Early access to health products in France: Major advances of the French “Conseil stratégique des industries de santé” (CSIS) to be implemented (modalities, regulations, funding)

Albin, Nicolas; Chassagnol, Frédéric; Bergmann, Jean‐François; Boudali, Lotfi; Blin, Olivier; Chalus, Thibault de; Fruchet, Estelle; Grande, Mathilde; Grude, F.; Hoog-Labouret, Natalie; Kauv, Céline; Lavie, Frédéric; Jeunne, Claire Le; Meillier, Fabrice; Pouletty-Lefebvre, Brigitte; Richard, Anne-Céline; Mazaleyrat, B.; Monard, Adrien

doi:10.1016/j.therap.2018.12.002

Cited by 7 publications

(7 citation statements)

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“…Within their report on the implementation of the EMA-EUnetHTA work plan 2017–2021 EMA and EUnetHTA provide in-depth insights regarding their discussion of controversial methodological challenges [ 15 ]. Continuation of those discussions and clarification of evidence requirements within the regulatory and HTA framework including parallel early advice, early access schemes, as well as regulatory and HTAs is urgently required to ‘ensuring a strong EU voice globally’ in line with chapter 5 of the European Commission’s pharmaceutical strategy for Europe [ 13 , 16 , 17 ]. The management of uncertainty after successful EMA approval leading to additional national data requirements as a toll needed for covering the gaps and discrepancies between the regulatory and HTA data needs is another key challenge affecting any future oncology HTA within Europe [ 18 ].…”

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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How can a joint European health technology assessment provide an ‘additional benefit’ over the current standard of national assessments?

et al. 2022

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Objectives We conducted a multi-stakeholder survey to determine key areas where a joint European health technology assessment (HTA) could provide ‘additional benefit’ compared to the status quo of many parallel independent national and subnational assessments. Methods Leveraging three iterative Delphi cycles, a semiquantitative questionnaire was developed covering evidence challenges and heterogeneity of value drivers within HTAs across Europe with a focus on hematology/oncology. The questionnaire consisted of five sections: i) background information; ii) value drivers in HTA assessments today; iii) evolving evidence challenges; iv) heterogeneity of value drivers across Europe; v) impact of Europe’s Beating Cancer Plan (EBCP). The questionnaire was circulated across n = 189 stakeholder institutions comprising HTA and regulatory bodies, clinical oncology associations, patient representatives, and industry associations. Results N = 30 responses were received (HTA bodies: 9; regulators: 10; patients’ and physicians’ associations: 3 each; industry: 5). Overall, 17 countries and EU level institutions were represented in the responses. Consistency across countries and stakeholder groups was high. Most relevant value drivers in HTAs today (scale 1, low to 5, high) were clinical trial design (mean 4.45), right endpoints (mean 4.40), and size of comparative effect (mean 4.33). Small patient numbers (mean 4.28) and innovative study designs (mean 4.1) were considered the most relevant evolving evidence challenges. Heterogeneity between regulatory and HTA evidence requirements and heterogeneity of the various national treatment standards and national HTA evidence requirements was high. All clinical and patient participants stated to have been with EBCP initiatives. Conclusions For a European HTA to provide an ‘additional benefit’ over the multitude of existing national assessments key methodological and process challenges need to be addressed. These include approaches to address uncertainty in clinical development; comparator choice; consistency in approaching patient-relevant endpoints; and a transparent and consistent management of both HTA and regulatory procedures as well as their interface, including all involved stakeholder groups.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

How can a joint European health technology assessment provide an ‘additional benefit’ over the current standard of national assessments?

et al. 2022

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“…Other schemes in Europe allow the pharmaceutical industry to access a market pre-authorization and derive revenue from a product, albeit for a short period of time. An example is the French Temporary Utilisation Program (Autorisation Temporaire d’Utilisation, ATU), which can be used for individuals or cohorts of patients and allows the manufacturer to set the price of the product freely for 1 year, with any variation in the price paid back if there is a difference post-launch ( 40 , 41 ).…”

Section: Discussionmentioning

confidence: 99%

Early access provision: Awareness, educational needs and opportunities to improve oncology patients’ access to care

Krendyukov

Singhvi²,

Green-Morrison³

et al. 2022

Front. Oncol.

