Basket trials represent a paradigm shift in precision oncology, moving beyond organ-specific treatment to a “tumor-agnostic” approach. These contemporary trial designs evaluate a single targeted therapy across multiple diseases that all share a common molecular driver. For PharmD students, understanding the basket trial design is crucial for interpreting the evidence that leads to landmark approvals based on a biomarker rather than a specific cancer type.
1. What is the defining characteristic of a basket trial design in precision medicine?
- It tests a single targeted therapy in patients who have different types of diseases but share a common molecular alteration.
- It tests multiple targeted therapies in patients who all have the same type of disease.
- It tests a single drug against a placebo in a broad, unselected patient population.
- It focuses on dose-finding and safety in a small group of healthy volunteers.
Answer: It tests a single targeted therapy in patients who have different types of diseases but share a common molecular alteration.
2. A basket trial is designed to test the efficacy of one drug across multiple:
- Dosing schedules
- Biomarkers
- Disease types or histologies
- Age groups
Answer: Disease types or histologies
3. What unifying factor links all patients enrolled in a basket trial, regardless of their cancer type?
- They are all treated at the same hospital.
- They all have the same stage of cancer.
- They all share a specific genetic mutation or biomarker that the study drug targets.
- They have all failed the same previous chemotherapy regimen.
Answer: They all share a specific genetic mutation or biomarker that the study drug targets.
4. How does a basket trial differ from an umbrella trial?
- Basket trials test one drug in many diseases; umbrella trials test many drugs in one disease.
- Basket trials test many drugs in one disease; umbrella trials test one drug in many diseases.
- Basket trials do not use biomarkers for patient selection.
- There is no difference; the terms are interchangeable.
Answer: Basket trials test one drug in many diseases; umbrella trials test many drugs in one disease.
5. A major advantage of the basket trial design is its efficiency in studying:
- A single, common type of cancer.
- The effect of a single drug on a rare mutation found across many different cancer types.
- The safety of a new drug in healthy volunteers.
- The interaction between two different experimental drugs.
Answer: The effect of a single drug on a rare mutation found across many different cancer types.
6. The concept of a “tumor-agnostic” approval, where a drug is approved for use in any solid tumor with a specific biomarker, is a direct outcome of which trial design?
- Traditional randomized controlled trials
- Basket trials
- Dose-escalation trials
- Phase 4 post-marketing trials
Answer: Basket trials
7. A clinical trial enrolls patients with lung cancer, colon cancer, and melanoma, but only if they all have a BRAF V600E mutation, to test a new BRAF inhibitor. This is an example of a(n):
- Umbrella trial
- Basket trial
- Bioequivalence trial
- Observational study
Answer: Basket trial
8. Each different cancer type within a basket trial is referred to as a separate:
- Umbrella
- Cohort or “basket”
- Placebo group
- Phase
Answer: Cohort or “basket”
9. A potential challenge in interpreting the results of a basket trial is that:
- The targeted therapy may show different levels of efficacy in different tumor types (baskets), even with the same mutation.
- The trial design is too simple for statistical analysis.
- It is impossible to find patients with the same mutation in different cancers.
- The results are not considered valid by regulatory agencies.
Answer: The targeted therapy may show different levels of efficacy in different tumor types (baskets), even with the same mutation.
10. What variable is held constant for the drug being tested in a basket trial?
- The dose
- The mechanism of action
- The route of administration
- All of the above
Answer: All of the above
11. What variable differs between the cohorts of a basket trial?
- The specific molecular alteration being targeted
- The experimental drug being tested
- The type of cancer or histology
- The primary investigator for the entire trial
Answer: The type of cancer or histology
12. The success of a basket trial depends on a validated link between the:
- Cancer type and the drug’s efficacy.
- Biomarker and the drug’s mechanism of action.
- Patient’s age and the biomarker.
- Drug’s cost and its mechanism of action.
Answer: The biomarker and the drug’s mechanism of action.
