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BACKGROUND: Despite the high prevalence of brain metastases (BM) secondary to non-small-cell lung cancer (NSCLC) (NSCLC/BM), patients' experiences (symptoms and impacts) are not fully understood. This study sought to understand the patient experience with NSCLC/BM and identify a patient-reported outcome (PRO) measure fit to capture the most important NSCLC/BM symptoms and impacts. METHODS: A targeted literature review was completed; the National Comprehensive Cancer Network (NCCN)/Functional Assessment of Cancer Therapy-Brain Symptom Index, 24-item version (NFBrSI-24) was identified as a relevant measure that assessed the core symptoms and impacts associated with NSCLC/BM. Qualitative interviews composed of concept elicitation and cognitive debriefing with oncologists (n = 3) and adult patients (n = 16) with NSCLC/BM were conducted to confirm the content validity and evaluate the relevance and appropriateness of the NFBrSI-24 for this condition. RESULTS: The NSCLC/BM symptoms and impacts identified in the literature and reported by oncologists and patients were consistent and captured in the NFBrSI-24. Study participants reported significant burden associated with the symptoms (commonly fatigue, headache) and impacts of NSCLC/BM. Participants indicated that the NFBrSI-24 captured their most salient experiences with NSCLC/BM and that symptom improvement or a delay in progression, as measured by the NFBrSI-24, would be meaningful. During the cognitive debriefing, participants generally indicated that the NFBrSI-24 was comprehensive and easy to understand/answer and that it assessed symptoms they considered most important to treat. CONCLUSIONS: These results suggest that the NFBrSI-24 adequately captures an appropriate measure of NSCLC/BM symptoms and impact.
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Neoplasias Encefálicas , Carcinoma Pulmonar de Células não Pequenas , Neoplasias Pulmonares , Oncologistas , Adulto , Humanos , Neoplasias Encefálicas/secundário , Carcinoma Pulmonar de Células não Pequenas/patologia , Neoplasias Pulmonares/patologia , Medidas de Resultados Relatados pelo PacienteRESUMO
BACKGROUND: To identify and describe the breast cancer-specific health-related quality of life (HRQoL) instruments with evidence of validation in the breast cancer population for potential use in patients treated for breast cancer (excluding surgery). METHODS: We conducted a systematic literature review using PubMed, Embase, and PsycINFO databases to identify articles that contain psychometric properties of HRQoL instruments used in patients with breast cancer. Relevant literature from January 1, 2009, to August 19, 2019, was searched. Articles published in English that reported psychometric properties (reliability, validity) of HRQoL instruments were identified. RESULTS: The database search yielded 613 unique records; 131 full-text articles were reviewed; 80 articles presented psychometric data for instruments used in breast cancer (including generic measures). This article reviews the 33 full articles describing psychometric properties of breast cancer-specific HRQoL instruments: EORTC QLQ-C30, EORTC QLQ-BR23, FACT-B, FBSI, NFBSI-16, YW-BCI36, BCSS, QuEST-Br, QLICP-BR, INA-BCHRQoL, and two newly developed unnamed measures, one by Deshpande and colleagues (for use in India) and one by Vanlemmens and colleagues (for use among young women and their partners). The articles that described the EORTC QLQ-C30, QLQ-BR23, and FACT-B centered on validating translations, providing additional support for content validity, and demonstrating acceptability of electronic patient-reported outcome administration. Psychometric properties of the measures were acceptable. Several new measures have been developed in Asia with an emphasis on development on cultural relevance/sensitivity. Others focused on specific populations (i.e., young women with breast cancer). CONCLUSIONS: Historically, there have been limited options for validated measures to assess HRQoL of patients with breast cancer. A number of new measures have been developed and validated, offering promising options for assessing HRQoL in this patient population. This review supports the reliability and validity of the EORTC QLQ-C30 and FACT-B; new translations and electronic versions of these measures further support their use for this population.
