Latent therapeutic demand model for the immunoglobulin replacement therapy of primary immune deficiency disorders in the <scp>USA</scp>

Stonebraker, Jeffrey S.; Hajjar, Joud

doi:10.1111/vox.12651

Cited by 6 publications

(12 citation statements)

References 60 publications

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Section: Discussionsupporting

confidence: 51%

“…This study augments previous work by Stonebraker and colleagues in which decision analysis methodology is used to estimate the unconstrained demand for plasma protein therapies [6, 7, 16]. The level of uncertainty and professional variation underpinning many of the key elements contributing to demand enable this methodology, through the sensitivity analyses which are a feature of the model, to synthesize the various values offered through clinical research and professional into an estimate for LTD.…”

Section: Discussionmentioning

confidence: 74%

“…For modelling the LTD for IG in the three neuropathies described in the Introduction, we used the decision analysis methodology similar to the methods developed by Stonebraker et al [6,7] for modelling the LTD of IVIG in PID. The variables used in the model are shown in Tables 1 and 2.…”

Section: Model Structurementioning

confidence: 99%

“…Estimating the potential demand for IG indications is important for adequate planning of plasma collection and management of the limited supply. In previous work, the concept of latent therapeutic demand (LTD) has been used to estimate the requirements for IG in PID [6,7] in the hypothetical scenario when usage is unconstrained by supply and reimbursement.…”

Section: Introductionmentioning

confidence: 99%

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Estimation of the latent therapeutic demand for immunoglobulin therapies in autoimmune neuropathies in the United States

Farrugia

Bansal

Marjanović³

2021

Vox Sanguinis

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Background and Objectives The use of immunoglobulin (IG) solutions as an immunomodulatory therapy in certain neurological conditions has become an established modality and represents a significant proportion of total IG use. The estimation of the evidence‐based potential demand designated as the latent therapeutic demand (LTD) for IG in these diseases is required for adequate planning of the plasma supply required to manufacture the product. Materials and Methods The diseases studied included chronic inflammatory demyelinating polyneuropathy (CIDP), Guillain–Barré syndrome (GBS) and multifocal motor neuropathy (MMN). The LTD for IG was assessed using a decision analysis model, using Microsoft Excel. The model analysed the epidemiological and clinical factors contributing to IG usage. One‐way sensitivity analysis and probabilistic sensitivity analysis derived the LTD in grams per 1000 inhabitants. The key variables included the treatment schedule and the prevalence of the disease. Results The model estimates that an average annual IG demand and standard deviation for CIDP, GBS and MMN in the United States is 83.05 ± 24.5, 6.1 ± 3.2 and 36.1 ± 25.5 g/1000 inhabitants, respectively. Conclusion Together with previous work on the LTD for IG in immunodeficiencies, these results indicate that current IG usage reflects the estimated LTD for the main indications for IG in the United States The wide range of LTD found in all these studies emphasizes the need for more precise assessment of the underlying variables, particularly disease prevalence and dosage. Further studies on other indications such as secondary immunodeficiencies will augment these results and will assist in guiding demand planning for IG use and plasma collection in the United States and inform blood policy in other countries.

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Section: Discussionsupporting

confidence: 51%

Section: Discussionmentioning

confidence: 74%

Section: Model Structurementioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Estimation of the latent therapeutic demand for immunoglobulin therapies in autoimmune neuropathies in the United States

Farrugia

Bansal

Marjanović³

2021

Vox Sanguinis

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“…The combined evidence based underlying demand for IG in these necrologies approaches, therefore, 140 g/1000 inhabitants. Combined with the estimated demand of 105 g/1000 population for the primary immune deficiencies 23 , this suggests that the amount of IG used currently in the countries using high amounts IG reflects the consumption expected from decision analysis modelling. As diagnosis and treatment access improve in other, lower consuming countries, the actual usage is expected to approach the levels estimated in these models.…”

Section: Discussionmentioning

confidence: 92%

Latent Therapeutic Demand of Immunoglobulin therapies in neuropathies – chronic inflammatory demyelinating polyneuropathy (CIDP), Guillain–Barré syndrome (GBS) and multifocal motor neuropathy (MMN) in the United States

Farrugia

Bansal

2020

Preprint

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Chronic inflammatory demyelinating polyneuropathy (CIDP) is a neurological disorder characterized by progressive weakness and impaired sensory function in the legs and arms. Guillain-Barre syndrome is a disorder in which the body's immune system attacks part of the peripheral nervous system. The first symptoms of this disorder include varying degrees of weakness or tingling sensations in the legs (NIH). Multifocal motor neuropathy is a progressive muscle disorder characterized by muscle weakness in the hands, with differences from one side of the body to the other in the specific muscles involved (NIH). We have modeled a latent therapeutic demand (LTD) of IVIg for CIDP and similar neuropathies in the US. We used the decision analysis methodology similar to the methods used by Stonebraker et al 1 for modeling LTD of IVIg. The model is based on the relationships of the epidemiological and clinical factors. Most of the usage patterns and dosage level of albumin are according to the epidemiological studies and clinical trials. The model is built in Microsoft Excel. The analysis is conducted based on one-way sensitivity analysis and probabilistic sensitivity analysis. The demand in terms of grams per 1,000 inhabitants is calculated depending on the treatment schedule and the prevalence of the disease. The model for CIDP has eight variables including prevalence of CIDP, patients using IVIg, dosage and treatment patterns. The annual demand of IVIg is based on initial treatment of 24 weeks followed by a maintenance period, with lower dosage and frequency of treatment for another 24 weeks 2. The model for GBS has eight variables with a loading dose for 3-6 days followed by a second dose in case of relapse. The model for MMN has nine variables. It has a loading dose followed by maintenance dose every 1-6 weeks depending on the clinical factors of the patient. On an average, IVIg use was calculated as 100 gms, 5.6 gms and 35 gms per 1,000 inhabitants for CIDP, GBS and MMN, respectively, in the US annually.

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The Importance of Primary Immune Deficiency Registries

Lopes

Cunningham‐Rundles

2020

Immunology and Allergy Clinics of North America

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Latent therapeutic demand model for the immunoglobulin replacement therapy of primary immune deficiency disorders in the USA

Abstract: Estimating country-specific LTD is important to ensure an adequate supply of IgGRT and an optimal treatment for patients with PIDDs and for improving national healthcare policymaking and production planning.

Cited by 6 publications

References 60 publications

Estimation of the latent therapeutic demand for immunoglobulin therapies in autoimmune neuropathies in the United States

Estimation of the latent therapeutic demand for immunoglobulin therapies in autoimmune neuropathies in the United States

Latent Therapeutic Demand of Immunoglobulin therapies in neuropathies – chronic inflammatory demyelinating polyneuropathy (CIDP), Guillain–Barré syndrome (GBS) and multifocal motor neuropathy (MMN) in the United States

The Importance of Primary Immune Deficiency Registries

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