Introduction

Once again, 2021 was a year where the spotlight was focused on the life sciences sector. COVID-19 and vaccination campaigns continuously dominated the news, but focus on the sector spanned beyond the media. The pandemic uncovered various sore points that cautioned EU and national policymakers that more was needed than just the short-term management of the pandemic. Forceful resolutions were made on revisiting certain aspects of the entire life sciences sector and healthcare systems.

On 25 November 2020, the European Commission adopted the Pharma Strategy for Europe. This ambitious policy document aims to ensure that the EU has a solid, fair and competitive life sciences industry that is able to deliver healthcare products to patients, even in times of crisis. In the context of this European pharmaceutical policy, many legislative initiatives are expected from the European Commission.

Belgian policymakers have already stated that they will actively prepare and defend the Belgian position with regard to said upcoming legal framework. This article will focus on some of the policy statements and legislative initiatives that the Belgian government and regulators are about to implement or have already implemented and that echo the same goal expressed in the European Commission’s Pharma Strategy for Europe: secure the continued access of patients to qualitative, effective and affordable treatments.

Accessibility, availability and affordability are fundamental concepts for an effective healthcare system. These concepts have been very sharply challenged by the pandemic. Understandably, they run through these initiatives and statements like a golden thread.

Accessibility, Availability and Affordability à la Belge: What to Expect?

Accessibility

Accessibility is definitely a multifaceted concept. For patients, accessibility means access to safe, effective and innovative medicinal products that cover their medical needs. Governments (together with regulators and payors) want to have access to robust and reliable data to ensure that patient access is given only to medicines that are safe, effective and affordable. The industry, in order to be able to continuously deliver for patients, wants to have access to a well-functioning market (including access to R&D and manufacturing capabilities and capacity and an appropriate return on investment) with a transparent and workable regulatory framework. 

Access to innovative therapies and healthcare

Actions are being taken or were announced in order to accelerate or improve access by patients to innovative therapies.

The procedures of the Federal Agency for Medicines and Health Products (FAMHP) regarding the accelerated access to innovative therapies for unmet medical needs (compassionate use – medical need programmes), are being further optimised; this also includes the intent to find a more aligned approach with the National Institute for Health & Disability Insurance (NIHDI) to improve the process for early temporary reimbursement.

The Belgian general association of the pharmaceutical industry (pharma.be) has issued a set of recommendations to accelerate patients’ access to Advanced Therapeutic Medicinal Products (ATMPs), which include gene therapy, cell therapy and tissue-based therapy. ATMPs are a fast-growing field of innovative therapies. EMA expects the submission or approval of ten to 20 ATMPs per year during the next five years. In 2021, there were 2.600 trials (phase I to III) for ATMPs ongoing in the world, of which 50% in the field of oncology. The recommendations mainly address the regulatory and access challenges of ATMPs and are presented as a basis for dialogue and reform. In particular, pharma.be proposes to improve two procedures to enable faster access to ATMPs (but also other ground-breaking therapies): the "improved unmet medical need procedure" and the "faster reimbursement procedure". These improvements would allow patients to have access to medicines 17 and ten months earlier, respectively.

A framework is in the making for the broader and structural (ie, not only during epidemics and pandemics) integration of telemedicine into the Belgian healthcare system, including possibilities for reimbursement. This primarily concerns:

• teleconsultations between patient and healthcare provider;
• telemonitoring and mobile applications and devices within a new, existing or modified healthcare process; and
• tele-expertise between healthcare providers.

The framework will set requirements for all of the aforementioned domains of telemedicine (technical criteria, quality criteria, etc). Also, the existing procedure for reimbursement of mobile applications is being re-examined. Together with the Belgian Healthcare Knowledge Centre (KCE), a process is being worked out around the application of digital health technology assessments (DHTA).

Access to a well-functioning market with a transparent and workable regulatory framework 

Some important EU regulations came into force, which required and will continue to require implementation into Belgium’s regulatory framework.

On 31 January 2022, the Clinical Trials Regulation (CTR) 536/2014 on medicinal products for human use entered into force. In order to facilitate the transition, the old and the new legislation on clinical trials will coexist during a transition period of three years (until 31 January 2025). Belgium’s policymakers and regulators seem to be confident that, considering the experience with the pilot projects and training sessions that took place, the implementation will go smoothly so that Belgium can continue to profile itself within Europe as an important reference member state for the processing of clinical trial applications. However, government and sponsors will have to learn working with the Clinical Trial Information System (CTIS), which of course will require the necessary training. A specific national IT application, linked to CTIS, is currently under development.

On 26 May 2021, EU Regulation 2017/745 on medical devices entered into force. This Regulation takes account of the increasing innovation in the sector and wants to ensure the availability of products guaranteeing patient safety on the European territory, including by reinforcing pre-market product control and intensifying post-market surveillance. Furthermore, with respect to the traceability of devices, the Regulation provides for the introduction of a unique identification system for devices which will facilitate the recall of defective products and combat counterfeiting.

Belgium currently participates at the meetings on EU level regarding the procedure for the co-ordinated review of applications. EU Regulation 2017/746 on in vitro diagnostic medical devices (IVD) will enter into force on 26 May 2022. The FAMHP is currently preparing legislative work for the further implementation of said Regulation on national level, including in relation to the so-called performance studies. The latter are studies intended to test the performance of an in vitro diagnostic medical device in medical laboratories or in any other suitable environment outside the manufacturer’s own premises. These preparations also concern the necessary IT applications.

