Equity and capacity to benefit from early access to medicines schemes

Abstract Many diseases, especially rare ones, have not gained the attention or support needed to attract dedicated research interest to be able to develop successful medicines. There is, thus, a significant unmet clinical need, not all of which is (or indeed can be) addressed through the evaluation...

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Main Authors: Sarah J. L. Edwards, Paul Aliu, Joe Brierley, Søren Holm, Peter J. Pitts
Format: Article
Language:English
Published: BMC 2025-04-01
Series:International Journal for Equity in Health
Online Access:https://doi.org/10.1186/s12939-025-02416-3
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author Sarah J. L. Edwards
Paul Aliu
Joe Brierley
Søren Holm
Peter J. Pitts
author_facet Sarah J. L. Edwards
Paul Aliu
Joe Brierley
Søren Holm
Peter J. Pitts
author_sort Sarah J. L. Edwards
collection DOAJ
description Abstract Many diseases, especially rare ones, have not gained the attention or support needed to attract dedicated research interest to be able to develop successful medicines. There is, thus, a significant unmet clinical need, not all of which is (or indeed can be) addressed through the evaluation of investigational treatments introduced within the confines of clinical trials. People with severe life-threatening conditions who are not eligible to participate in any ongoing clinical trials may be able to try investigational medicines through schemes facilitating early use of or expanded access to innovative medicines. Here, we focus on the issue of equity in such programmes. Standard metrics of clinical need which inform operational decisions about equity in resource allocation primarily rely on social goods which have already been evaluated, providing evidence to support the standard assessment of patient ‘capacity to benefit’ from given medical interventions. Notions of equity have only relatively recently been discussed within research and innovation generally and within the ethics of clinical trials in particular. Considerations of equity, however, require an overview of all these different patient pathways. We suggest that a new formal method to assess eligibility for early use of or expanded access to innovative medicines be used to capture both the severity of the condition and capacity for scientific or social value alongside clinical trials.
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spelling doaj-art-18502661199e450da8aff1cb5d3a5fb12025-08-20T03:10:09ZengBMCInternational Journal for Equity in Health1475-92762025-04-012411710.1186/s12939-025-02416-3Equity and capacity to benefit from early access to medicines schemesSarah J. L. Edwards0Paul Aliu1Joe Brierley2Søren Holm3Peter J. Pitts4Center for Medicine in the Public InterestCenter for Medicine in the Public InterestCenter for Medicine in the Public InterestCenter for Medicine in the Public InterestCenter for Medicine in the Public InterestAbstract Many diseases, especially rare ones, have not gained the attention or support needed to attract dedicated research interest to be able to develop successful medicines. There is, thus, a significant unmet clinical need, not all of which is (or indeed can be) addressed through the evaluation of investigational treatments introduced within the confines of clinical trials. People with severe life-threatening conditions who are not eligible to participate in any ongoing clinical trials may be able to try investigational medicines through schemes facilitating early use of or expanded access to innovative medicines. Here, we focus on the issue of equity in such programmes. Standard metrics of clinical need which inform operational decisions about equity in resource allocation primarily rely on social goods which have already been evaluated, providing evidence to support the standard assessment of patient ‘capacity to benefit’ from given medical interventions. Notions of equity have only relatively recently been discussed within research and innovation generally and within the ethics of clinical trials in particular. Considerations of equity, however, require an overview of all these different patient pathways. We suggest that a new formal method to assess eligibility for early use of or expanded access to innovative medicines be used to capture both the severity of the condition and capacity for scientific or social value alongside clinical trials.https://doi.org/10.1186/s12939-025-02416-3
spellingShingle Sarah J. L. Edwards
Paul Aliu
Joe Brierley
Søren Holm
Peter J. Pitts
Equity and capacity to benefit from early access to medicines schemes
International Journal for Equity in Health
title Equity and capacity to benefit from early access to medicines schemes
title_full Equity and capacity to benefit from early access to medicines schemes
title_fullStr Equity and capacity to benefit from early access to medicines schemes
title_full_unstemmed Equity and capacity to benefit from early access to medicines schemes
title_short Equity and capacity to benefit from early access to medicines schemes
title_sort equity and capacity to benefit from early access to medicines schemes
url https://doi.org/10.1186/s12939-025-02416-3
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AT sørenholm equityandcapacitytobenefitfromearlyaccesstomedicinesschemes
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