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Associação Portuguesa da Indústria Farmacêutica

APIFARMA

O objectivo primordial da APIFARMA é, através da prestação de serviços às empresas associadas, defender os interesses comuns dos seus associados, abrangendo todos os campos multidisciplinares relevantes para o sector, nomeadamente: - Defesa de elevados padrões de qualidade, segurança e eficácia dos medicamentos produzidos. - Defesa de um sistema de aprovação dos medicamentos célere e eficiente. - Protecção dos direitos de propriedade industrial. - Proximidade com o Doente, primeiro destinatário da sua actividade. - Interacção com a comunidade científica, quer pela permuta do conhecimento, quer pela preservação do património científico. - Promoção de uma cultura de defesa ambiental, como salvaguarda da Saúde Pública. - Proximidade com a Comunidade, para divulgação da sua missão e valores. - Respeito integral pela legislação que lhe é aplicável, não admitindo qualquer forma de trabalho forçado ou compulsório nem de trabalho infantil no desenvolvimento das suas actividades.

Lobbying Activity

Response to Health technology assessment – Joint scientific consultations on medicinal products for human use

29 Oct 2024

The EU HTA Regulation marks a significant change in assessing clinical evidence for new technologies across the EU, with its impact expected within four months. The Joint Scientific Consultation (JSC) was created to continue the successful HTA advisory framework from EUnetHTA Joint Actions, focusing on: 1. A synchronized calendar with EMA, ensuring timely advice aligned with the development program, 2. Consolidated PICO advice, supporting a single European HTA voice that promotes EU-based trials, and 3. Prompt outcome letters that enable simultaneous HTA and global regulatory discussions. APIFARMA has serious concerns with the draft JSC implementing act, particularly around infrequent, limited open calls. This approach lacks visibility and predictability, complicating planning for health technology developers (HTDs) who need regular, accessible advice slots. The only option to make JSC interesting for developers is a monthly call with predictable steps for the submission of the briefing package, for the meeting schedule, and for the outcome document release. It would be unreasonable to expect that global development plans would adjust or paused their schedule to either wait for an open call or postpone the project waiting for the scheduling of the discussion meeting and the approval of the outcome letter. The JSC system introduces a new framework that is not aligned with the current advice ecosystem, which will by default be at a disadvantage compared to the well-established and predictable national advice systems. In particular, the unpredictability of the JSC system is in stark contrast to how national advice is set up in member states such France and Germany, where the calendar of the advice slots is made transparently and readily available, allowing for appropriately planning by both the HTDs and the authorities. The low capacity at EU-level will also promote further national advice, which will give advantage for those countries offering them for getting their PICO needs addressed. This may come at a cost for the rest of the member states and limit the opportunity for them to provide a meaningful early consultation. Early engagement and PICO anticipation are critical for comprehensive JCA dossier planning due to too short time for dossier preparation. APIFARMA believes the JSC system can succeed if the draft implementing act recognizes key stakeholder roles, and the HTA Secretariat and Coordination Group consider these recommendations: - Request periods should closely approach a rolling system with adequate capacity, minimizing selection criteria; - JSC and JCA procedures should be continuous, with JSC advice shaping evidence for JCA submissions; - Clarification is needed on JSC eligibility for all assets, regardless of development phase, applying prioritization criteria only if maximum capacity is reached; - Further guidance on JSC and parallel JSC/EMA procedures is essential, including synchronized timing, streamlined information exchange, and coordinated submission processes when conducted with EMA; - The JSC should support HTA bodies in harmonizing recommendations, involving all Coordination Group Member States in outcome endorsement; - The outcome document must consolidate advice, explaining any national differences transparently, and focusing on actionable insights for HTDs; - Ensuring IT platform security, confidentiality, and strict data-sharing principles will build trust and protect sensitive information. - For JSC success, APIFARMA urges that outcomes be actionable, providing focused, timely recommendations for JCA submissions, with adequate resources to meet demand within the EU HTA framework, including options for follow-up advice. In conclusion, APIFARMA sees the EU HTA Regulation as an opportunity to enhance patient access and reduce inefficiencies in national HTA processes, with JSC as a critical phase for successful implementation.
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Response to Health technology assessment – Procedural rules for the assessment and management of conflicts of interest in joint wo

