CSP CSP - Certified Specialty Pharmacist Clinical Management: Neurology and Rare Disease Questions and Answers 1 — Questions and Answers
Question 1: Which disease-modifying therapy for multiple sclerosis (MS) requires REMS program enrollment due to risks of progressive multifocal leukoencephalopathy (PML)?
- Interferon beta-1a (Avonex)
- Natalizumab (Tysabri) (Correct answer)
- Glatiramer acetate (Copaxone)
- Dimethyl fumarate (Tecfidera)
Correct answer: Natalizumab (Tysabri)
Natalizumab (Tysabri) carries a REMS requirement called TOUCH because of the risk of PML, a potentially fatal brain infection caused by JC virus reactivation.
Question 2: A patient with spinal muscular atrophy (SMA) is prescribed nusinersen (Spinraza). What is a key specialty pharmacy role in managing this therapy?
- Compounding the intrathecal formulation on-site
- Coordinating benefits, site-of-care logistics, and patient support for this high-cost intrathecal therapy (Correct answer)
- Dispensing the medication to the patient for self-administration at home
- Performing the lumbar puncture procedure required for administration
Correct answer: Coordinating benefits, site-of-care logistics, and patient support for this high-cost intrathecal therapy
Nusinersen is an intrathecal injection administered by a neurologist; the specialty pharmacy's role centers on benefits investigation, authorization, site-of-care coordination, and patient/caregiver support.
Question 3: Which monitoring parameter is MOST critical for patients on alemtuzumab (Lemtrada) for multiple sclerosis?
- Liver function tests monthly
- Thyroid function tests, complete blood count, and urinalysis monthly for 48 months after last dose (Correct answer)
- Serum creatinine weekly
- Fasting lipid panel quarterly
Correct answer: Thyroid function tests, complete blood count, and urinalysis monthly for 48 months after last dose
Alemtuzumab carries risks of serious autoimmune conditions, including thyroid disorders, immune thrombocytopenia, and nephropathy, requiring monthly monitoring of thyroid function, CBC, and urinalysis for 4 years post-treatment.
Question 4: For a patient with Gaucher disease type 1 starting enzyme replacement therapy (ERT) with imiglucerase (Cerezyme), what is the MOST important pharmacist counseling point regarding administration?
- The medication can be self-injected subcutaneously at home without training
- Infusion reactions may occur, and premedication and close monitoring during infusions are recommended (Correct answer)
- The drug should be taken with food to improve absorption
- The patient must avoid all live vaccines while on therapy
Correct answer: Infusion reactions may occur, and premedication and close monitoring during infusions are recommended
Infusion-related reactions are the most common adverse effects of imiglucerase; patients should be informed about symptoms and the importance of premedication and monitoring at the infusion site.
Question 5: A CSP is counseling a patient newly prescribed fingolimod (Gilenya) for relapsing MS. Which assessment must be completed BEFORE the first dose?
- Bone mineral density scan
- Cardiac evaluation including ECG and first-dose observation for bradycardia (Correct answer)
- Colonoscopy to assess GI tolerability
- Pulmonary function testing
Correct answer: Cardiac evaluation including ECG and first-dose observation for bradycardia
Fingolimod causes bradycardia and AV block with the first dose; an ECG is required beforehand and patients must be observed for at least 6 hours after the first dose for cardiac effects.
Question 6: Which orphan drug act provision is MOST relevant to specialty pharmacies dispensing rare disease therapies?
- It requires specialty pharmacies to dispense orphan drugs at no charge
- It grants manufacturers market exclusivity for 7 years, which often results in limited competition and high drug prices that specialty pharmacies must navigate with payers (Correct answer)
- It mandates that orphan drugs bypass FDA approval
- It allows specialty pharmacies to compound orphan drugs without FDA oversight
Correct answer: It grants manufacturers market exclusivity for 7 years, which often results in limited competition and high drug prices that specialty pharmacies must navigate with payers
The Orphan Drug Act's 7-year market exclusivity limits generic/biosimilar competition, contributing to high costs that specialty pharmacies must address through prior authorizations, manufacturer assistance programs, and payer negotiations.
CSP CSP - Certified Specialty Pharmacist Clinical Management: Neurology and Rare Disease Questions and Answers 1
Which disease-modifying therapy for multiple sclerosis (MS) requires REMS program enrollment due to risks of progressive multifocal leukoencephalopathy (PML)?