National early access programs and clinical trials: What opportunities for early access to therapeutic innovations for patients with malignant melanoma?

Kempf, Emmanuelle; Zalcman, Gérard; Lebbe, Célèste

doi:10.1002/cncr.33495

Cited by 2 publications

(5 citation statements)

References 19 publications

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“…Reimbursement is complicated by uncertainties across different treatments in upcoming new techniques and biomarkers. A temporary use authorisation system provides access to innovative drugs, allowing access for patients with cancer who have no therapeutic option to off-label and as-yet-unavailable drugs [ 22 ].…”

Section: Resultsmentioning

confidence: 99%

“…Novel therapeutic approaches now appearing are improving patients’ overall survival and quality of life and making early access to treatment—and accurate targeting—all the more important [ 104 , 107 ]. An international survey across 30 European countries concluded that 27% of 19,600 patients with advanced MM did not get access to the standard first-line therapy, often because of long delays between marketing authorisation and drug reimbursement [ 22 , 108 ]. Guidelines from European medical organisations promote the integration of care between medical and paramedical specialities and better management of MM from primary melanoma diagnosis through advanced disease palliation.…”

Section: Resultsmentioning

confidence: 99%

“…Equity of access to new anticancer drugs is consequently not guaranteed. Hospital pharmacies have formed consortia to negotiate prices with pharmaceutical companies directly [ 22 , 111 ].…”

Section: Resultsmentioning

confidence: 99%

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How Can the EU Beating Cancer Plan Help in Tackling Lung Cancer, Colorectal Cancer, Breast Cancer and Melanoma?

et al. 2022

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Cancer is the second leading cause of mortality in EU countries, and the needs to tackle cancer are obvious. New scientific understanding, techniques and methodologies are opening up horizons for significant improvements in diagnosis and care. However, take-up is uneven, research needs and potential outstrip currently available resources, manifestly beneficial practices—such as population-level screening for lung cancer—are still not generalised, and the quality of life of patients and survivors is only beginning to be given attention it merits. This paper, mainly based on a series of multistakeholder expert workshops organised by the European Alliance for Personalised Medicine (EAPM), looks at some of those specifics in the interest of planning a way forward. Part of this exercise also involves taking account of the specific nature of Europe and its constituent countries, where the complexities of planning a way forward are redoubled by the wide variations in national and regional approaches to cancer, local epidemiology and the wide disparities in health systems. Despite all the differences between cancers and national and regional resources and approaches to cancer care, there is a common objective in pursuing broader and more equal access to the best available care for all European citizens.

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Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

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How Can the EU Beating Cancer Plan Help in Tackling Lung Cancer, Colorectal Cancer, Breast Cancer and Melanoma?

et al. 2022

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“…In the European Union (EU) there are two ways to marketing authorization (MA) of new drugs: a centralized process through the European Medicines Agency (EMA) and a national procedure (managed by national drug agencies) [ 1 – 4 ]. While MA is a precondition, it is not the only causal factor of market access in Europe [ 3 ].…”

Section: Introductionmentioning

confidence: 99%

“…While MA is a precondition, it is not the only causal factor of market access in Europe [ 3 ]. Pricing and reimbursement (P&R) negotiation, which occurs at the national level, represents the second step of the access process [ 4 ]. P&R is complex to manage due to large variability in procedures, timelines, and criteria in each country’s P&R decision-making process [ 3 , 5 ].…”

Section: Introductionmentioning

confidence: 99%

Early access programs for medicines: comparative analysis among France, Italy, Spain, and UK and focus on the Italian case

Tarantola

Otto

Armeni

et al. 2023

J of Pharm Policy and Pract

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Early access programs (EAPs) generally refer to patient access to medicines/indications before marketing authorization, possibly extended to price and reimbursement approval. These programs include compassionate use, which is usually covered by pharmaceutical companies, and EAPs reimbursed by third-party payers. This paper aims at comparing EAPs in four European countries (France, Italy, Spain, UK) and providing empirical evidence on EAPs in Italy. The comparative analysis was conducted through a literature review (including scientific and grey literature), complemented by 30-min semi-structured interviews with local experts. The Italian empirical analysis employed data available on the National Medicines Agency website. Although EAPs are very different across countries, they exhibit some common features: (i) eligibility criteria refer to the absence of valid therapeutic alternatives and a presumed favourable risk–benefit profile; (ii) payers do not allocate a pre-determined budget to these programs; (iii) total spending on EAPs is unknown. The French EAPs seem to be the most structured, financed through social insurance, covering pre-marketing, post-marketing and pre-reimbursement phases and providing for data collection. Italy’s approach to EAPs has been varied, with several programs covered by different payers, including the cohort-based 648 List (for both early access and off-label use), the nominal-based 5% Fund, and Compassionate Use. Most applications to EAPs are from the Antineoplastic and immunomodulating drug class (ATC L). Some 62% of indications in the 648 List are either not under clinical development or have never been approved (pure off-label use). For those subsequently approved, most approved indications coincide with those covered through EAPs. Only the 5% Fund provides data on economic impact (€ 81.2 million in 2021; average cost per patient € 61.5K). Diverse EAPs are a possible source of inequalities in access to medicines across Europe. A harmonization of these programs, though difficult to achieve, could be modelled on the French EAPs and provide key advantages, not least of which a common effort to collect real-world data in parallel with clinical trials and clear separation between EAPs and off-label use programs.

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National early access programs and clinical trials: What opportunities for early access to therapeutic innovations for patients with malignant melanoma?

Abstract: Tailoring clinical trial methods and design according to drug class and gathering systematically long‐term real‐life clinical data from national early access programs might be options to improve the reliability of drug development in metastatic melanoma.

Cited by 2 publications

References 19 publications

How Can the EU Beating Cancer Plan Help in Tackling Lung Cancer, Colorectal Cancer, Breast Cancer and Melanoma?

How Can the EU Beating Cancer Plan Help in Tackling Lung Cancer, Colorectal Cancer, Breast Cancer and Melanoma?

Early access programs for medicines: comparative analysis among France, Italy, Spain, and UK and focus on the Italian case

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