25 Jun 2024

The EU HTA Regulation represents a step-change in the assessment of clinical evidence of new technologies at EU level and has the potential to accelerate and improve access to patients as well as reduce inefficiencies and duplications, goals that the pharmaceutical industry shares. This Regulation 2021/2282 on health technology assessment (HTA) stipulates that the joint work should aim to achieve the highest level of quality, transparency and independence. To preserve the scientific integrity of the joint clinical assessments and joint scientific consultations, rules should be developed to ensure the independence and impartiality of patients, clinical experts and other relevant experts involved, and avoid conflicts of interest. APIFARMA supports a policy that aims for a high level of quality, transparency and independence in joint work. However, the European Commission's proposal to guarantee independence is not balanced in terms of ensuring quality. APIFARMA is concerned that the proposed strict rules for dealing with conflicts of interest will exclude the right people with relevant, in-depth expertise and will ultimately reduce the scientific quality of the joint work. APIFARMA recommends a pragmatic approach to involving the best available expertise, including individuals representing organizations, with transparent documentation of the declared interests. It is crucial that the new EU HTA system find specific forms of engagement of external experts in situations of potential conflict, considering their magnitude, extent, and potential undue advantages, considering the full picture.The involvement of patients and experts should also include individuals representing organizations. The participation should not be restricted based on conflicts of interest. APIFARMA believes that EU HTA Regulation is an opportunity to accelerate and improve access to patients as well as reduce inefficiencies in national HTA process. This joint work should aim for high quality, transparency and independence and involve more impartial experts, like patients, clinical experts, and others. Please find attached the APIFARMA's contribution.
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Response to Health technology assessment - Joint clinical assessments of medicinal products

27 Mar 2024

APIFARMA's contribution to JCA implementing act public consultation The EU HTA Regulation represents a step-change in the assessment of clinical evidence of new technologies at the EU level and has the potential to accelerate and improve access to patients as well as reduce inefficiencies and duplications, goals that the pharmaceutical industry shares. The EU HTA Regulation is a reality and will start to affect HTA bodies, health technology developers and their technologies as well as patients and clinicians in less than 10 months. The success of the EU HTA Regulation is contingent on the ability of the system to deliver high-quality JCA reports which provide added-value to national decision-makers. As such, the JCA process needs to: i. facilitate early engagement with the health technology developer (HTD), to ensure a timely and optimally informed start of the process and to enable the systematic exchange of relevant information with the HTD. ii. to provide all participants with the necessary information and evidence for the scoping and assessment. iii. guarantee sufficient time for HTDs to conduct the relevant analyses required for preparing and finalizing their clinical evidence submission dossier. iv. allow assessors sufficient time to review the JCA dossier and to develop a high-quality JCA report. APIFARMA is concerned about several glaring shortcomings in this draft JCA implementing act, especially regarding the workability of the JCA process, where it undermines the capacity of the HTD (from SMEs and biotech to larger pharmaceutical companies) to provide a good quality submission to the assessors and co-assessors. Several reasons drive this concern: the unbalanced time allocation between scoping and submission stages, the absence of visibility on the submissions scope coupled with the expected size/complexity of the assessment scope driven by a mechanistic need to satisfy all needs of all Member States and the lack of meaningful participation of the HTDs at key steps of the process. APIFARMA strongly believes that the draft JCA implementing act can set the path towards a successful EU JCA system if it recognizes the role of key stakeholders in the process and enables a rebalancing of workload in line with respective remits and obligations. In practice, this means that: The assessment scope should be finalized within 90 days of the start of the JCA procedure and the HTD should have at least 135 days to prepare a high-quality submission dossier. On the assessment scope the consolidation of Member States feedback needs to be faster and maintain enough time to Member States to do a correct scope preparation and feedback. The HTD should have the opportunity to leverage all relevant information, evidence, and knowledge as a key input into the definition of the assessment scope. This information could be useful for Member States to prepare in a more efficient way their feedback. The HTD should have an opportunity to meet and discuss with the assessor/co-assessor its views on the draft assessment scope. The HTD should have visibility on the draft assessment scope proposed by the assessor/co-assessor as well as on the PICO survey responses from Member States (in an anonymized manner) at the earliest possible stage, to be able appropriate time to submit a high-quality submission response. The assessment report needs to be friendly to Member States to extract useful information for their decisions. This is very important to avoid national delays in the therapeutic value definition. There is a need to regulate some points of this process out of the implementing act, namely criteria to re-initiate this process based on different therapeutic indications, criteria to update JCA and other technical points. In conclusion, APIFARMA believes that EU HTA Regulation is an opportunity to accelerate and improve access to patients as well as reduce inefficiencies in the national HTA process.
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Response to Evaluation and revision of the general pharmaceutical legislation

