Harmonising patient-access programmes: the Dutch DRUG Access Protocol platform

Zeverijn, Laurien J.; Doorn-Khosrovani, Sahar van Waalwijk van Barjesteh; Roy, A; Timmers, Lonneke; Tran, Thuy-An; Boer, Jolanda E de; Wit, Gijsbrecht F de; Geurts, Birgit S; Gelderblom, Hans; Verheul, H.; Blijlevens, N.M.A.; Wymenga, A.N.M.; Eskens, Ferry A.L.M.; Smit, Egbert F.; Bloemendal, Haiko J.; Voest, Emile E.

doi:10.1016/s1470-2045(21)00707-5

Cited by 21 publications

(13 citation statements)

References 6 publications

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“…For many years, new cancer treatments have been introduced in Italy through managed-entry, risk-sharing, and payment-by-result agreements with pharmaceutical companies [ 10 ]. Shared cost/variable cohort models, such as DRUP [ 11 ], would be a logical extension of standard negotiation. At the same time, the Italian MTB network could ideally provide overarching governance and links to international initiatives/regulatory bodies.…”

Section: Discussionmentioning

confidence: 99%

Bridging therapeutic opportunities: a survey by the Italian molecular tumor board workgroup of Alliance Against Cancer

Ciliberto

Canfora

Terrenato

et al. 2022

J Exp Clin Cancer Res

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Background Molecular tumor boards (MTBs) match molecular alterations with targeted anticancer drugs upon failure of the available therapeutic options. Special and local needs are most likely to emerge through the comparative analysis of MTB networks, but these are rarely reported. This manuscript summarizes the state-of-art of 16 active Italian MTBs, as it emerges from an online survey curated by Alliance Against Cancer (ACC). Main text Most MTBs (13/16) are exclusively supported through local Institutional grants and meet regularly. All but one adopts a fully virtual or a mixed face-to-face/virtual calling/attendance meeting model. It appears that the ACC MTB initiative is shaping a hub-and-spoke virtual MTB network reminiscent of non-redundant, cost-effective healthcare organization models. Unfortunately, public awareness of MTB opportunities presently remains insufficient. Only one center has a website. Dedicated e-mail addresses are for the exclusive use of the MTB staff. More than half of ACC members consider a miscellanea of most or all solid and hematological malignancies, and more than one-third consider neoplasms arising at any anatomical location. The average number of Staff Members in MTBs is 9. More than 10 staff members simultaneously attend MTB meetings in 13 MTBs. A medical oncologist is invariably present and is in charge of introducing the clinical case either with (45%) or without previous discussion in organ-specific multidisciplinary Boards. All but two MTBs take charge of not only patients with no standard-of-care (SoC) therapy option, but also cases receiving NGS profiling in SoC settings, implying a larger number of yearly cases. All MTBs run targeted NGS panels. Three run whole-exome and/or RNAseq approaches. ESCAT-ESMO and/or Onco-KB levels of evidence are similarly used for diagnostic reporting. Most MTBs (11) provide a written diagnostic report within 15 days. Conclusions are invariably communicated to the patient by the medical oncologist. Conclusions MTB networking is crucial not only for molecular diagnosis and therapy assignment, but also for healthcare governance. Survey results show that MTBs review therapeutic opportunities at the crossover between standard-of-care with off-label, the former task being much beyond their scope. Societal and scientific implications of this beyond-the-scope MTB function may be relevant for healthcare in Italy and abroad.