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Neoplasias da Mama/psicologia , Medidas de Resultados Relatados pelo Paciente , Qualidade de Vida , Inquéritos e Questionários/normas , Adulto , Feminino , Humanos , Pessoa de Meia-Idade , Psicometria , Reprodutibilidade dos Testes , TraduçõesRESUMO
BACKGROUND: The economic impact of intra-articular hyaluronic acid (IAHA) for the treatment of knee pain associated with osteoarthritis (OA) has been evaluated in the United States, but not systematically summarized. OBJECTIVE: We reviewed the literature to determine the economic impact of IAHA for pain associated with knee OA in the United States. METHODS: A literature review was performed in PubMed (including MEDLINE and MEDLINE In-Process), Embase, the Cochrane Database of Systematic Reviews, and National Health Service Economic Evaluation Database and was limited to English language human studies published from January 2000 to October 2020. RESULTS: The literature search identified 215 unique abstracts; of these, 47 were selected for full-text review and 21 studies met the inclusion criteria. Intra-articular hyaluronic acid injections delayed progression to total knee arthroplasty (TKA), and repeated courses of treatment successfully delayed TKA by more than 5 years. Intra-articular hyaluronic acid was found to reduce the use of pain medications overall and reduce the number of patients receiving opioid prescriptions by 6% (P < .001). Several studies showed that IAHA is more cost-effective in treating pain associated with knee OA compared with conventional care with nonsteroidal anti-inflammatory drugs (NSAIDs), analgesics, and corticosteroids, and several authors concluded that IAHA should be the dominant treatment strategy. CONCLUSIONS: Current studies suggest that IAHA may reduce the use of pain medications, such as NSAIDs and opioids, and impact time to TKA procedures, thus potentially decreasing overall treatment costs of knee OA over time. Furthermore, IAHA was determined to be cost-effective against NSAIDs, corticosteroids, analgesics, and conservative treatment. As the safety and efficacy of IAHA for knee OA have been well established, the findings from our literature review may be used to inform future economic evaluations.
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BACKGROUND: Until recently, many clinical trials in patients with pulmonary arterial hypertension (PAH) evaluated exercise capacity with 6-minute walk distance (6MWD) as the primary endpoint. Common secondary endpoints include PAH functional class (FC), which assesses symptoms, and either brain natriuretic peptide (BNP) or the inactive N-terminal cleavage product of its prohormone (NT-proBNP), which assesses cardiac function. OBJECTIVE: Examine the relationships among 6MWD, FC, and BNP/NT-proBNP measured at baseline or follow-up with long-term outcomes in PAH studies. METHODS: Relevant literature from January 1990 to April 2018 were obtained by searching PubMed, Embase, and Cochrane. Articles in English reporting on associations between 6MWD, FC, or BNP/NT-proBNP and outcomes in PAH were identified. Each endpoint was evaluated individually. Prespecified inclusion and exclusion criteria were applied at level 1 (titles/abstracts) and level 2 (full-text review). RESULTS: The database search yielded 836 unique records; 65 full-text articles were reviewed. Twenty-five studies were eligible for inclusion. Findings supported the importance of measuring PAH noninvasive endpoints in predicting long-term outcomes. Patients with shorter or decreased 6MWD, poor (III/IV) or declining FC (e.g., from II to III), or elevated or increasing BNP/NT-proBNP had a higher risk of death and costly events (e.g., hospitalization, lung transplant). FC also predicted health care resource utilization and costs. Collectively, these endpoints establish risk groups that predict likelihood of complications from PAH or death. CONCLUSION: Assessment of 6MWD, FC, and BNP/NT-proBNP provides low-cost, efficient, and noninvasive means of predicting long-term health and economic outcomes in patients with PAH.
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Estado Funcional , Peptídeo Natriurético Encefálico/sangue , Fragmentos de Peptídeos/sangue , Hipertensão Arterial Pulmonar/fisiopatologia , Teste de Caminhada , Biomarcadores/sangue , Progressão da Doença , Hospitalização/estatística & dados numéricos , Humanos , Transplante de Pulmão/estatística & dados numéricos , Mortalidade , Avaliação de Resultados em Cuidados de Saúde , Prognóstico , Hipertensão Arterial Pulmonar/sangue , Hipertensão Arterial Pulmonar/mortalidade , Índice de Gravidade de DoençaRESUMO
BACKGROUND AND OBJECTIVE: Patient perspectives regarding treatment experience and satisfaction may be useful for clinicians when making treatment strategies. This US-based study assessed the feasibility of evaluating real-world, patient-reported narratives regarding symptom improvement and treatment satisfaction among patients with psoriatic arthritis treated with secukinumab. METHODS: A cross-sectional, web-based survey collected data on demographics, disease characteristics, symptoms before and after secukinumab use, and treatment satisfaction with secukinumab. RESULTS: Of 2755 patients screened, 200 patients with psoriatic arthritis were eligible and included in the analysis. Their mean age was 36.0 (standard deviation, 10.0) years; 55.5% were male and 75.0% were white. Most (87.5%) were biologic experienced; the primary reason for discontinuation of their previous treatment was lack of effectiveness (28.6%). Most patients (79.9%) reported overall psoriatic arthritis symptom improvement after secukinumab initiation compared with before secukinumab initiation; a similar trend was observed for all individual symptoms evaluated. Approximately half of patients reported improvement within 4 weeks after starting secukinumab treatment, and > 90% reported improvement within 6 months. Most patients (≥ 96%) expressed overall satisfaction with secukinumab regarding symptom improvement, speed of symptom improvement, frequency of administration, method of administration, ease of use, patient support services, and side effects, if any. CONCLUSIONS: Patient-reported perspectives may be feasibly collected to provide insights into treatment experience and satisfaction of secukinumab. Most patients with psoriatic arthritis in our real-world study experienced symptom improvement after initiating secukinumab; > 50% of patients reported symptom improvement within 4 weeks. Additionally, almost all patients reported satisfaction with secukinumab treatment.