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BackgroundAn unmet medical need exists for many oncology patients who cannot be treated satisfactorily by available therapeutic options. Early access provision (EAP) is endorsed by competent authorities to improve patient access to innovative medicinal products (InMPs). This paper determined awareness and understanding among practicing physicians of integrated EAP protocols, and of the procedures involved in EAP applications for oncology trials prior to marketing authorization.MethodsAn on-line, fully anonymous survey reaching out to more than 3,258 physicians (including practicing oncologists) was initiated between November 2020 - January 2021. Participants were questioned about their knowledge and understanding of EAP and the decision processes involved, level of experience, interest for further educational activities and opportunities to improve the process, both in general and specifically during the COVID-19 pandemic. The frequency of EAP protocols for oncology InMPs was identified by a search of ClinicalTrials.gov and EU Clinical Trials registers.ResultsSurvey results (75% oncologists) indicated 75% of respondents were ‘very comfortable’ or ‘comfortable’ with using EAP for their patients, but only 54.5% correctly answered the specific knowledge-based question related to the EAP definition. For 56% of respondents, experience with EAP in daily practice was very limited. Two-thirds indicated an average or lower level of understanding about the application process and regulatory requirements involved (65.2% and 66.0%, respectively). Knowledge on data collection and serious adverse event reporting under EAP was lower at 57.8% and 50.5% of respondents, respectively. Awareness of physician responsibilities was high in 59.7% of respondents, but fewer understood roles and responsibilities of manufacturing companies (31.2%). Most indicated they would consider clinical efficacy and safety data from comparative phase III randomized controlled trials as of high importance to support their decision to apply for EAP (93.4% and 86.8%, respectively). During the COVID-19 pandemic, the majority of respondents highlighted the need to improve and adapt EAP with regard to the application process and documentation (83.8%), InMP supply and logistics (88.4), and safety reporting process (78.0%). Of identified oncology trials with a ClinicalTrials.gov protocol, only 149 (0.4%) included EAP, and 23 used the data to receive a marketing authorization during the period Jan 2015 to December 2020. Of oncology trials with a EudraCT protocol, only 21 (0.23%) included EAP, of which 6 were used to receive a conditional or full marketing authorisation over the same period.ConclusionUse of EAP in daily practice remains limited. Challenges posed by the EAP process, together with a lack of education on this topic, might contribute to its under-utilization and influence access of oncology patients to care. Continuous educational efforts from different stakeholders are required to better inform and support practicing oncologists during the EAP application process and regulatory framework follow up. Education should also be provided on EAP roles and responsibilities, monitoring, and potential adaptations when faced with specific challenges, such as the current COVID-19 pandemic.

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“…To address financing limitations and accelerate access to orphan drugs with pending authorization or under development, several countries adopt alternative access schemes [26]. Examples are, compassionate use programs (CUP) provided by pharmaceutical companies and medical need programs (MNP), such as the French ' Autorisation Temporaire d'Utilisation d'extension' (ATU) [27][28][29]. In some instances, people living with a rare disease are treated with a medicinal product not yet indicated or authorized for their specific condition under, so-called, off-label use [30].…”

Section: Introductionmentioning

confidence: 99%

Exploring alternative financing models and early access schemes for orphan drugs: a Belgian case study

Abdallah

Claes

Huys

et al. 2022

Orphanet J Rare Dis

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Background Although some jurisdictions have implemented particular adjustments to accommodate often-expensive orphan drugs in their healthcare systems, availability of these drugs remains complex. This study investigates alternative financing models and early access schemes for orphan drugs in the context of the Belgian healthcare system. Methods Three focus group discussions were held with a panel of eleven experts from the Belgian Drug Reimbursement Committee, the Colleges for Orphan Drugs, the pharmaceutical industry, physicians, ethicists and pharmacists. Retrieved data were pseudonymised, analysed and coded according to the Qualitative Analysis Guide of Leuven. Results Experts disfavoured the insulated fund as well as private insurance for financing orphan drugs, as, respectively, isolation of a separate budget and a mostly profit-driven mechanism would contradict the Belgian fundamental principle of solidarity. Moreover, an insulated fund could, albeit on a smaller scale, reproduce the same budgetary constraints as the general reimbursement system. As the Special Solidarity Fund is intended for urgent care and exclusively accommodates financial needs subject to eligibility criteria, its design would not allow general financing of orphan drugs. Overall, implementation of an alternative financing model was not endorsed, instead, improving the current reimbursement system was preferred. Suggestions mentioned were; increased collaboration and transparency, robust and quality real-world evidence but also digitalization of data. Alleviating administrative burden and simplifying the admission process of compassionate use program, medical need program and early treatment reimbursement should be prioritized to facilitate early access. Furthermore, a legal framework for off-label use could stimulate proper implementation. Efforts on collaboration of expertise centres and coordination of orphan drug databases across Europe could foster a robust data network to support orphan drug availability in individual countries. Conclusions This research reveals that reassessing current financing models and early access schemes by eliminating inadequacies, may be more conducive than establishing alternative systems to increase availability of orphan drugs in Belgium. Other jurisdictions may rely on this information to review their own models of early access and financing to cultivate a more sustainable delivery of orphan drugs.

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Early access to health products in France: Major advances of the French “Conseil stratégique des industries de santé” (CSIS) to be implemented (modalities, regulations, funding)

Cited by 7 publications

References 4 publications

How can a joint European health technology assessment provide an ‘additional benefit’ over the current standard of national assessments?

How can a joint European health technology assessment provide an ‘additional benefit’ over the current standard of national assessments?

Early access provision: Awareness, educational needs and opportunities to improve oncology patients’ access to care

Exploring alternative financing models and early access schemes for orphan drugs: a Belgian case study

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