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Section: Discussionmentioning

confidence: 99%

Bridging therapeutic opportunities: a survey by the Italian molecular tumor board workgroup of Alliance Against Cancer

Ciliberto

Canfora

Terrenato

et al. 2022

J Exp Clin Cancer Res

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“…Drug access protocol (DAP) is a programme in the Netherlands to combine earlier access to medicine with structured data collection [36]. The benefits of the DAP in providing conditional reimbursement of registered drugs and thereby creating access are evident.…”

Section: Resource Implications and Politicsmentioning

confidence: 99%

Controversies in the management of patients with soft tissue sarcoma: Recommendations of the Conference on State of Science in Sarcoma 2022

Rothermundt¹,

Andreou²,

Blay³

et al. 2023

European Journal of Cancer

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“…In France, a system for expanded access has been in place since 1994 known as the 'Autorisation temporaire d'utilisation' (ATU), which allows for early access to investigational treatments in exceptional circumstances prior to a 'marketing authorisation' (MA) decisions by the EMA (Jacquet et al, 2021). As part of this process, the product in question is subject to an authorisation for use granted by the National Agency of Medicine and Health Product Safety More recently, in the Netherlands, another approach has been developed for the expanded access use of investigational anticancer drugs known as the 'DRUG Access Protocol' (DAP) (Zeverijn et al, 2022). This protocol aims to coordinate on a national level, access to novel anticancer drugs while supporting research by obtaining additional data on drug safety and activity in a real-world setting (Zeverijn et al, 2022).…”

Section: Declarations Data Availabilitymentioning

confidence: 99%

“…As part of this process, the product in question is subject to an authorisation for use granted by the National Agency of Medicine and Health Product Safety More recently, in the Netherlands, another approach has been developed for the expanded access use of investigational anticancer drugs known as the 'DRUG Access Protocol' (DAP) (Zeverijn et al, 2022). This protocol aims to coordinate on a national level, access to novel anticancer drugs while supporting research by obtaining additional data on drug safety and activity in a real-world setting (Zeverijn et al, 2022). Through this system, treatment is provided free of charge for up to 4 months by a pharmaceutical company, provided the relevant drug meets a prede ned clinically meaningful bene t threshold.…”

Section: Declarations Data Availabilitymentioning

confidence: 99%

The Impact of Expanded Access Programs for Systemic Anticancer Therapy in an Irish Cancer Centre

Cronin

Ronayne

O'Donovan

et al. 2023

Preprint

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Purpose Expanded access programs (EAPs) allow patients with cancer who have unmet clinical needs to obtain access to pre-authorisation treatments. There is no standardised process or registry for implementing these programs nationally and real world data on their impact is lacking. We evaluated their prevalence and impact in a cancer centre.Methods Data relating to adult cancer patients treated via EAPs from 2011 to 2021 in three Cork university hospitals was collated. Descriptive statistics were employed to get an overview of the impact these programs currently have on cancer care provision.Results We identified 193 patients who accessed EAPs during the study period, availing of 33 separate drugs for a total of 50 different cancer indications. The prevalence of EAP usage was shown to have been trending upwards in recent years with a total of 189 programs being accessed throughout the period. Drugs provided were from a number of different anti-cancer drug classes, particularly targeted therapies (n = 18) and immune checkpoint inhibitors (n = 17). Cancers from a wide range of both solid and liquid tumour types were identified as having been treated with EAP drugs and patients treated were from across a broad spectrum of ages (26–82, SD:11.99).Conclusion EAPs have an increasing role in access to novel cancer therapies in our community and by extension nationally. Equity of EAP access would be facilitated by a national registry of available agents which we have now established. Assessment of their benefits and toxicities would be enhanced by the requirement for a real world data base as a condition of EAP approval.

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Harmonising patient-access programmes: the Dutch DRUG Access Protocol platform

Cited by 21 publications

References 6 publications

Bridging therapeutic opportunities: a survey by the Italian molecular tumor board workgroup of Alliance Against Cancer

Bridging therapeutic opportunities: a survey by the Italian molecular tumor board workgroup of Alliance Against Cancer

Controversies in the management of patients with soft tissue sarcoma: Recommendations of the Conference on State of Science in Sarcoma 2022

The Impact of Expanded Access Programs for Systemic Anticancer Therapy in an Irish Cancer Centre

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