ACCELERATE – Five years accelerating cancer drug development for children and adolescents

Pearson, Andrew D.J.; Weiner, Susan; Adamson, Peter C.; Karres, Dominik; Reaman, Gregory H.; Rousseau, Raphaël F.; Blanc, P.; Norga, Koen; Skolnik, Jeffrey; Kearns, Pamela; Scobie, Nicole; Barry, Elly; Marshall, Lynley V.; Knox, Leona; Caron, Hubert; Wariabharaj, Darshan; Pappo, Alberto S.; DuBois, Steven G.; Gore, Lia; Kieran, Mark W.; Weigel, Brenda; Fox, Elizabeth; Nysom, Karsten; Rojas, Teresa de; Vassal, Gilles

doi:10.1016/j.ejca.2022.01.033

Cited by 35 publications

(42 citation statements)

References 44 publications

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“…Indeed, HTA bodies have already defined patient-relevant endpoints used for relative effectiveness assessment in Europe [ 13 ]. However, it appears important to include all relevant stakeholders in the definition of trial endpoints that are relevant to patients in a specific disease setting [ 14 – 16 ], as well as determination of the appropriate comparator [ 17 ]. The question remains how to approach the challenge of what level of uncertainty may be considered acceptable in disease setting e.g., what might constitute appropriate criteria, who would be involved in making a decision, etc.…”

Section: Discussionmentioning

confidence: 99%

Shaping a research agenda to ensure a successful European health technology assessment: insights generated during the inaugural convention of the European Access Academy

Julian

Pavlovic²,

Solà-Morales³

et al. 2022

Health Econ Rev

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Objectives Key challenges for a joint European Health Technology Assessment (HTA) include consolidated approaches towards the choice of adequate comparator(s), selection of endpoints that are relevant to patients with a given disease, dealing with remaining uncertainties as well as transparent and consistent management of related processes. We aimed to further crystallize related core domains within these four areas that warrant further research and scrutiny. Methods Building on the outcomes of a previously conducted questionnaire survey, four key areas, processes, uncertainty, comparator choice and endpoint selection, were identified. At the inaugural convention of the European Access Academy dedicated working groups were established defining and prioritizing core domains for each of the four areas. The working groups consisted of ~ 10 participants each, representing all relevant stakeholder groups (patients/ clinicians/ regulators/ HTA & payers/ academia/ industry). Story books identifying the work assignments were shared in advance. Two leads and one note taker per working group facilitated the process. All rankings were conducted on an ordinal Likert Response Scale scoring from 1 (low priority) to 7 (high priority). Results Identified key domains include for processes: i) address (resource-) challenge of multiple PICOs (Patient/ Intervention/ Comparator/ Outcomes), ii) time and capacity challenges, iii) integrating all involved stakeholders, iv) conflicts and aligning between different multi-national stakeholders, v) interaction with health technology developer; for uncertainty: i) early and inclusive collaboration, ii) agreement on feasibility of RCT and acceptance of uncertainty, iii) alignment on closing evidence gaps, iv) capacity gaps; for comparator choice: i) criteria for the choice of comparator in an increasingly fragmented treatment landscape, ii) reasonable number of comparators in PICOs, iii) shape Early Advice so that comparator fulfils both regulatory and HTA needs, iv) acceptability of Indirect Treatment Comparisons (ITC), v) ensure broad stakeholder involvement in comparator selection; for endpoint selection: i) approaching new endpoints; ii) patient preferences on endpoints; iii) position of HTA and other stakeholders; iv) long-term generation and secondary use of data; v) endpoint challenges in RCTs. Conclusions The implementation of a joint European HTA assessment is a unique opportunity for a stronger European Health Union. We identified 19 domains related to the four key areas, processes, uncertainty, comparator choice and endpoint selection that urgently need to be addressed for this regulation to become a success.