8 Nov 2023

Commissions proposal is the start of the discussion for a balanced package. It is an opportunity to enhance Europe's attractiveness for research, development, and production of medicines. APIFARMA would like to contribute to this public consultation based on some of its specific points of view. The market exclusivity for new drugs strongly penalizes innovation if a medication is not available in all Member States within 2 years, which we see as a near-impossible target for companies. It would discourage companies from investing in Europe and make it more difficult for SME companies to get money to invest. Additionally, EU should not be overly dependent on medicine producers or suppliers of raw materials elsewhere in the world. The bolar exemption should be limited to marketing approval, preserving the effective enforcement of IP rights for pharmaceuticals and the reliability of the IP regime in the EU. It is also important that the revised legislation does not negatively affect innovation, particularly when it comes to orphan drugs. This is why incentives must be included to promote research and development and reduce unnecessary bureaucracy. The reduction and modulation of incentives could disincentivize investment in R&D and discourage risk-taking for innovation. Regarding UMN and HUMN definitions, they are too narrow and discard the value of iterative innovation. Continuous innovation is essential, as it allows for improved modes of action, which potentially improves patients daily life and addresses possible limitations associated with the current standard of care. It is also important to understand which products may fall within the criteria and how patients will be involved in those decisions. The stringent conditions associated with the TEV proposed significantly undermine the effectiveness of the incentive. It is important to focus on their clinical benefit and effectiveness in combating resistance rather than on predetermined criteria for TEV eligibility. It is also important to conduct regular reviews ensuring that the program addresses evolving challenges in AMR. APIFARMA is apprehensive about the inclusion of antimicrobials as prescription criteria, as we believe that should be applicable to antibiotics to which AMR has been proven. APIFARMA welcomes the ECs proposals to future-proof and upgrade the EU regulatory system, making it more efficient and competitive. The establishment of regulatory sandboxes would provide a good opportunity to test innovative and potentially promising products for patients in a controlled environment. APIFARMA fully supports the provision of an electronic format and recommends the implementation phase be as short as possible to increase access to information for patients and to support the mitigation of supply challenges (eg: multi-country packs). A focus on supply must guarantee greater competitiveness while responding to shortages. In this sense, robust testing and strong environmental standards must be ensured in production. It must be ensured that important medicines are available to people and countries whenever needed. The proposal of shortage prevention plans should be practical and consider unexpected high demand, as happened during the pandemic. We have concerns about a definition of critical medicines being potentially disproportionate. We are also concerned whether pharmaceutical companies should be required to maintain safety stocks to help mitigate the impact of supply chain disruptions. Environmental measures are globally appreciated, but there is concern that the new ERA provisions may be disproportionate and cause additional administrative burden for companies. On the other hand, with the potential refusal of the marketing authorisation if the ERA is incomplete, or if the environmental risk has not been adequately addressed by the applicant, this could result in patients not having access to the medication they need.
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Response to Vaccine-Preventable Cancers

3 Feb 2023

The goal of preventive health is to prevent or identify diseases at an early stage. Despite improvements in survival, cancer is the no. 1 cause of disease burden (DALYs) in Portugal (Wilking, N. 2020). In Portugal, the human papillomavirus (HPV) is potentially associated with approximately 1466 cases of cancer, annually, in 8 main locations: the cervix, the oropharynx and hypopharynx, the vulva, the anus, the penis and the vagina. Since 2008, the National Immunization Program (NIP) includes the vaccine against HPV infections for female children and, since 2020, male children have also been covered by this vaccine. Portugal presents a population coverage for vaccination against the human papillomavirus virus in female children of 91%, since 2012. Currently, the vaccine is available against 9 serotypes of HPV and offers greater protection than previously used vaccines (4 serotypes), with an effectiveness of 90% in preventing HPV infections, which cause carcinogenic lesions in the cervix. Since 1995, the NIP has included the vaccine against the hepatitis B virus, reaching 98% coverage in successive cohorts of children. It is estimated that 30,000 Portuguese are infected with the hepatitis C virus and 40,000 with the hepatitis B virus, according to data from the national serological survey (INSA 2016), most of whom are asymptomatic (Data by DG of Health, 2022). In conclusion, access to vaccination is important. APIFARMA is completely aligned with the strategies proposed by Vaccines Europe, encouraging the European Commission and the Member States to: 1) Leverage forums and platforms to share best practices and monitor progress: Promote the use of vaccination as an effective and safe strategy to prevent and control cancers with policy makers, and the general public; Ensure that the deliverables of the EU4Health projects aimed at improving vaccination through evidence on vaccination programmes, implementation and monitoring, data systems, and communication are shared widely; Implement a European Council Recommendation on vaccine-preventable cancers, with clear KPIs for Member States; Call on the ECDC to further develop the monitoring of current HPV and HBV vaccination at EU level; Use the new expert group on public health from the European Commission to monitor progress made in vaccination rates and implementation of the EBCP; Expand sites for vaccination to increase uptake, especially for adults, such as community pharmacies. Monitor the implementation of health components of national Rescue and Resilience Plans; Improve access to vaccination against Hepatitis B to achieve WHO goals of a 90% reduction in cases by 2030; 2) Ensure robust program performance by investing in digitalization and data systems: Vaccinate at least 90% of the target population with HPV vaccines by 2030 and, expand recommendations and objectives beyond cervical cancer, to target other HPV-related cancers and diseases in both genders with a comprehensive HPV prevention approach; Screen at least 95% of the eligible population by 2025, and treat at least 90% of HPV related cases and precancerous lesions; Strengthen electronic monitoring and registry systems for vaccines preventable cancers coverage rates; Develop the European Cancer Information system and improve health literacy for cancer prevention and care; 3) Encourage sustainable immunization financing at MS level: Invest in health service and implementation research for strategies to reach under-served and at-risk (adult) population groups and increase and maintain confidence in vaccines; Develop action plans for hepatitis prevention and control with sufficient funding in Europe; Raise awareness of the various EU tools for investment in sustainable cancer-related financing.
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Response to Evaluation and revision of the general pharmaceutical legislation

