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Growth in the availability of cell and gene therapies (CGTs) promises significant innovation in the treatment of serious diseases, but the high cost and one-time administration of CGTs has also raised concern about strain on health plan budgets and inequity in access. We used coverage information from the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) database for 18 large commercial health plans in the US and information from state Medicaid websites to examine variation in coverage of 11 CGTs in August 2021. We found that US commercial and Medicaid health plans imposed restrictions in 53.5 % and 68.3 % of their coverage policies for the 11 included CGTs, respectively. In addition, we identified significant variation in access to CGTs across commercial plans and across Medicaid plans. Coverage restrictions for certain CGTs were more common than others; clinical requirements were often (but not always) consistent with the inclusion criteria for the clinical trial central to the drug's approval. We conclude that there is variation in access to CGTs, creating differential patient access.
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OBJECTIVES: First, to examine 7 large Medicare Advantage (MA) plans' use of step therapy. Second, to compare step therapy that health plans imposed in their MA and commercial (employer) drug coverage policies. STUDY DESIGN: Database analysis. METHODS: Using data from the Tufts Medical Center Specialty Drug Evidence and Coverage Database, we evaluated 7 large MA plans' use of step therapy in their Part B drug coverage policies. First, we determined the frequency with which different MA plans applied step therapy. Second, we determined the frequency with which plans imposed step therapy protocols across International Classification of Diseases, Tenth Revision, Clinical Modification categories. Third, we compared each step therapy protocol against each drug's corresponding FDA label indication. Fourth, we examined the consistency of step therapy protocols between the same insurer's MA and commercial lines of business. RESULTS: The frequency with which the included MA plans imposed step therapy ranged from 26.1% to 63.7%. Step therapy was most common for dermatology conditions (90.2%) and least common for oncology conditions (28.6%). On average, MA plans' step therapy requirements were consistent with the drug's FDA label indication 29.0% of the time. MA plans' and commercial plans' use of step therapy differed for the same drug-indication pairs 46.1% of the time. CONCLUSIONS: MA plans vary in the frequency with which they impose step therapy protocols in their Part B drug coverage policies. Moreover, insurers often impose different step therapy protocols in their MA plan and commercial plan offerings. Differences in plans' step therapy requirements may result in variability in patients' access to care within MA.
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Medicare Part C , Estados Unidos , Humanos , Anciano , Comercio , Bases de Datos Factuales , Planificación en Salud , HospitalesRESUMEN
BACKGROUND: Health plans apply utilization management criteria to guide their enrollees' access to prescription drugs. Patient subgroup restrictions (ie, clinical prerequisites for drug coverage) are a form of utilization management that have not been thoroughly investigated. OBJECTIVE: To examine the frequency with which large US commercial health plans impose patient subgroup restrictions beyond the US Food and Drug Administration (FDA) label in their coverage policies for orphan drugs and for drugs included in 1 or more FDA-expedited programs. To determine how consistently these patient subgroup restrictions align with eligibility criteria specified in each drug's pivotal clinical trial(s). METHODS: The Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) database was used, which includes coverage policies issued by 17 large US commercial health plans. SPEC contained 3,786 orphan drug policies and 4,027 FDA-expedited drug policies (current as of December 2020). SPEC data on plans' patient subgroup restrictions were assessed for the first objective. Each patient subgroup restriction was benchmarked against the corresponding eligibility criteria for a drug's pivotal clinical trial(s) for the second objective. To do so, the "Clinical Studies" section of the drug's FDA label was reviewed or, if necessary, the published manuscript describing the drug's pivotal trial(s). Patient subgroup restrictions were categorized as follows: (1) "consistent," the restriction and trial eligibility criterion are equivalent; (2) "same measure, more stringent," the restriction and trial eligibility criteria depend on the same measure, but the plan coverage is more restrictive; (3) "same measure, less stringent," the restriction and trial eligibility criteria depend on the same measure, but the plan coverage is less restrictive; and (4) "not consistent," the restriction and trial eligibility criteria depend on different measures. RESULTS: Health plans imposed patient subgroup restrictions in 20.2% of orphan drug policies (frequency varied by health plan, 11.7%-36.6%), and in 21.8% of FDA-expedited drug policies (frequency varied by health plan, 11.1%-47.9%). Of the 936 patient subgroup restrictions in orphan drug policies, 60.3% were categorized as consistent; 7.3% as same measure, more stringent; 12.0% as same measure, less stringent; and 20.5% as not consistent. Of the 1,070 patient subgroup restrictions in FDA-expedited drug policies, 57.5% were categorized as consistent; 6.7% as same measure, more stringent; 16.0% as same measure, less stringent; and 19.8% as not consistent. CONCLUSIONS: Patient subgroup restrictions for orphan drugs and FDA-expedited programs varied substantially across health plans, potentially resulting in inconsistent access to a given therapy across the approved patient population. Patient subgroup restrictions tend to be consistent with eligibility criteria specified in pivotal clinical trials. DISCLOSURES: This study was funded by Sarepta Therapeutics, Inc. Alexa C Klimchak and Lauren E Sedita are employees of Sarepta Therapeutics, Inc., and may own stock/options in the company.
