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Van Calcar S.C.,University of Wisconsin - Madison | Bernstein L.E.,University of Colorado at Denver | Rohr F.J.,Harvard University | Scaman C.H.,University of British Columbia | And 2 more authors.
Molecular Genetics and Metabolism | Year: 2014

The galactose-restricted diet is life-saving for infants with classic galactosemia. However, the benefit and extent of dietary galactose restriction required after infancy remain unclear and variation exists in practice. There is a need for evidence-based recommendations to better standardize treatment for this disorder. This paper reviews the association between diet treatment and outcomes in classic galactosemia and evaluates the contribution of food sources of free galactose in the diet. Recommendations include allowing all fruits, vegetables, legumes, soy products that are not fermented, various aged cheeses and foods containing caseinates. Further research directions are discussed. © 2014 Elsevier Inc. Source


Halpern R.,Health Economics and Outcomes Research | Agarwal S.,Biogen Idec | Dembek C.,Biogen Idec | Borton L.,Health Economics and Outcomes Research | Lopez-Bresnahan M.,Medical and Scientific Affairs
Patient Preference and Adherence | Year: 2011

Purpose: To compare adherence and persistence among patients with multiple sclerosis (MS) initiated on disease-modifying therapy (DMTs), including intramuscular (IM) interferon beta-1a (IFNβ-1a), subcutaneous (SC) IFNβ-1a, IFNβ-1b, or glatiramer acetate (GA). Methods: MS patients initiated on IM-IFNβ-1a, SC-IFNβ-1a, IFNβ-1b, or GA between January 1, 2000 and January 2, 2008 were identified from a retrospective claims database study associated with a large US health plan. The date of DMT initiation was the index date; patients were observed for 6 months before and 12-36 months after the index date. Adherence to the index DMT was measured with a medication possession ratio (MPR), the proportion of days patients possessed their index DMTs; MPR $ 0.80 was considered adherent. Persistence was time in days from index date until the earlier of a minimum 60-day gap in DMT therapy or the last DMT claim during follow-up. Adherence and persistence were modeled with logistic and Cox proportional hazard regressions, respectively. Results: The study population comprised 6,680 patients in the DMT cohorts: IM-IFNβ-1a (N = 2,305, 34.5%); IFNβ-1b (N = 894, 13.4%); GA (N = 2,270, 34.0%); and SC-IFNβ-1a (N = 1,211, 18.1%). The IM-IFNβ-1a cohort had significantly higher regression-adjusted odds of adherence relative to the other cohorts: 52.4% higher odds versus the IFNβ-1b cohort (OR = 0.656, CI = 0.561-0.768); 33.5% higher odds versus the GA cohort (OR = 0.749, CI = 0.665-0.844); and 20.6% higher odds versus the SC-IFNβ-1a cohort (OR = 0.829, CI = 0.719-0.957). There were no consistent differences in persistence between the cohorts. Conclusion: IM-IFNβ-1a patients had significantly higher odds of adherence compared with other DMT cohorts, possibly attributable to IM-IFNβ-1a's less frequent dosing schedule. The benefits of adherence may include better quality of life, lower risk of relapse, and fewer hospitalizations and emergency visits, making adherence a critical component of MS management. © 2011 Halpern et al. Source


Teagarden J.R.,Medical and Scientific Affairs
Journal of Managed Care Pharmacy | Year: 2014

Necessity and fairness require that health plans limit the products and services they cover. The basis for these decisions refers to population averages and related population parameters. However, individuals vary and may not be accurately represented by the parameters used to establish coverage policies. Health plans, therefore, are obligated to anticipate and manage heterogeneity among their member groups. This commentary offers considerations for managing heterogeneity in prescription drug benefits. © 2014, Academy of Managed Care Pharmacy. Source


Teagarden J.R.,Medical and Scientific Affairs
Expert Opinion on Orphan Drugs | Year: 2014

Scientific breakthroughs themselves often do not carry the force needed for acceptance or adoption. By their very nature, scientific breakthroughs challenge the status quo or Normal Science. Therefore, those who forge scientific breakthroughs will have to break through Normal Science if their discoveries are to be adopted. Needing to break through Normal Science may not come as a surprise to people educated and training in the biosciences, but it will to many people who come to rare diseases from outside the biosciences and through necessity. In this commentary, I describe the basis and components of Normal Science, and how it can impede the progress of scientific breakthroughs. I refer to Thomas Kuhn's concept of the 'structure of scientific revolutions' as an explanatory model, and I use three case studies to illustrate it. I also provide ideas on measures that can be taken to help people in rare disease research recognize and prepare for the resistance Normal Science may pose for their breakthroughs. © 2014 Informa UK, Ltd. Source


Olivares J.M.,Complejo Hospitalario Universitario Of Vigo | Sermon J.,Janssen Cilag SAS | Hemels M.,ACCESS Europe | Schreiner A.,Medical and Scientific Affairs
Annals of General Psychiatry | Year: 2013

Relapse in patients with schizophrenia has devastating repercussions, including worsening symptoms, impaired functioning, cognitive deterioration and reduced quality of life. This progressive decline exacerbates the burden of illness on patients and their families. Relapse prevention is identified as a key therapeutic aim; however, the absence of widely accepted relapse definition criteria considerably hampers achieving this goal. We conducted a literature review in order to investigate the reporting of relapses and the validity of hospitalization as a proxy for relapse in patients with schizophrenia. The primary aim was to assess the range and validity of methods used to define relapse in observational or naturalistic settings. The secondary aim was to capture information on factors that predicted or influenced the risk of relapse. A structured search of the PubMed database identified articles that discussed relapse, and hospitalization as a proxy of relapse, in patients with schizophrenia. National and international guidelines were also reviewed. Of the 150 publications and guidelines identified, 87 defined relapse and 62% of these discussed hospitalization. Where hospitalization was discussed, this was as a proxy for, or a component of, relapse in the majority of cases. However, hospitalization duration and type varied and were not always well defined. Scales were used to define relapse in 53 instances; 10 different scales were used and multiple scales often appeared within the same definition. There were 95 references to factors that may drive relapse, including non-adherence to antipsychotic medication (21/95), stress/depression (11/95) and substance abuse (9/95). Twenty-five publications discussed the potential of antipsychotic therapy to reduce relapse rates-continuous antipsychotic therapy was associated with reduced frequency and duration of hospitalization. Non-pharmacological interventions, such as psychoeducation and cognitive behavioural therapy, were also commonly reported as factors that may reduce relapse. In conclusion, this review identified numerous factors used to define relapse. Hospitalization was the factor most frequently used and represents a useful proxy for relapse when reporting in a naturalistic setting. Several factors were reported to increase the risk of relapse, and observation of these may aid the identification of at-risk patients. © 2013 Olivares et al.; licensee BioMed Central Ltd. Source

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