26 Apr 2021

For APIFARMA, equity should be the baseline in the future healthcare policy. Europe has the opportunity to foster investment in research and innovation paving the way for a new generation of highly innovative treatments and of highest added value. Only by developing treatments for the unmet needs, reducing delays, and ensuring equal access to therapies can we truly leave no citizen suffering a disease behind. The regulatory and Intellectual Property (IP) revisions may transform the balance between incentives and rewards for pharmaceutical companies, particularly concerning Supplementary Protection Certificates and patent term extension, both fundamentally important for the pharmaceutical industry. The revision of the primary pharmaceutical legislation, coupled with the Pharmaceutical Strategy’s increased emphasis on access and affordability of medicines, supply chain resilience, market authorisations and competition principles, could have long range effects on the pharmaceutical industry and the healthcare industry in general. APIFARMA aligned with EU Pharmaceutical Industry Federation is calling for: • Reinforce expertise-driven assessment and enable a more agile centralised authorisation framework by removing unnecessary interfaces between EC, EMA &Committees • Enhance expedited pathways framework supporting innovation • Expand the role of EMA in the assessment of drug-device/diagnostic combination products • Replace the paper patient information leaflets with electronic versions • For the future foreseen innovative approaches to medicines R&D eg by ensuring the acceptance of RWE in medicines evaluation both pre and post-authorisation. • Simplify and modernise the EU post-approval framework – e.g revision of Variations Regulation. Fast-paced developments in medicine, devices, data and digitalisation will lead to more personalised healthcare solutions. The legislation framework must set out the principles, whilst allowing for policy adaptations at a later point without the need for further legislative revision. Updates to existing policy and guidance documents can help to: • Encourage the use of novel clinical trial designs • Ensure ongoing scientific dialogue throughout product development • Enhance security, transparency and oversight of supply chains through a targeted stakeholder approach and incentives driven reforms based on facts. Industry calls for using the data stored in the interoperable network of national repositories (Falsified Medicines Directive) to provide additional intelligence in monitoring shortages • Support environmental risk assessments to cover product lifecycle and address sustainability via initiatives like #Medsdisposal. Competitiveness drives innovation and innovation saves lives. Europe has a role to play by encouraging and protecting innovation, driving research and development investments into areas of unmet medical need. IP underpins the ability to invest in the long, complex, risky and costly process of delivering new medicines to patients, given the central and increasingly important role of IP in Europe’s economy as well as its ability to enhancing the EU’s resilience to global health threats and address ongoing health challenges. Since market access is dependent on mostly national and health system related factors as well as market dynamics we expect to see a stronger commitment from national governments to identify and remove outstanding barriers and to address the negative effects of international reference pricing allowing trade will continue to evolve in a more innovation-driven way. Finally, the Industry is committed and call for a High-Level Forum on Access to Health Innovation enabling a comprehensive discussion and analysis of the root causes of unequal patient access and supply of medicines, also taking into consideration the fragmentation of the EU single market and the different national approaches to pricing and reimbursement as well as healthcare investment and organisation.
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