary tract** (kidneys, ureters) and is typically part of a complete hematuria workup.
- However, when the clinical picture strongly suggests **bladder pathology** (as in this case), **cystoscopy is the more direct and definitive diagnostic test**.
- CT urogram would be complementary imaging but cannot replace cystoscopy for evaluating the bladder mucosa and obtaining tissue diagnosis.
- In practice, both studies are often performed, but cystoscopy is the **most appropriate initial study** for suspected bladder cancer.
*Chest X-ray*
- While the patient has **wheezing** (likely related to his smoking history), a chest X-ray does not evaluate the source of **hematuria**.
- It might be useful for staging if bladder cancer is confirmed, but it is not the appropriate diagnostic study for evaluating urinary tract bleeding.
*Ureteroscopy*
- **Ureteroscopy** is indicated for evaluating and treating lesions within the **ureters or renal pelvis**, typically after imaging suggests an upper tract abnormality.
- It is more invasive than cystoscopy and is not the first-line approach when clinical features point to a **bladder source**.
- There are no signs suggesting upper tract pathology (no flank pain, no hydronephrosis).
Pilot and feasibility studies US Medical PG Question 9: A first-year medical student is conducting a summer project with his medical school's pediatrics department using adolescent IQ data from a database of 1,252 patients. He observes that the mean IQ of the dataset is 100. The standard deviation was calculated to be 10. Assuming that the values are normally distributed, approximately 87% of the measurements will fall in between which of the following limits?
- A. 85–115 (Correct Answer)
- B. 95–105
- C. 65–135
- D. 80–120
- E. 70–130
Pilot and feasibility studies Explanation: ***85–115***
- For a **normal distribution**, approximately 87% of data falls within **±1.5 standard deviations** from the mean.
- With a mean of 100 and a standard deviation of 10, the range is 100 ± (1.5 * 10) = 100 ± 15, which gives **85–115**.
*95–105*
- This range represents **±0.5 standard deviations** from the mean (100 ± 5), which covers only about 38% of the data.
- This is a much narrower range and does not encompass 87% of the observations as required.
*65–135*
- This range represents **±3.5 standard deviations** from the mean (100 ± 35), which would cover over 99.9% of the data.
- Thus, this interval is too wide for 87% of the measurements.
*80–120*
- This range represents **±2 standard deviations** from the mean (100 ± 20), which covers approximately 95% of the data.
- While a common interval, it is wider than necessary for 87% of the data.
*70–130*
- This range represents **±3 standard deviations** from the mean (100 ± 30), which covers approximately 99.7% of the data.
- This interval is significantly wider than required to capture 87% of the data.
Pilot and feasibility studies US Medical PG Question 10: A patient is in the ICU for diabetic ketoacidosis and is currently on an insulin drip. His electrolytes are being checked every hour and his potassium is notable for the following measures:
1. 5.1 mEq/L
2. 5.8 mEq/L
3. 6.1 mEq/L
4. 6.2 mEq/L
5. 5.9 mEq/L
6. 5.1 mEq/L
7. 4.0 mEq/L
8. 3.1 mEq/L
Which of the following is the median potassium value of this data set?
- A. 6.05
- B. 5.10
- C. 5.16
- D. 5.45 (Correct Answer)
- E. 3.10
Pilot and feasibility studies Explanation: ***5.45***
- To find the **median**, first arrange the potassium values in ascending order: 3.1, 4.0, 5.1, 5.1, 5.8, 5.9, 6.1, 6.2.
- Since there are **eight** (an even number) values, the median is the average of the two middle values (the 4th and 5th values): (5.1 + 5.8) / 2 = 10.9 / 2 = **5.45**.
*6.05*
- This value might be obtained by incorrectly averaging a different pair of numbers or miscalculating the average of the sorted data set.
- It is not the correct median for this particular data set of potassium values.
*5.10*
- While 5.1 is present twice in the data set, and is one of the middle values, it is not the **median** because the **median** for an even number of values is the average of the two middle numbers, not just one of them.
- This would be the median if the values were 3.1, 4.0, 5.1, 5.1, 5.1, 5.8, 5.9, 6.1.
*5.16*
- This value does not correspond to any of the numbers in the data set nor does it result from the correct calculation of the **median**.
- It might represent an incorrect average or a miscalculation of a percentile.
*3.10*
- This value is the **minimum** potassium level recorded, not the median.
- The median represents the middle value in a sorted data set, while the minimum is the lowest value.
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