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Medicamentos bajo Prescripción , Estados Unidos , Humanos , United States Food and Drug Administration , Producción de Medicamentos sin Interés ComercialRESUMEN
Health plans guide their enrollees' access to specialty drugs through coverage policies. We examined a set of health plan policies to determine if they have become more or less stringent over time. We did so by comparing the consistency of policies with Food and Drug Administration (FDA) label indications. We considered coverage policies for the same 187 specialty drugs issued by 17 large US commercial health plans from 2017 through 2021. Overall, the proportion of policies that were consistent with the FDA label declined from 57.1% in 2017 to 45.1% in 2021; the proportion of policies that were more restrictive than the FDA label increased from 39.5% to 51.7%. The proportion of policies excluding drug coverage remained approximately constant (3.4% in 2017; 3.2% in 2021). Trends in coverage restrictiveness varied across plans. For 13 plans, the proportion of policies with restrictions increased over time, while for 4 plans it declined.
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Maternal endocrine disorders can have detrimental effects on the fetus and the pregnancy can affect the course of a pre-exisiting endocrinopathy or induce the onset of one of these disorders. Therapies for endocrine disorders are not always safe to administer during pregnancy. Before administering any therapy to the mother, the effects on the fetus, the degree of placental trespassing as well as the potential damaging effects must be assessed. An accurate evaluation of the risks/benefits of any drug to be used on the mother is needed, assessing above all a potential theratogenic effect. In this review, the incidence of the main endocrine disorders, their evolution during pregnancy, their effects on mothers and fetuses and new acquisition on the treatment during pregnancy are discussed.
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Anomalías Inducidas por Medicamentos/etiología , Enfermedades del Sistema Endocrino/tratamiento farmacológico , Feto/efectos de los fármacos , Hormonas/uso terapéutico , Complicaciones del Embarazo/tratamiento farmacológico , Acromegalia/tratamiento farmacológico , Enfermedad de Addison/tratamiento farmacológico , Adulto , Niño , Síndrome de Cushing/tratamiento farmacológico , Diabetes Insípida/tratamiento farmacológico , Femenino , Hormonas/efectos adversos , Humanos , Hipercalcemia/tratamiento farmacológico , Hiperparatiroidismo/tratamiento farmacológico , Hipertiroidismo/tratamiento farmacológico , Hipoparatiroidismo/tratamiento farmacológico , Hipotiroidismo/tratamiento farmacológico , Recién Nacido , Neoplasias Hipofisarias/tratamiento farmacológico , Neoplasias Hipofisarias/cirugía , Embarazo , Complicaciones Neoplásicas del Embarazo/tratamiento farmacológico , Complicaciones Neoplásicas del Embarazo/cirugía , Prolactinoma/tratamiento farmacológico , Prolactinoma/cirugía , Medición de RiesgoRESUMEN
One of the main advances in the field of metabolic control of body weight and obesity treatment was the identification of the OB protein or leptin, that plays an important role in controlling body weight, signalling to the CNS the amount of body fat. Indeed, leptin levels are positively correlated to indices of body fat, namely total fat mass, percent body fat and body mass index (BMI). This protein may be also the signal that indicates the nutritional status to the reproductive axis. Whether this signal is exerted directly on the gonads or through the neuroendocrine axis is still to be determined. A sexual dimorphism between male and female in serum leptin levels has been observed, with the latter showing higher serum leptin levels. This evidence has led to the hypothesis that estrogens might have a stimulatory role in leptin secretion. To evaluate this hypothesis, several authors have determined serum leptin levels in postmenopausal women that have estrogen levels comparable to those present in men. The results of these studies are contradictory and the aim of this article has been the revision of data present in the literature regarding serum leptin levels in menopause and to correlate them to body composition changes taking place during menopause.
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Leptina/sangre , Menopausia/sangre , Adulto , Factores de Edad , Animales , Composición Corporal , Peso Corporal , Ensayos Clínicos como Asunto , Terapia de Reemplazo de Estrógeno , Estrógenos/fisiología , Femenino , Humanos , Masculino , Persona de Mediana Edad , Ovariectomía , Posmenopausia/sangre , Ratas , Reproducción/fisiología , Factores SexualesRESUMEN
BACKGROUND: To evaluate the feasibility of ovarian drilling using minilaparoscopy under local anesthesia and to determine its efficacy in the surgical treatment of polycystic ovarian syndrome. METHODS: Prospective randomized study carried out in an out patient service on 62 women affected by PCOS divided into two groups: 32 patients (group A) underwent bilateral ovarian drilling by minilaparoscopy under local anesthesia and 30 patients (group B) underwent bilateral ovarian drilling by traditional laparoscopy under general anesthesia. RESULTS: Operation times were not different between the two groups. Discharge time was significantly lower in group A in comparison to group B. The rate of patients discharged after 2 hours was significantly higher in group A. The need for additional analgesia was lower in group A in comparison to group B. Serum LH, A and T levels were significantly reduced after surgery in both groups. Pregnancy rate after 1-year follow-up was higher, although not significantly in group A. Ovulation and abortion rates were not different between the two groups. CONCLUSIONS: Ovarian drilling in minilaparoscopy under local anesthesia is a new option for gynecologists, allowing similar therapeutical results to those achieved by traditional laparoscopy, but with the benefits of a less invasive technique that can be carried out in an outpatient service without the need for general anesthesia.