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Section: Discussionmentioning

confidence: 99%

Shaping a research agenda to ensure a successful European health technology assessment: insights generated during the inaugural convention of the European Access Academy

Julian

Pavlovic²,

Solà-Morales³

et al. 2022

Health Econ Rev

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“…Greater reliance on international multi stakeholder collaboration has been functioning in ACCELERATE, a model since 2015 among academia, advocacy, industry, and regulatory agencies to improve and accelerate new drug development for children and adolescents with cancer in what is described as “a patient-centric organisation to solve problems.” The ACCELERATE Paediatric Strategy Forums are run in partnership with the EMA with the Food and Drug Administration (FDA) with the goal to share information in a pre-competitive setting between all stakeholders, to evaluate science on a malignancy or class of compounds, and discuss the landscape of assets in development in the light of defined unmet medical need. This can, it was argued, inform paediatric drug development strategies and subsequent decisions in a way that improves the selection and prioritisation of innovative drugs being evaluated, so that the process is driven by science and by patients’ unmet needs [ 46 ].…”

Section: Discussion: Different Challenges and Opportunitiesmentioning

confidence: 99%

Towards Better Pharmaceutical Provision in Europe—Who Decides the Future?

et al. 2022

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Significant progress has been achieved in human health in the European Union in recent years. New medicines, vaccines, and treatments have been developed to tackle some of the leading causes of disease and life-threatening illnesses. It is clear that investment in research and development (R&D) for innovative medicines and treatments is essential for making progress in preventing and treating diseases. Ahead of the legislative process, which should begin by the end of 2022, discussions focus on how Europe can best promote the huge potential benefits of new science and technology within the regulatory framework. The challenges in European healthcare were spelled out by the panellists at the roundtable organised by European Alliance for Personalised Medicine (EAPM). Outcomes from panellists’ discussions have been summarized and re-arranged in this paper under five headings: innovation, unmet medical need, access, security of supply, adapting to progress, and efficiency. Some of the conclusions that emerged from the panel are a call for a better overall holistic vision of the future of pharmaceuticals and health in Europe and a collaborative effort among all stakeholders, seeing the delivery of medicines as part of a broader picture of healthcare.

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“…As a result, the current incentives are suboptimal for these companies and mainly benefit large pharmaceutical companies. However, smaller biotechnology companies are arguably major drivers of early innovation and have the potential to provide novel drugs to children with cancer, but because of their financial structure, cannot afford to wait for late rewards 7 . This would align with both our proposals (1 and 2) to change to a segmented reward approach, in which early rewards are offered as part of the PMR and of the Orphan Regulation.…”

Section: Revising Incentives To Accelerate Paediatric Cancer Drug Dev...mentioning

confidence: 91%

“…ACCELERATE, an international paediatric oncology platform involving multiple stakeholders (academia, industry, regulatory bodies and patients and families) was established to hasten paediatric oncology drug development within the current regulatory framework 5,19 . A Working Group of ACCELERATE was convened to propose more effective incentives for paediatric‐specific oncology drug development 7 . This Working Group's conclusions are very timely in view of the ongoing revision of the European PMR and Orphan regulations.…”

Section: Introductionmentioning

confidence: 99%

Changing incentives to ACCELERATE drug development for paediatric cancer

Rojas¹,

Kearns

Blanc³

et al. 2023

Cancer Medicine

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Background: More effective incentives are needed to motivate paediatric oncology drug development, uncoupling it from dependency on adult drug development. Although the current European and North-American legislations aim to promote drug development for paediatrics and rare diseases, children and adolescents with cancer have not benefited as expected from these initiatives and cancer remains the first cause of death by disease in children older than one. Drug development for childhood cancer remains dependent on adult cancer indications and their potential market. The balance between the investment needed to execute a How to cite this article: de Rojas T, Kearns P, Blanc P, et al. Changing incentives to ACCELERATE drug development for paediatric cancer.

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ACCELERATE – Five years accelerating cancer drug development for children and adolescents

Cited by 35 publications

References 44 publications

Shaping a research agenda to ensure a successful European health technology assessment: insights generated during the inaugural convention of the European Access Academy

Shaping a research agenda to ensure a successful European health technology assessment: insights generated during the inaugural convention of the European Access Academy

Towards Better Pharmaceutical Provision in Europe—Who Decides the Future?

Changing incentives to ACCELERATE drug development for paediatric cancer

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