13. A basket trial tests ______ drug(s) based on ______ biomarker(s) across ______ disease(s).
- one; one; multiple
- multiple; multiple; one
- one; multiple; one
- multiple; one; multiple
Answer: one; one; multiple
14. A key logistical requirement for running a successful basket trial is:
- Access to a large, diverse patient population for broad molecular screening.
- A focus on only one type of cancer.
- The ability to manufacture multiple different drugs.
- A very short study duration.
Answer: Access to a large, diverse patient population for broad molecular screening.
15. If a drug shows significant activity in one “basket” (e.g., melanoma) but not in another (e.g., colon cancer), this suggests:
- The biomarker is irrelevant in all cancers.
- The drug is ineffective in all situations.
- The biology of the tumor type (tissue context) plays a role in the drug’s efficacy.
- The trial was poorly designed.
Answer: The biology of the tumor type (tissue context) plays a role in the drug’s efficacy.
16. From a patient’s perspective, what is a major opportunity of a basket trial?
- It guarantees a cure.
- It provides access to a potentially effective targeted therapy, especially for patients with rare cancers.
- It allows them to choose which drug they receive.
- It involves no medical procedures.
Answer: It provides access to a potentially effective targeted therapy, especially for patients with rare cancers.
17. The “Precision Oncology – Contemporary Trial Designs” lecture provides the foundation for understanding:
- The history of traditional chemotherapy.
- How to manage pharmacy inventory.
- The shift towards biomarker-driven trials like the basket design.
- The principles of sterile compounding.
Answer: The shift towards biomarker-driven trials like the basket design.
18. A pharmacist interpreting a basket trial publication should pay close attention to the:
- Response rates within each individual cancer cohort.
- The overall response rate only, ignoring differences between cohorts.
- The cost of the molecular screening test.
- The number of trial sites.
Answer: The response rates within each individual cancer cohort.
19. The sotorasib trial mentioned in the syllabus, which targets the KRAS G12C mutation, is a classic example of a study that could be designed as a(n):
- Bioequivalence study.
- Traditional randomized controlled trial.
- Basket trial, as KRAS G12C occurs in multiple cancer types.
- Post-marketing safety study.
Answer: Basket trial, as KRAS G12C occurs in multiple cancer types.
20. The rise of basket trials is directly tied to advances in:
- Surgical techniques.
- Next-generation sequencing (NGS) and molecular profiling.
- Radiation therapy.
- Patient counseling.
Answer: Next-generation sequencing (NGS) and molecular profiling.
21. A potential limitation of a basket trial is that:
- Each individual “basket” may be too small to provide statistically powerful results on its own.
- They are more expensive to run than multiple separate trials.
- They are less efficient than traditional trial designs.
- They cannot be used to study targeted therapies.
Answer: Each individual “basket” may be too small to provide statistically powerful results on its own.
22. An umbrella trial differs from a basket trial because the “umbrella” covers:
- One disease type with multiple arms for different biomarkers and drugs.
- One drug being tested in many different diseases.
- One biomarker being tested with multiple drugs.
- One patient receiving all the drugs in the trial.
Answer: One disease type with multiple arms for different biomarkers and drugs.
23. The “baskets” in a basket trial refer to the:
- Different drugs being tested.
- Different biomarkers being targeted.
- Different patient cohorts, each defined by a specific disease type.
- Different clinical trial sites.
Answer: Different patient cohorts, each defined by a specific disease type.
24. For a pharmaceutical company, a basket trial can efficiently:
- Test a single drug’s potential across a wide range of indications.
- Get a drug approved without any clinical data.
- Test multiple different drugs from their pipeline at once.
- Determine the maximum tolerated dose in healthy volunteers.
Answer: Test a single drug’s potential across a wide range of indications.
25. A pharmacist counseling a patient about a “tumor-agnostic” drug approved based on a basket trial would explain that the drug is indicated based on:
- Their type of cancer (e.g., lung cancer).