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Anticorpos Monoclonais Humanizados/uso terapêutico , Artrite Psoriásica/tratamento farmacológico , Internet , Satisfação do Paciente , Adulto , Estudos Transversais , Estudos de Viabilidade , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Medidas de Resultados Relatados pelo Paciente , Inquéritos e QuestionáriosRESUMO
PURPOSE: This real-world study evaluated the feasibility of assessing patient-reported symptom improvement and treatment satisfaction using a web-based survey among patients with ankylosing spondylitis (AS) treated with secukinumab. METHODS: This cross-sectional, web-based survey collected data on demographics, symptoms, treatment history, and treatment satisfaction from US patients with AS who were receiving secukinumab at survey participation. Patients reported AS symptoms experienced before and after secukinumab initiation, time to symptom improvement, and satisfaction with secukinumab treatment. RESULTS: Of 2755 patients screened, 200 with AS were included in the analysis. The mean (SD) age of patients was 34.4 (10.6) years; 86.5% were biologic experienced. Most (74.0%) reported overall improvement ("a little," "moderately," or "much better") in AS symptoms since secukinumab initiation compared with before secukinumab initiation; a similar trend was observed for all the individual symptoms analyzed (pain disrupting sleep, fatigue, morning stiffness, pain and stiffness in lower back or neck, sore areas other than joints, and ankle or heel pain [indicating enthesitis]). Approximately 41.9% of patients reported overall symptom improvement within 4 weeks of secukinumab treatment. Most expressed overall satisfaction ("very," "mostly," or "somewhat satisfied") with secukinumab regarding symptom improvement (99.0%), speed of symptom improvement (97.0%), frequency and method of administration (96.0% and 91.5%, respectively), ease of use (93.5%), patient support services (97.0%), and side effects, if any (93.0%). CONCLUSION: Most patients reported overall symptom improvement and satisfaction with treatment. Our study indicates that patient-reported perspectives may be feasibly collected using a web-based survey to provide insights into treatment experience and satisfaction.
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OBJECTIVES: To evaluate the psychometric properties of Patient's Knee Implant Performance Questionnaire (PKIP), a new patient-reported measure of knee implant functional performance assessing patient experiences before and after primary total knee arthroplasty (TKA). METHODS: The psychometric analysis sample (n = 761) was based on an ongoing, multisite, prospective, noncomparative, longitudinal study of patients undergoing TKA. The PKIP was completed at three study visits: presurgery (visit 1), less than 1 year (visit 3, days 1-303), and minimum 1 year (visit 4, days 304-668). Visit 2 was an operative visit. Supporting outcome measures were collected at each study visit. The PKIP structure and its psychometric properties were assessed as part of a secondary data analysis using an interim data cut before the end of the clinical trial. RESULTS: The PKIP includes four subscales (confidence, stability, activity modification, and satisfaction) and an overall PKIP score. The overall PKIP score met internal consistency (visit 4 = 0.79) and test-retest reliability (intraclass correlation coefficient = 0.77) standards. Correlations between the PKIP and other measures (e.g., the American Knee Society score and the Knee Injury and Osteoarthritis Outcome Score) provided evidence of construct validity. The PKIP was capable of discriminating between groups of patients with better or worse knee function. The effect size for the overall PKIP score was 2.96 from baseline to visit 4, indicating that the PKIP was highly responsive. CONCLUSIONS: The reliability, validity, and responsiveness of the PKIP support its use among patients undergoing TKA.
