1. Attributes of fibrodysplasia ossificans progressiva (FOP), an ultra-rare disease of abnormal bone growth, include all of the following EXCEPT:
A. Heterotopic ossification (HO)
B. Elongation of molar teeth
C. Malformation of the great toes
D. Progressive loss of mobility
E. Shortened lifespan
F. Unsure
2. Which of the following statements about the diagnosis of FOP is TRUE?
A. FOP has distinctive clinical attributes, therefore misdiagnosis is rare
B. Formal diagnostic criteria make diagnosis of FOP clear and straightforward
C. Misdiagnosis of FOP is uncommon due to widespread understanding of the disease
D. Misdiagnosis of FOP is common due in part to the rarity of the disease
E. Unsure
3. FOP is an autosomal dominant genetic disorder characterized by a mutation in ACVR1. The mutation causes which of the following?
A. Gain-of-function effect in ACVR1 such that the protein, activin A, exhibits agonist activity
B. Gain-of-function effect in ACVR1 such that the protein, activin A, exhibits antagonist activity
C. Loss-of-function effect in ACVR1 such that the protein, activin A, exhibits agonist activity
D. Loss-of-function effect in ACVR1 such that the protein, activin A, exhibits antagonist activity
E. Unsure
4. Palovarotene is a drug under investigation for the treatment of FOP. Which of the following statements best describes the proposed mechanism of action of palovarotene in FOP?
A. A tyrosine kinase inhibitor that inhibits the activity of mutated ACVR1
B. A retinoic acid receptor (RAR)-γ agonist that leads to the inhibition of chondrogenesis
C. A fully human monoclonal antibody that binds to activin A to halt the signaling cascade leading to HO
D. An inhibitor of mechanistic target of rapamycin (mTOR) kinase that disrupts the mTOR signaling pathway, which is upregulated in FOP
E. Unsure
5. Garetosmab is a drug under investigation for the treatment of FOP. Which of the following best describes the proposed mechanism of action of garetosmab in FOP?
A. A tyrosine kinase inhibitor that inhibits the activity of mutated ACVR1
B. An RAR-γ agonist that leads to inhibition of chondrogenesis
C. A fully human monoclonal antibody that binds to activin A to halt the signaling cascade leading to HO
D. An inhibitor of mTOR kinase that disrupts the mTOR signaling pathway, which is upregulated in FOP
E. Unsure
6. Which of the following styles of therapy can be used to help people with FOP engage in active range of motion, cardiopulmonary, and resistive exercise in a safe, low-impact environment?
A. Warm water hydrotherapy
B. Art therapy
C. Drama therapy
D. Music therapy
E. Unsure
7. HMB is a 15-year-old girl who was previously diagnosed with FOP based on clinical manifestations (inflammatory soft tissue swellings, bilateral malformation of the great toe) and confirmed via genetic testing. HMB is due for her yearly influenza vaccination. Which of the following is NOT TRUE regarding her influenza vaccination?
A. Intramuscular injection must be avoided to prevent HO from muscle trauma at the injection site
B. Only subcutaneous or nasal administration of the influenza vaccination should be used for people with FOP
C. Immunizations should not be given to people with FOP during an active flare-up
D. No special considerations are necessary for vaccinations in people with FOP
E. Unsure
8. Which of the following statements about prescription drug costs is FALSE?
A. The costs of prescription drugs continue to rise and to require greater proportions of healthcare spending
B. Drug spending will increase to $190 to $225 billion in 2023
C. Prescription drug costs increased by 5.7% from 2018 to 2019
D. Prescription drug costs for specialty drugs may be of even more of a concern
E. Unsure
9. When a payer reviews drugs to be covered for rare and ultra-rare diseases, which of the following should be prioritized?
A. Drug development costs
B. Phase 1 data in healthy volunteers
C. Clinical trial data in relevant patient populations
D. Availability of copayment assistance
E. Unsure
10. In the United States, drugs for rare diseases are often developed with special incentives provided under which of the following acts?
A. Federal Food, Drug, and Cosmetic Act of 1938
B. Drug Price Competition and Patent Term Restoration Act of 1984
C. Biologics Price Competition and Innovation Act of 2009
D. Orphan Drug Act of 1983
E. Unsure
Evaluation Questions
11. How confident are in your treatment choice for HMB in question #7?
A. Not at all confident
B. Somewhat confident
C. Confident
D. Highly confident