- Their specific biomarker status (e.g., MSI-H), regardless of where the cancer is in their body.
- Their age.
- The stage of their disease.
Answer: Their specific biomarker status (e.g., MSI-H), regardless of where the cancer is in their body.
26. The statistical analysis of a basket trial may involve:
- Pooling data from all baskets if the drug effect is similar across them.
- Analyzing each basket independently.
- Using Bayesian methods to borrow strength across cohorts.
- All of the above.
Answer: All of the above.
27. A key question answered by a basket trial is:
- “Is this drug safe in healthy people?”
- “Is this molecular target important across different types of cancer?”
- “Which of these five drugs is best for lung cancer?”
- “What is the standard of care for this disease?”
Answer: “Is this molecular target important across different types of cancer?”
28. Which of these scenarios describes a basket trial?
- Testing an anti-HER2 drug in HER2-positive breast, gastric, and bladder cancers.
- Testing five different drugs in patients with breast cancer.
- Testing a new pain medication for post-operative pain.
- Testing a generic drug against its brand-name equivalent.
Answer: Testing an anti-HER2 drug in HER2-positive breast, gastric, and bladder cancers.
29. The master protocol for a basket trial must clearly define:
- The single targeted therapy being used.
- The specific biomarker required for entry.
- The different cancer types that will form the “baskets.”
- All of the above.
Answer: All of the above.
30. The primary endpoint in most basket trial cohorts is typically:
- Overall survival.
- Objective response rate (ORR).
- Quality of life.
- The cost of treatment.
Answer: Objective response rate (ORR).
31. The main difference between the patient populations in an umbrella trial versus a basket trial is:
- The umbrella trial population is homogenous by disease; the basket trial population is heterogenous by disease.
- The umbrella trial population is heterogenous by disease; the basket trial population is homogenous by disease.
- The umbrella trial population does not need biomarker testing.
- The basket trial population receives multiple drugs.
Answer: The umbrella trial population is homogenous by disease; the basket trial population is heterogenous by disease.
32. For a student pharmacist, learning about basket trials is important for understanding the basis of:
- Tumor-agnostic drug approvals.
- The pharmaceutical supply chain.
- Medication compounding regulations.
- The structure of a community pharmacy.
Answer: Tumor-agnostic drug approvals.
33. The “Precision Oncology” module covers both umbrella and basket trials because they are both:
- Types of traditional randomized trials.
- Examples of master protocols that use biomarker-based patient selection.
- Used exclusively for non-cancer diseases.
- Designs that test multiple drugs in multiple diseases.
Answer: Examples of master protocols that use biomarker-based patient selection.
34. The success of drugs like larotrectinib and entrectinib in basket trials led to landmark FDA approvals for tumors with what specific alteration?
- KRAS mutations.
- ALK rearrangements.
- NTRK gene fusions.
- BRAF mutations.
Answer: NTRK gene fusions.
35. A key consideration when designing a basket trial is:
- The prevalence of the target biomarker across different tumor types.
- The cost of the placebo.
- The number of available hospital beds.
- The weather at the clinical trial sites.
Answer: The prevalence of the target biomarker across different tumor types.
36. From a drug development standpoint, a positive signal in a single basket of a basket trial could lead to:
- The immediate discontinuation of the drug.
- A larger, traditional trial focused on that specific cancer type.
- A price increase for the drug.
- A change in the drug’s chemical structure.
Answer: A larger, traditional trial focused on that specific cancer type.
37. When you see a clinical trial testing a single drug in “pan-tumor” cohorts based on a mutation, you are likely looking at a:
- Umbrella trial.
- Basket trial.
- Dose-finding study.
- Patient preference study.
Answer: Basket trial.
38. The alternative name for a basket trial that reflects its design is:
- A disease-specific trial.
- A biomarker-selected trial.
- A randomized placebo-controlled trial.