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Artroplastia do Joelho , Articulação do Joelho/cirurgia , Joelho/cirurgia , Movimento , Medidas de Resultados Relatados pelo Paciente , Satisfação do Paciente , Inquéritos e Questionários , Atividades Cotidianas , Adulto , Idoso , Idoso de 80 Anos ou mais , Feminino , Humanos , Traumatismos do Joelho/cirurgia , Estudos Longitudinais , Masculino , Pessoa de Meia-Idade , Osteoartrite/cirurgia , Estudos Prospectivos , Psicometria , Reprodutibilidade dos TestesRESUMO
BACKGROUND: A review of new drug approvals (NDAs) by the Food and Drug Administration (FDA) for 2006 to 2010 showed that 24.1% of new drugs had patient-reported outcome (PRO) labeling. OBJECTIVES: To review PRO labeling for NDAs for 2011 to 2015 and to compare key findings reported previously. METHODS: A review of the FDA drug approval reports for NDAs was conducted using the FDA Web site to determine the number of NDAs for the period 2011 to 2015. For all identified NDAs, drug approval package and product labeling were reviewed to identify PRO end-point status and PRO labeling. NDAs for the period 2006 to 2015 were grouped by disease category as per the International Classification of Diseases, Tenth Revision. Data were summarized for all NDAs and for approvals in diseases that traditionally rely on PROs for evaluating treatment benefit (PRO-dependent). Results were compared with NDAs for the period 2006 to 2010. RESULTS: In the period 2011 to 2015, 16.5% of the 182 NDAs had PRO labeling. For PRO-dependent NDAs, this figure was 46.9% and 46.0% for the period 2006 to 1010 and the period 2011 to 2015, respectively. Most of the PRO labeling for the period 2011 to 2015 was based on primary end points (76.7%). Almost all PRO labeling was for concepts proximal to the disease. CONCLUSIONS: There is potential for increased PRO labeling, especially for drug approvals in diseases that traditionally rely on PROs for evaluating treatment benefit to satisfy regulatory needs. Less PRO labeling based on secondary end points may be indicative of drug manufacturers' reluctance to aid and enhance the value propositions of their products to all stakeholders, including patients.
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Aprovação de Drogas/estatística & dados numéricos , Rotulagem de Medicamentos/estatística & dados numéricos , Medidas de Resultados Relatados pelo Paciente , Doença Crônica/tratamento farmacológico , Humanos , Medicamentos sob Prescrição/uso terapêutico , Rotulagem de Produtos , Doenças Raras/tratamento farmacológico , Estados Unidos , United States Food and Drug AdministrationRESUMO
BACKGROUND: The emergence of new biological therapies showing high and sustained level of Psoriasis Area and Severity Index (PASI) 90 response has provided the possibility of both greater skin clearance and increased quality of life (QOL). OBJECTIVE: To evaluate the association of greater response in skin clearance with improvements in skin-related QOL up to 52 weeks. METHODS: Subjects achieving various levels of skin clearance (PASI 90-100 or PASI 75-89) and Dermatology Life Quality Index (DLQI) (0/1) response were compared using ERASURE and FIXTURE trial data. Similar analyses with IGA ratings of Clear or Almost Clear were performed. RESULTS: Significantly more PASI 90-100 responders at week 12 had DLQI 0/1 response than PASI 75-89 (69.4% vs. 47.1%; p < .001) and sustained DLQI 0/1 response at week 52 (74.0% vs. 56.7%; p < .001). IGA results were similar. CONCLUSIONS: These results show that PASI 90-100 is a relevant therapeutic goal in moderate to severe psoriasis compared to PASI 75-89 when considering patients' QOL.
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Anticorpos Monoclonais/uso terapêutico , Fatores Imunológicos/uso terapêutico , Psoríase/tratamento farmacológico , Qualidade de Vida , Adulto , Anticorpos Monoclonais Humanizados , Método Duplo-Cego , Esquema de Medicação , Cálculos da Dosagem de Medicamento , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Efeito Placebo , Psoríase/patologia , Índice de Gravidade de Doença , Resultado do TratamentoRESUMO
BACKGROUND: Psoriasis is a chronic condition with negative impact on patients' quality of life that most often requires lifelong effective and safe treatment. OBJECTIVE: This analysis focused on the effect of secukinumab treatment on patient-reported health-related quality of life as assessed by the Dermatology Life Quality Index (DLQI) in patients with moderate to severe psoriasis. METHODS: The proportion of subjects achieving DLQI score 0/1 response at week 24, time to DLQI score 0/1 response, and sustained DLQI score 0/1 response up to week 52 were compared between secukinumab and etanercept. RESULTS: Of 1470 subjects, 1144 received secukinumab and 326 received etanercept. DLQI score 0/1 response rates were significantly higher for secukinumab than for etanercept at week 24. The median time to DLQI score 0/1 response was significantly shorter for secukinumab versus etanercept (12 vs 24 weeks; P < .01). The majority of secukinumab-treated subjects achieved DLQI score 0/1 response at week 24 and sustained it through week 52 along with a 90% to 100% reduction in the Psoriasis Area and Severity Index total score response. LIMITATIONS: Placebo comparisons are limited during the maintenance period because of rerandomization at week 12. CONCLUSION: Secukinumab treatment provided faster and greater sustained improvements in quality of life than etanercept over 52 weeks, consistent with greater clinical response.
