Cost Effectiveness of Monoclonal Antibody Therapy for Rare Diseases: A Systematic Review

Park, Taehwan; Griggs, Scott K.; Suh, Dong‐Churl

doi:10.1007/s40259-015-0135-4

Cited by 17 publications

(15 citation statements)

References 37 publications

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“…Lack of translational implications brings two facts for reflection: recombinant antibody administrations are still expensive and therefore far from the governments and the main populations affected by leishmaniasis; and there are not enough scientists and funding to sponsor those clinical studies. Biological treatments are still costly and restricted to populations who can afford that ( 213 , 214 ). It is still unclear whether these treatments are going to expand and consequently drop in price so that treatment of other diseases may benefit as well.…”

Section: Translational Implicationsmentioning

confidence: 99%

Unraveling the Role of Immune Checkpoints in Leishmaniasis

Silva

Stebut

2021

Front. Immunol.

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Leishmaniasis are Neglected Tropical Diseases affecting millions of people every year in at least 98 countries and is one of the major unsolved world health issues. Leishmania is a parasitic protozoa which are transmitted by infected sandflies and in the host they mainly infect macrophages. Immunity elicited against those parasites is complex and immune checkpoints play a key role regulating its function. T cell receptors and their respective ligands, such as PD-1, CTLA-4, CD200, CD40, OX40, HVEM, LIGHT, 2B4 and TIM-3 have been characterized for their role in regulating adaptive immunity against different pathogens. However, the exact role those receptors perform during Leishmania infections remains to be better determined. This article addresses the key role immune checkpoints play during Leishmania infections, the limiting factors and translational implications.

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Section: Translational Implicationsmentioning

confidence: 99%

Unraveling the Role of Immune Checkpoints in Leishmaniasis

Silva

Stebut

2021

Front. Immunol.

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“…Since their introduction in the 1990s, monoclonal antibody (mAb) therapies have revolutionized the treatment of many diseases [40]. Often described as 'magic bullets' due to their high selectivity and specificity for their targets, they are generally well tolerated and have found great utility in the treatment of rare diseases with many being granted orphan status in both the USA and Europe, with a recent review identifying 24 already licenced mAbs with orphan status [41]. However, being complex proteins, they are costly to develop and manufacture.…”

Section: Biosimilar Medicinesmentioning

confidence: 99%

Commonly setting biological standards in rare diseases

O’Connor¹,

Buckland

Almond

et al. 2019

Expert Opinion on Orphan Drugs

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Introduction: Standardization is important across the life cycle of medicinal products, supporting the diagnosis, treatment, and prevention of a wide range of diseases. For rare diseases, standardization is even more important, as patient groups are small, presenting significant challenges in the design, conduct, analysis, and interpretation of clinical studies. It is here that standardization institutions, including the UK's National Institute for Biological Standards and Control (NIBSC), can have a key role. Areas covered: A considerable proportion of NIBSC's work supports the better understanding, diagnosis, treatment, and prevention of rare diseases. NIBSC is also part of the UK's Medicines and Health care products Regulatory Agency (MHRA), creating an agency that is uniquely placed to combine scientific and regulatory expertize for the benefit of public health. This review provides an overview of NIBSC's work in rare diseases and highlights the positive impact of the work of standardization institutions in this field. Expert opinion: Standardization in product development is key for patients with rare diseases. The work of standardization institutions is increasingly being recognized as crucial for supporting scientific and clinical advancements, and early and collaborative interactions can provide drug developers with the necessary expertize, when standards matter most.

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“…Similarly, a recent survey of oncologists indicated that many physicians have encountered barriers to accessing rituximab for treatment in patients with NHL or CLL [1]. Restrictions on or disparity in access to biologics, including rituximab, have also been found in regional analyses and assessments of patient access and cost-effectiveness in rare diseases [2, 5]. A number of factors may be involved in the patient's lack of access to rituximab for the treatment of RA, including restrictive treatment guidelines, administrative hurdles, and financial considerations, for example, insurance/public payer coverage for the biologic and the infusion facility, reimbursement, and out-of-pocket cost to the patient [1, 3, 4].…”

Section: Rituximabmentioning

confidence: 99%

“…The development of biologic therapies has transformed the treatment of a number of serious diseases; however, patient access to these life-changing therapies may be limited [1–5]. Patents and other periods of exclusivity on a number of biologics are nearing expiration or have already expired, and regulatory pathways have been established to allow the development and approval of products called “biosimilars” [6].…”

Section: Introductionmentioning

confidence: 99%

Biosimilars Have Arrived: Rituximab

2018

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A biosimilar is a biologic product that is highly similar to a licensed biologic (“originator”) such that there are no clinically meaningful differences in safety, purity, or potency between the biosimilar and the originator. As patent protection and data exclusivity for the biologic rituximab expire, several potential biosimilars to rituximab are in development, which could soon lead to the availability of numerous rituximab biosimilars. Biosimilars are evaluated using thorough and rigorous analyses of the potential biosimilar versus the originator biological to confirm similar structure, function, and clinical efficacy as well as safety. Approval of a biosimilar is based upon the totality of the evidence demonstrating similarity to the originator. An understanding of the process of the interchangeable designation of a biosimilar is important in the context of patient outcomes. We conducted an analysis of the properties and benefits of rituximab in the treatment of inflammatory diseases, the development and approval of biosimilars, and the potential benefits of rituximab biosimilars. PubMed and ClinicalTrials.gov databases were searched for “biosimilar” and “rituximab” and regulatory and pharmaceutical company web pages were screened regarding biosimilars in development and specific guidelines developed for the approval of biosimilars. The results indicate that, at present, six rituximab biosimilar candidates are undergoing comparative clinical development, and two were recently approved in the European Union. Our analysis indicates rituximab biosimilars are expected to have a continuing role in treating inflammatory conditions such as rheumatoid arthritis.

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Cost Effectiveness of Monoclonal Antibody Therapy for Rare Diseases: A Systematic Review

Cited by 17 publications

References 37 publications

Unraveling the Role of Immune Checkpoints in Leishmaniasis

Unraveling the Role of Immune Checkpoints in Leishmaniasis

Commonly setting biological standards in rare diseases

Biosimilars Have Arrived: Rituximab

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