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European Alliance For Vision Research and Ophthalmology

EU-EYE

The main goals are to raise political and societal awareness for the increasing need for Vision Research and Ophthalmology at European level.

Lobbying Activity

Response to Evaluation and revision of the general pharmaceutical legislation

8 Nov 2023

Access to affordable treatments for rare diseases remain a challenge in eye diseases.We applaud the European Commission's support to the retention & strengthening of Hospital Exemption (HE) as it contributes in reducing the current misalignment between public health priorities & industry outputs particularly in diseases with no commercial interest to industry.However its uneven implementation contributes to fragmented EU health research system & creates health inequities (health outcomes & access to therapies).We ask that the European Commission pays particular attention to non routine and hospital,the two terms which we believe contribute to this uneveness: e.g.: France refers to ATMPs as Médicaments de thérapie innovante préparés ponctuellement (MTI-PPs) - ad hoc preparations instead of non-routine; differentiating between authorisations to prepare and use with two categories: MTI-PP á usage humain based on national authorisation & MTI-PP à usage humain utilisé dans le cadre de recherche biomédicale for manufacturing the same HE products within the scope of a clinical trial. The current definition of ATMPs under HE needs to be more inclusive/flexible given national specificities or organisational developments e.g.emerging manufacturing units to assist GMP compliance of product. We propose: ..prepared on a non-routine basis according to specific quality standards in hospital where the product will be also delivered or in other specific authorised centres, and used within the same Member State etc. A registry and data collection/monitoring are welcome but we call for all ATMPs (commercial and under HE) be included for benchmarking of therapies. A monitoring list for medical devices & a platform for healthcare professionals/citizens to report supply issues are much needed. We are cautious about the introduction of biosimilars with no additional controls as the text of (27) p 24 on experience with approved biosimilars does not reflect the mixed experience of ophthalmologye.g. in uveitis. https://pubmed.ncbi.nlm.nih.gov/32795432/; https://pubmed.ncbi.nlm.nih.gov/33121931/ Overall we do not oppose biosimilars, but rules are needed with the freedom to prescribe original drug in cases of difficult disease management and with already reduced vision.Current pharmacovigilance practices may not be effective when prescribed drugs are changed due to insurance changes at patient level: patients change biosimilars depending on which drug is covered by the insurance company as different insurance companies have contracts with different manufacturers. (50) p28:Aligning the concept of would require that the definition of unmet medical needs (UMN) should include availability/adequacy of available treatments, disease severity/burden & patient population size.Quantification however of UMN depends on the scope and the value framework in which it is used based on different stakeholder preferences and responsibilities.Reflection will be needed regarding what elements are important for consideration in each case, and how one would measure UMN within the broader regulatory or value framework applicable. article 44 1(g):Post-authorisation obligation to substantiate clinical benefit when surrogate endpoints (SEs) are used for MA,should be made mandatory with clear official ramifications for products which fail to meet their obligations.Market authorizations should be revoked for those failing to improve quality of life or overall survival.Relying on a self-decommissioning process whereby ineffective therapies are quickly identified & rejected at market level,is not sufficient to ensure patient safety & trust in regulatory process.Additional obligation should be the publicly accessible information by patients/clinicians on the use of SEs for approval of therapies & validation status to reduce misperceptions about benefit & ensure informed decisions consistent with patient goals and values.
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Response to Evaluation and revision of the general pharmaceutical legislation