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Anticorpos Monoclonais/uso terapêutico , Fármacos Dermatológicos/uso terapêutico , Etanercepte/uso terapêutico , Imunossupressores/uso terapêutico , Psoríase/tratamento farmacológico , Adulto , Anticorpos Monoclonais Humanizados , Método Duplo-Cego , Feminino , Humanos , Injeções Subcutâneas , Interleucina-17/antagonistas & inibidores , Masculino , Pessoa de Meia-Idade , Satisfação do Paciente , Psoríase/psicologia , Qualidade de Vida , Recidiva , Inquéritos e Questionários , Resultado do Tratamento , Fator de Necrose Tumoral alfa/antagonistas & inibidoresRESUMO
BACKGROUND: Psoriasis can greatly impact patients' lives, influencing what clothing they wear and impairing their sexual functioning. OBJECTIVE: To provide a detailed comparison of the impact of secukinumab vs. etanercept on enabling patients with psoriasis to have more normal lives with respect to daily activities (DA) (e.g. choosing clothing) and personal relationships (PR) (e.g. sexual functioning). METHODS: Baseline to Week 52 ERASURE and FIXTURE data for secukinumab 300 mg and etanercept were analyzed. Treatment differences in mean scores on the DA and PR subscales and items from the Dermatology Life Quality Index were compared. The proportions of subscale and item responders (score = 0) were compared. RESULTS: Subjects receiving secukinumab (n = 572) achieved greater mean improvement in DA and items (q3 and q4) than subjects receiving etanercept (n = 326; all p < .01 through Week 52). Subjects receiving secukinumab achieved greater mean improvement in PR and items (q8 and q9) than subjects receiving etanercept (subscale: p < .05 at Weeks 8 and 12). Response rates were significantly higher for secukinumab than for etanercept for DA (all p < .0001) and PR (all p < .05 except Weeks 4 and 36). CONCLUSIONS: Secukinumab 300 mg provides greater improvements and more effective relief from psoriasis impact on DA and PR than etanercept.
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Anticorpos Monoclonais/uso terapêutico , Etanercepte/uso terapêutico , Imunossupressores/uso terapêutico , Psoríase/tratamento farmacológico , Atividades Cotidianas , Adulto , Anticorpos Monoclonais Humanizados , Método Duplo-Cego , Relações Familiares , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Efeito Placebo , Psoríase/patologia , Índice de Gravidade de Doença , Resultado do TratamentoRESUMO
BACKGROUND: Secukinumab is a human interleukin-17A antagonist indicated for the treatment of moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy. The objective of this analysis was to measure the treatment response on psoriasis-related itching, pain, and scaling via the Psoriasis Symptom Diary (PSD)(©). METHODS: ERASURE (n = 738) and FIXTURE (n = 1306) were double-blind, multicenter phase 3 studies in adults randomized to secukinumab (300, 150 mg, n = 1144) or placebo (n = 574) (administered at Weeks 0, 1, 2, 3, and 4, followed by dosing every 4 weeks) or a biologic active control (FIXTURE only). Patient-reported itching, pain, and scaling were assessed during the first 12 weeks of treatment using the PSD. The results reported here are limited to subjects in the secukinumab and placebo treatment groups who completed the PSD. The proportions of subjects achieving prespecified responses (improvement:reduction of at least 2.2 points for itching, 2.2 points for pain, or 2.3 points for scaling) were compared for secukinumab versus placebo. RESULTS: Overall, 39% of subjects completed the PSD at baseline and Week 12 (n = 453 secukinumab; 225 placebo). Subjects treated with secukinumab achieved significantly greater improvements in itching, pain, and scaling at Week 12 versus placebo (all P < 0.0001) and had significantly greater proportions of itching, pain, and scaling responders at Week 12 versus placebo (all P < 0.05). CONCLUSION: Secukinumab significantly improves patient-reported itching, pain, and scaling in adults with moderate to severe psoriasis compared with placebo.
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Anticorpos Monoclonais/uso terapêutico , Fatores Imunológicos/uso terapêutico , Psoríase/complicações , Psoríase/tratamento farmacológico , Adulto , Anticorpos Monoclonais Humanizados , Método Duplo-Cego , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Dor/etiologia , Medidas de Resultados Relatados pelo Paciente , Prurido/etiologia , Índice de Gravidade de Doença , Avaliação de SintomasRESUMO
OBJECTIVES: This analysis aimed to confirm the reliability, validity, and responsiveness of the Psoriasis Symptom Diary (PSD) using data from two Phase III studies in patients with moderate to severe chronic plaque psoriasis. METHODS: Data from two randomized, double-blind, double-dummy, placebo-controlled, multicenter Phase III studies (n = 820) assessing the efficacy and safety of secukinumab were used. The PSD (24-h recall; 0-10 numeric rating scale) was electronically administered each evening. Test-retest reliability was determined using intraclass correlations. Construct validity hypotheses were evaluated via correlations with the Psoriasis Area and Severity Index (PASI), Investigator's Global Assessment (IGA), Dermatology Life Quality Index (DLQI), EuroQoL 5-Dimension Health Status Questionnaire, and Patient Global Impression of Change (PGIC). Discriminating ability and responsiveness were evaluated by estimating mean differences and effect sizes between known groups (using the PASI and IGA). Phase II-derived, anchor-based PGIC thresholds and cumulative distribution function (CDF) plots described meaningful change. RESULTS: Items on the PSD yielded high intraclass coefficients (>0.90). Correlations were in the anticipated direction and by week 12 were moderate to strong (0.41-0.73) in magnitude, demonstrating construct validity. Average PSD item scores differed predictably and significantly between known groups. Responsiveness effect size estimates were moderate to large (0.6-1.5), and CDF plots showed the percentage of responders to be consistently higher in treatment than in placebo arms across the range of change in PSD scores. CONCLUSIONS: The PSD is reliable, valid, and responsive, and represents a valid tool to enhance treatment decisions in patients with moderate to severe plaque psoriasis.