- A dose-escalation trial.
Answer: A biomarker-selected trial.
39. A pharmacist sees a new drug indication for “adult and pediatric patients with solid tumors that have a specific genetic feature.” This was likely approved based on data from a(n):
- Umbrella trial.
- Basket trial.
- Phase 1 safety trial.
- Post-marketing surveillance study.
Answer: Basket trial.
40. The main question a basket trial asks is: “Does this molecular alteration predict response to this drug, ________?”
- in this one specific cancer type
- regardless of the cancer’s tissue of origin
- only in pediatric patients
- only when combined with chemotherapy
Answer: regardless of the cancer’s tissue of origin
41. The main question an umbrella trial asks is: “For this specific cancer, ________?”
- does this one drug work for everyone
- can we match patients with different molecular alterations to different targeted drugs
- what is the cause of the cancer
- how can we prevent this cancer
Answer: can we match patients with different molecular alterations to different targeted drugs
42. Which of these is NOT a characteristic of a basket trial?
- Tests a single drug.
- Enrolls patients with multiple different diseases.
- Enrolls patients with multiple different biomarkers.
- All patients share one common biomarker.
Answer: Enrolls patients with multiple different biomarkers.
43. A major shift in thinking prompted by basket trials is moving from an organ-based to a(n) ________-based view of cancer.
- age
- geographically
- molecularly
- observationally
Answer: molecularly
44. The “contemporary trial designs” discussed in the syllabus are critical for the advancement of:
- Generic drug manufacturing.
- Precision medicine.
- Over-the-counter drug sales.
- Pharmacy benefit management.
Answer: Precision medicine.
45. For a pharmacist, a key aspect of understanding basket trials is recognizing that a patient’s eligibility for a new drug may depend on:
- The results of a genomic test rather than just their cancer type.
- Their insurance plan only.
- Their ability to travel to a specific hospital.
- The pharmacy’s inventory.
Answer: The results of a genomic test rather than just their cancer type.
46. A potential criticism of early-phase basket trials is that they often lack a:
- Biomarker for selection.
- Concurrent control or randomization group.
- Targeted therapy.
- Clear endpoint.
Answer: Concurrent control or randomization group.
47. The efficiency of a basket trial comes from not needing to:
- Open a brand new, separate trial for every single cancer type you want to test the drug in.
- Obtain patient consent.
- Get regulatory approval.
- Analyze any of the data.
Answer: Open a brand new, separate trial for every single cancer type you want to test the drug in.
48. In a basket trial, if a drug is highly effective in the lung cancer cohort but completely ineffective in the colon cancer cohort, this provides valuable information about:
- The importance of tissue context in drug response.
- The failure of the biomarker.
- The poor design of the trial.
- The drug’s safety profile.
Answer: The importance of tissue context in drug response.
49. An understanding of basket trials helps a pharmacist appreciate why:
- All cancer patients receive the same chemotherapy.
- Two patients with different cancers might receive the same targeted therapy.
- Molecular testing is not important in modern oncology.
- New drugs are becoming less expensive.
Answer: Two patients with different cancers might receive the same targeted therapy.
50. The ultimate goal of both basket and umbrella trial designs is to:
- Make clinical research more difficult and expensive.
- Accelerate the development of effective therapies for the right patient populations.
- Prove that targeted therapies do not work.
- Eliminate the need for pharmacists in clinical trials.
Answer: Accelerate the development of effective therapies for the right patient populations.
I am a Registered Pharmacist under the Pharmacy Act, 1948, and the founder of PharmacyFreak.com. I hold a Bachelor of Pharmacy degree from Rungta College of Pharmaceutical Science and Research. With a strong academic foundation and practical knowledge, I am committed to providing accurate, easy-to-understand content to support pharmacy students and professionals. My aim is to make complex pharmaceutical concepts accessible and useful for real-world application.
Mail- Sachin@pharmacyfreak.com