Therapeutics first approved without clinical postmarketing requirements or commitments included widely prescribed therapeutics (eg, apixaban, first approved for reduction of risk of stroke and systemic embolism caused by nonvalvular atrial fibrillation) and therapeutics with orphan designations for rare disease subtypes (eg, ivacaftor, first approved for patients with cystic fibrosis with a G551D mutation in the cystic fibrosis transmembrane conductance regulator gene) (eTable 1 in the Supplement). Here, CFTR is linked to stroke disorder.