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Medidas de Resultados Relatados pelo Paciente , Psoríase , Autorrelato , Avaliação de Sintomas , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Anticorpos Monoclonais/uso terapêutico , Anticorpos Monoclonais Humanizados , Ensaios Clínicos Fase III como Assunto , Fármacos Dermatológicos/uso terapêutico , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Estudos Multicêntricos como Assunto , Psoríase/tratamento farmacológico , Psicometria , Qualidade de Vida , Ensaios Clínicos Controlados Aleatórios como Assunto , Reprodutibilidade dos Testes , Índice de Gravidade de Doença , Inquéritos e Questionários , Adulto JovemRESUMO
OBJECTIVE: To describe the health-related quality of life (HRQOL) burden of cervical dystonia (CD) and report on the HRQOL and patient perception of treatment benefits of abobotulinumtoxinA (Dysport). DESIGN: The safety and efficacy of a single injection of abobotulinumtoxinA for CD treatment were evaluated in a previously reported international, multicenter, double-blind, randomised trial. HRQOL measures were assessed in the trial and have not been previously reported. SETTING: Movement disorder clinics in the USA and Russia. PARTICIPANTS: Patients had to have a diagnosis of CD with symptoms for at least 18â months, as well as a total Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) score of at least 30; a Severity domain score of at least 15; and a Disability domain score of at least 3. Key exclusion criteria included treatment with botulinum toxin type A (BoNT-A) or botulinum toxin type B (BoNT-B) within 16â weeks of enrolment. INTERVENTIONS: Patients were randomised to receive either 500 U abobotulinumtoxinA (n=55) or placebo (n=61). PRIMARY AND SECONDARY OUTCOME MEASURES: Efficacy assessments included TWSTRS total (primary end point) and subscale scores at weeks 0, 4, 8, 12; a pain visual analogue scale at weeks 0 and 4; and HRQOL assessed by the SF-36 Health Survey (SF-36; secondary end point) at weeks 0 and 8. RESULTS: Patients with CD reported significantly greater impairment for all SF-36 domains relative to US norms. Patients treated with abobotulinumtoxinA reported significantly greater improvements in Physical Functioning, Role Physical, Bodily Pain, General Health and Role Emotional domains than placebo patients (p≤0.03 for all). The TWSTRS was significantly correlated with Physical Functioning, Role Physical and Bodily Pain scores, for those on active treatment. CONCLUSIONS: CD has a marked impact on HRQOL. Treatment with a single abobotulinumtoxinA injection results in significant improvement in patients' HRQOL. TRIAL REGISTRATION NUMBER: The trial is registered at ClinicalTrials.gov, numbers NCT00257660 and NCT00288509.
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Inibidores da Liberação da Acetilcolina/uso terapêutico , Toxinas Botulínicas Tipo A/uso terapêutico , Distonia/tratamento farmacológico , Músculos do Pescoço , Cervicalgia/tratamento farmacológico , Satisfação do Paciente , Qualidade de Vida , Adulto , Idoso , Método Duplo-Cego , Distonia/complicações , Feminino , Nível de Saúde , Humanos , Injeções Intramusculares , Masculino , Pessoa de Meia-Idade , Cervicalgia/etiologia , Medição da Dor , Resultado do Tratamento , Adulto JovemRESUMO
BACKGROUND: Quantitative assessment of postsurgical knee motion provides sensitive measurements, but results are technical and may not be meaningful to patients. Although several knee-specific instruments exist, no patient-reported outcome (PRO) measure correlates function with improved stability, motion, satisfaction, and confidence. OBJECTIVE: To address both the above limitations by developing a PRO measure to assess the phenomenon of a "normal" knee after primary total knee arthroplasty (TKA). METHODS: A draft conceptual model linking the impact of clinical mechanics to hypothesized functional outcomes was generated after a literature review of available assessment tools. Participants aged 18 to 80 years having undergone TKA within the past 10 to 18 months were identified and screened by clinical sites to participate in phase 1 focus groups or phase 2 in-depth interviews. Participants were asked to describe their TKA experiences, including how their knee feels now, followed by cognitive debriefing of Patient's Knee Implant Performance (PKIP) draft items. RESULTS: Phase 1 results indicated that concepts of confidence, stability, and satisfaction in patients' replacement knee when performing certain activities were distinct and important in the patients' assessment of their TKA. Phase 2 efforts yielded a final version of the PKIP measure containing nine items assessing the broader concepts of stability, confidence, and satisfaction in association with activities. Presurgical and postsurgical versions of the measure were created. CONCLUSIONS: Results of this qualitative study support use of the PKIP as a complementary PRO measure to assess performance after primary TKA. Psychometric evaluation of the PKIP is planned.
