Khattab Ahmed, Kim Hyunwoo, Mulrooney Mary, Leinwand Brian, Hadker Nandini, Cicero Samantha, Cheng Henry
Rutgers University, New Brunswick, NJ, United States.
Neurocrine Biosciences, San Diego, CA, United States.
Front Endocrinol (Lausanne). 2025 Sep 9;16:1603701. doi: 10.3389/fendo.2025.1603701. eCollection 2025.
Glucocorticoid (GC) therapies treat many chronic conditions such as congenital adrenal hyperplasia (CAH), but long-term use carries risks of side effects (e.g., skeletal, cardiometabolic, mental health issues) that can negatively impact clinical and economic outcomes. Consequently, patients and providers seek to balance the lowest efficacious dose and side effect risk. To our knowledge, no research has analyzed US payer coverage decisions on medications that reduce GC reliance.
To understand the significance and implications of US payer perceptions and coverage/access decisions for therapies reducing GC doses, which may be of relevance to new therapies for CAH.
A literature review was paired with primary market research to identify and characterize 5 GC-reducing therapies to evaluate payer coverage policies. No identified therapies were currently approved or studied in CAH. Qualitative interviews (n=13) were also conducted across managed care organizations, pharmacy benefit managers, and managed Medicaid payers to supplement publicly available information.
GC-reducing therapies were desirable and therapeutically beneficial from payer perspectives based on market research of payer coverage policies; all therapies were covered in place of or in addition to GCs. Despite premium pricing vs. low-cost alternatives, all therapies evaluated were covered by some or all payers with prior authorization to label indication or trial criteria. Qualitative interviews revealed that payers clearly understood the clinical burden of long-term GC use; however, the economic burden was less understood. Payers stated that GC reduction is a secondary decision-making driver due to the focus on trial primary endpoints, contracting dynamics, lack of competitors, and small trial sample sizes. A subset of payers was interested in GC reduction data as a primary endpoint for rare diseases without treatment alternatives and in pediatric populations.
Despite the premium price over GCs, GC-reducing therapies were covered in place of or in addition to GCs. Payers acknowledged the clinical value of reducing long-term GC use. Understanding what payers perceive as important criteria for coverage of GC-reducing medication may aid clinicians in evaluating utilization management criteria, such as step therapy, and increase access to medications aiming to reduce the patient burden associated with long-term GC use. This is particularly important in CAH where there is a high unmet need due to lifelong exposure to supraphysiologic doses of GCs.
糖皮质激素（GC）疗法可治疗多种慢性疾病，如先天性肾上腺皮质增生症（CAH），但长期使用存在副作用风险（如骨骼、心脏代谢、心理健康问题），可能对临床和经济结局产生负面影响。因此，患者和医疗服务提供者试图在最低有效剂量和副作用风险之间取得平衡。据我们所知，尚无研究分析美国医保支付方对降低GC依赖药物的覆盖决策。
了解美国医保支付方对降低GC剂量疗法的认知、覆盖/获取决策的意义和影响，这可能与CAH的新疗法相关。
文献综述与主要市场研究相结合，以识别和描述5种降低GC的疗法，评估医保支付方的覆盖政策。目前在CAH中未发现已获批或研究的疗法。还对管理式医疗组织、药品福利管理机构和管理式医疗补助支付方进行了定性访谈（n = 13），以补充公开可用信息。
根据对医保支付方覆盖政策的市场研究，从医保支付方的角度来看，降低GC的疗法是可取的且具有治疗益处；所有疗法都被覆盖，可替代GC或作为GC的补充。尽管与低成本替代方案相比价格高昂，但所有评估的疗法都被部分或所有医保支付方覆盖，需事先批准以符合标签适应症或试验标准。定性访谈表明，医保支付方清楚地了解长期使用GC的临床负担；然而，对经济负担的了解较少。医保支付方表示，由于关注试验主要终点、合同动态、缺乏竞争对手以及试验样本量小，降低GC是次要的决策驱动因素。一部分医保支付方对降低GC的数据感兴趣，将其作为没有治疗替代方案的罕见疾病以及儿科人群的主要终点。
尽管价格高于GC，但降低GC的疗法被覆盖，可替代GC或作为GC的补充。医保支付方认可降低长期GC使用的临床价值。了解医保支付方认为降低GC药物覆盖的重要标准，可能有助于临床医生评估利用管理标准，如逐步治疗，并增加获取旨在减轻患者长期使用GC相关负担的药物的机会。这在CAH中尤为重要，因为患者因终身暴露于超生理剂量的GC而存在大量未满足的需求。