26 Apr 2021

1. The roadmap needs to consider, or at least explore with stakeholders, the benefits, particularly for small organisations, of a standardised mandatory model contractual agreement forconduct of clinical studies between Sponsor and site/healthcare facility conducting the study. Potential benefits are: - attracting more hospitals in using their own clinical networks & enabling hospitals to become themselves Sponsors in clinical studies particularly in repurposing of medicines where commercial interest is low; - speeding up the entry of new medicines in market; - considerable savings for biotech companies in negotiation time & costs ; - attract biotech investment in EU. The current structure is rigid and it hinders cross border access to clinical trials (see point 3). Negotiating contracts in each Member State involves considerable costs and time as each Member State has its own contractual template for the conduct of clinical studies e.g. Contrat Unique (France); AIFA (Italy); pharma.be (Belgium); National Contract Template (Greece) regional contract templates (Spain); Nefarma (The Netherlands); KAV-tempate (Austria) etc. Such variations are counterproductive given that all CTs have to follow the same EU regulations - Clinical Trial Regulation, GCP and GDPR. Having also different templates and local requirements deter many organisations particularly the smaller ones in sponsoring studies. The EU EYE believes that it should be an interesting and cost-saving initiative to have a standardised mandatory model on contractual agreement for the conduct of clinical studies between the biotech/pharmaceutical company (Sponsor) and the healthcare facility (site) conducting the study. 2. Automated and computerised inventory management systems are needed to manage medicine supply at healthcare provider (HCP) level as they facilitate direct visibility of inventory, more accurate forecasting, and reduce costs throughout the healthcare system. HCPs require support in having the right technology in place to manage drug shortages. Investment in regional/national/European telematic infrastructures to support computerised/IT inventory management systems at least at acute care level will improve visibility of needs and better manage supplies. The roadmap could address how technology advances can facilitate the start up of such inventory system and how barriers can be overcome such as natural political limitations between countries. Furthermore a new management system will also provide insights as to which medicine shortage is a general European “problem” and which one is a national issue as this will prescribe different set of actions and solutions. It will not be sufficient in the long term to only have a platform that coordinates information from existing national notification systems such as the one run by the Danish Medicines Agency on current and expected interruptions in medicine supply: https://laegemiddelstyrelsen.dk/en/licensing/supervision-and-inspection/medicine-shortages/notifications-about-the-supply-of-medicines/ The notifications are not updated regularly and one has to get in contact with the supplier directly. Also not all countries have access to such notification system. 3. The roadmap should also consider to engage with the debate on cross-border access to clinical trials (CTs) by investigating the needs, challenges and potential frameworks that would improve access to CTs particularly for rare diseases or for therapies which are not available to patients in their country of residence. This will address the inequities in availability of clinical trial sites across the EU as sponsors choose specific countries due to availability of experienced sites, time to trial approval & potential reimbursement of developed product. Policy actions could bring CTs closer to patients by facilitating opening sites in more countries, or by adapting legislation for remote/decentralized CTs (ref attached).
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Response to Pharmaceutical Strategy - Timely patient access to affordable medicines

7 Jul 2020

The EU EYE welcomes the initiative Pharmaceutical Strategy with its multiple goals on equity in access to safe medicines; incentivise innovation; environmental impact of medicines and strategic autonomy. The Strategy can achieve a shift towards health outcomes-driven healthcare systems and support national health systems of small and lower income countries to address existing information asymmetry and inequalities in negotiating power.The Strategy needs to be complemented by: - a broader dialogue across the entire healthcare ecosystem to align innovation efforts to public health and health systems’ needs; - an incentive framework to stimulate engagement of all stakeholders of the healthcare ecosystem on equal footing and addresses clinical needs, access, innovation and environment with financial and non-financial commitments of all Member States; - coordination along the policy continuum to address current conflicting interests among interlinked policy frameworks and - collaboration at global level. The EU EYE believes that strengthening the interaction with patients and healthcare professionals as the end users in the pharmaceutical supply chain should focus on co-creation in the following areas: - the use of common endpoints; - when defining ‘added therapeutic value of medicines’; - formulation and priority setting in research strategies (private and public, clinical trials, new frameworks, etc). - continuous monitoring of policy impact to keep pace with changes e.g. new modes of healthcare delivery, digitalisation, etc. The EU EYE believes that there is a greater role for the Pharmaceutical Strategy. The recent pandemic has given rise to the belief that “crisis situations demand exceptions to high standards for quality” in research. The European Pharmaceutical Strategy should aspire to become the guardian of the moral mission of research at times of urgency as those created by the pandemic. In the words of London and Kimmelman, the mission is: “to reduce uncertainty and enable caregivers, health systems, and policy-makers to better address individual and public health. Rather than generating permission to carry out low-quality investigations, the urgency and scarcity of pandemics heighten the responsibility of key actors in the research enterprise to coordinate their activities to uphold the standards necessary to advance this mission.” The Pharmaceutical Strategy can be pivotal in spreading worldwide the EU value and perspectives to the right to health provided the strategy is complemented by: - a continuum of actions aiming to strengthen regulatory sciences, support regulatory scientific advice and constantly evaluate and monitor policy implementation and its impact (existing incentive schemes, orphan medicines, generics/biosimilars, off-label use, etc); - a wider reform that creates consistency and complementarity by addressing current conflicting interests among interlinked policy frameworks - trade policy, IP protection, free trade and public health; - broader transparent dialogue that focus R&D investment on societal benefits by delinking it as much as possible from financial motives. The recent pandemic demonstrated a need for rapid knowledge sharing when redirecting medicines to a new use. Despite EMA's warnings on lack of evidence regarding benefits, attempts to redirect hydroxychloroquine and chloroquine as antiviral agents against COVID-19 proceeded in high numbers and uncoordinated. An on-demand repurposing framework, as a hybrid between the piloted repurposing framework & the ERN model, can optimise rapid sharing on outcomes and clinical experience; behosted within the ordinary repurposing framework and be defined by: - the capability to be activated at times of emerging threats such as new infectious diseases and - by having its own coordinating body to overlook such efforts, preferably drawn from within the current regulatory community such as EMA's pandemic Task.
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Meeting with Annika Nowak (Cabinet of Commissioner Vytenis Andriukaitis)

21 Sept 2015 · EU research funding, on reducing the impact of eye disease