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Artroplastia do Joelho/psicologia , Avaliação de Resultados em Cuidados de Saúde , Satisfação do Paciente , Amplitude de Movimento Articular/fisiologia , Idoso , Idoso de 80 Anos ou mais , Avaliação da Deficiência , Feminino , Grupos Focais , Humanos , Entrevistas como Assunto , Masculino , Pessoa de Meia-Idade , Modelos Teóricos , Medição da Dor , Pesquisa Qualitativa , Recuperação de Função Fisiológica , Autorrelato , Inquéritos e QuestionáriosRESUMO
BACKGROUND: The US Food and Drug Administration (FDA) provides formal guidance for the use of patient-reported outcomes (PROs) in support of labeling claims, whereas the European Medicines Agency (EMA) offers insight in a reflection paper relating to health-related quality of life in lieu of formal guidance. OBJECTIVES: PRO label claims granted for new molecular entities and biologic license applications from 2006 through 2010 were reviewed to evaluate consistencies and discrepancies in PRO label claims granted by the FDA and the EMA and to highlight trends in the acceptance of PRO claims across agencies. METHODS: Products approved by both the FDA and the EMA were identified. By using US Drug Approval Packages and European Public Assessment Reports packages, any PRO label claims made for the same product by the same company were compared. RESULTS: Both agencies approved a total of 75 products. Of these, 35 (47%) had at least one EMA-granted PRO label claim compared with 14 (19%) by the FDA. Most FDA-grated claims focused on symptoms; however, EMA-granted claims were more likely to include higher order concepts. Few (~12%) were granted the same label claims. Despite this discordance between the two agencies, where PRO label claims were granted by both the FDA and the EMA, there was similarity in the type of label claim. CONCLUSIONS: The EMA is more likely than the FDA to grant PRO claims and for higher order constructs. On a macro level, there appears to be poor concordance between claims granted by both agencies. On close examination, however, there appears to be greater concordance than previously recognized, which may be instructive in formulating future PRO strategies. Further research to create strategic alignment across agencies may be beneficial.
Assuntos
Aprovação de Drogas/estatística & dados numéricos , Rotulagem de Medicamentos/estatística & dados numéricos , Preparações Farmacêuticas , Resultado do Tratamento , United States Food and Drug Administration/estatística & dados numéricos , Europa (Continente) , Humanos , Qualidade de Vida , Estados UnidosRESUMO
BACKGROUND: The purpose of this research was to fully explore the impact of endpoint type (primary vs. nonprimary) on decisions related to patient-reported outcome (PRO) labeling claims supported by PRO measures and to determine if nonprimary PRO endpoints are being fully optimized.This review examines the use of PROs as both primary and nonprimary endpoints in support of demonstration of treatment benefit of new molecular entities (NMEs) and biologic license applications (BLAs) in the United States in the years 2000 to 2012. METHODS: All NMEs and BLAs approved by the Food and Drug Administration (FDA) between January 2000 and June 2012 were identified using the FDA Drug Approval Reports Web page. Generic products granted tentative approvals were excluded. For all identified products, medical review sections from publicly available drug approval packages were reviewed to identify PRO endpoint status. Product labels (indication, clinical trials sections) were reviewed to determine the number and type of PRO claim. RESULTS: A total of 308 NMEs/BLAs were identified. Of these, 70 NMEs/BLAs (23%) were granted PRO claims. The majority of product claims were for disease- or condition-specific signs and symptoms. Of the 70 products with PRO claims, a PRO was a primary endpoint for the vast majority (57 [81%]). A total of 19 of the 70 products were granted a PRO claim based on a nonprimary endpoint. While nonprimary endpoints were used most often to support claims of improved signs or symptoms, nonprimary endpoints were much more likely to support claims of higher order impacts. CONCLUSIONS: Successful PRO labeling claims are typically based on primary endpoints assessing signs and symptoms. Based on this research, studies with PROs as primary endpoints are far more likely to facilitate positive regulatory review and acceptance of PROs in support of labeling claims. Although inclusion of PROs as nonprimary endpoints in clinical trials has its challenges, recent PRO labels granted by the FDA show that they can indeed be candidates for PRO labeling claims as long as they are supported by evidence.
Assuntos
Ensaios Clínicos como Assunto , Rotulagem de Medicamentos , Avaliação de Resultados em Cuidados de Saúde , Autorrelato , Ensaios Clínicos como Assunto/normas , Humanos , Preparações Farmacêuticas/normas , Indicadores de Qualidade em Assistência à Saúde , Qualidade de Vida , Estados Unidos , United States Food and Drug AdministrationRESUMO
We propose using appropriate-use criteria (AUC) as the methodology of choice for formulating and disseminating evidence-based medicine guidelines in sports medicine and arthroscopy. AUC provide a structured process for integrating findings from the scientific literature with clinical judgment to produce explicit criteria for determining the appropriateness of specific treatments. The use of AUC will enable surgeons to treat patients in a more consistent manner based on expert clinical consensus and evidence-based medicine. This methodology also will ensure that guidelines represent all stakeholders and available evidence.
Assuntos
Artroscopia/normas , Medicina Baseada em Evidências/métodos , Guias de Prática Clínica como Assunto/normas , Melhoria de Qualidade/normas , Medicina Esportiva/normas , Consenso , Medicina Baseada em Evidências/normas , Humanos , Ensaios Clínicos Controlados Aleatórios como Assunto , Mecanismo de Reembolso/normasRESUMO
This work aimed to provide an understanding of the current use and regulatory acceptability of patient-reported outcome (PRO) measures in labeling claims for central nervous system (CNS) agents. A subset of CNS agents was identified from all New Drug Approvals and Biologic License Applications for new drugs approved in the US from January 2006 to June 2012. Clinician-reported outcomes (ClinROs) (62%) and PROs (38%) were the most widely used primary outcome measures. The PROs were frequently used in combination with ClinROs. Twelve PRO claims were granted across 41% of CNS drug approvals: 83%, symptoms; 17%, functioning. The PROs are frequently utilized as primary and secondary end points in CNS agents, and labeling claims are granted at higher levels than for non-CNS agents (41% vs 24%, respectively). These claims are granted at a lower rate than expected, given that direct patient input may lend valuable insight to treatment impacts in most CNS diseases.
RESUMO
BACKGROUND: The Knee Injury and Osteoarthritis Outcome Score (KOOS) assesses acute and chronic knee injuries or early-onset osteoarthritis in young, active patients. The United States Food and Drug Administration guidelines recommend that patient-reported outcome instruments used to support clinical trial label claims should demonstrate content validity using patient input and have acceptable psychometric properties in the target population. To use the KOOS subscales in safety and efficacy trials assessing new treatments for patients with articular cartilage lesions, additional validation work, using input from patients with articular cartilage lesions, was necessary. PURPOSE: Qualitative and quantitative evaluations of the KOOS subscales' validity among patients with articular cartilage lesions were conducted to support their use as clinically meaningful end points in clinical trials. STUDY DESIGN: Cohort study (diagnosis); Level of evidence, 2. METHODS: For qualitative analysis, cognitive interviews involving concept elicitation and cognitive debriefing with the KOOS items were conducted with 15 participants aged 25 to 52 years. Participants either were candidates for cartilage repair or had undergone cartilage repair 6 months or more before the study. For the quantitative analysis, a psychometric evaluation of the KOOS was conducted with clinical trial data from 54 patients, aged 18 to 55 years, evaluating the Cartilage Autograft Implantation System in the United States (n = 29) and the European Union (n = 25). Data were collected before surgery and at 7 postsurgical visits up to 12 months. Internal consistency and test-retest reliability, construct validity, responsiveness, and estimates of the minimal detectable change (MDC) were assessed. Test-retest reliability was assessed using data from months 2 and 3 on a subset of stable patients. RESULTS: Qualitative research confirmed that concepts measured on the KOOS are important to patients with articular cartilage lesions. Most participants reported the KOOS was comprehensive and appropriate. In the quantitative research, KOOS subscales showed excellent internal consistency reliability (range, .74-.97 at baseline) and test-retest reliability (range, .78-.82). Construct validity results supported hypothesized relationships, with significant correlations (r ≥ .50) in the expected directions. Responsiveness analyses demonstrated excellent sensitivity to change; standardized response means ranged from 0.8 to 1.2, and MDC estimates ranged from 7.4 to 12.1. CONCLUSION: The study results support the use of the KOOS subscales among patients with articular cartilage lesions.