Over the past year, pledges were made to strengthen, including through public-private collaboration, Belgium’s position in R&D and manufacturing in the biopharmaceutical sector in the next ten to 20 years. These covenants stem from the awareness, presented by the pandemic, that a robust and reliable supply chain – which includes sufficient available manufacturing capacity, equipment, raw materials and strong human capital – is absolutely key for a well-functioning market that delivers for patients.

As part of the "Health & Biotech Valley of Tomorrow" initiative, the Belgian government signed a charter together with stakeholders from academia and industry, covenanting to strengthen Belgium’s status as a biotech powerhouse. Experts appointed by the Belgian government identified five specific areas in which Belgium should invest in order to reach that goal:

• better use of health data and connecting data sources;
• investing in human capital;
• strengthen regional and cross-border collaboration;
• deploy an end-to-end approach across the biopharma value chain; and
• investing in innovative platforms such as cell therapy and digital therapeutics.

On the basis of these expert conclusions, working groups are currently discussing the contours of a new strategic plan for the future of Belgium, which plan should be final by the end of April 2022.

As part of the aforementioned strategic plan, the construction of a state-of-the art training centre with a business accelerator (the EU Biotech School and Health Hub) is planned in Gosselies, which should be operational by 2025. This flagship project has four pillars:

• STEM promotion – stimulate young people to work in the biotechnology and biopharmaceutical sector;
• next-generation biomanufacturing – training adapted to the latest therapeutic developments and the increasing digitalisation and automation of production processes;
• digitalisation – training to accelerate digital transformation; and
• soft skills and mini MBA – helping start-ups acquire business knowledge and specific management training for life sciences.

A new European production centre for cell therapy will be constructed at the Tech Lane Ghent Science Park. For this purpose, Janssen Pharmaceutica will collaborate with Legend Biotech, a clinical-stage biopharma firm specialised in the development of cell therapies for oncology and other indications. The new site will be Janssen Pharmaceutica’s first cell therapy production centre in Europe.

A new pact on the future of the pharmaceutical industry in Belgium will soon be released. This new pact, which certainly will take into account the lessons learned from the pandemic, will incorporate – as far as possible – the priorities of all stakeholders (patients, health care providers, payors, government and industry). The idea of the pact is to align and co-ordinate objectives and commitments of all partners. 

Increasing evidence-based policymaking

According to the WHO, policy and practice which are based on systemic evidence are seen to produce better outcomes. Over the years, Belgian government has actively shifted away from an opinion-based policy towards a more evidence-based policy (EBP). An EBP gathers and uses high quality research evidence to inform policymaking, including for defining healthcare priorities and taking decisions on marketing authorisations and reimbursement.

Belgium has already showcased its EBP approach with a number of acclaimed initiatives, including:

• the KCE Clinical Trial programme (a programme of publicly funded, non-commercial, comparative, practice-oriented clinical trials that address questions that are generally not studied by the industry, despite their high societal impact);
• the BENEFIT collaboration (collaboration between KCE and The Netherlands Organisation for Health Research and Development (ZonMw) for funding international non-commercial clinical trials); and
• the BENELUXA initiative (the international co-operation initiative between the respective reimbursement authorities of Belgium, The Netherlands and Luxembourg for the sustainable accessibility to innovative medicines with actions on horizon scanning, health technology assessment (HTA), information sharing and joint price negotiations).

Belgium’s government has expressed its intent to intensify these types of initiatives and collaborations. 

In the Ministry of Health’s policy statement 2021–22, a number of additional priorities are outlined that clearly demonstrate Belgium’s policymakers’ intent to rigorously strengthen the path of EBP as a basis for their decision-making.

The FAMHP will support drug repurposing activities, ie, the search for new applications for existing medicines, through the participation by the FAMHP in the European Commission’s Expert Group on Safe and Timely Access to Medicines for Patients (STAMP) and, as a follow-up on STAMP, in the pilot project launched by the European Medicines Agency (EMA); the latter pilot project aims to support not-for-profit organisations and academia to gather or generate sufficient evidence on the use of an established medicine in a new indication with the view to have this new use formally authorised by a regulatory authority. As part of the pilot, the EMA and FAMHP (including other national medicines agencies) will provide regulatory support and scientific advice to help generating robust data for use in a future application by a pharmaceutical company. 

Not only the industry has discovered the need – and great potential of – real world evidence (RWE) in the healthcare decision-making process. RWE is evidence about the usage and potential benefits or risks of a health product or technology, derived from the analysis of real world data (RWD). Also, Belgian policymakers have realised the need of a bottom-down sound strategy for RWE. In collaboration with several other (public and private) stakeholders, the Belgian government has set up the "#dataforbetterhealth" initiative. This initiative aims to identify existing barriers to a policymaking based on findable, accessible, inter-operable and re-useable (FAIR) data, leading to improved EBP. The government, in the framework of this initiative and fitting in with the European initiative "European Health Data Space", also intends to incorporate a Belgian Health Data Authority (BE HDA). The BE HAD will centralise databases as a single point of contact for healthcare data in a GDPR-compliant manner.   

Availability

Developing innovative medicines or therapies and successfully routing these medicines and therapies to the market is one thing. Making sure these are also affordable and uninterruptedly available is another.

Many concerns have already been expressed about medicines being unavailable. The pandemic has brought this problem into even sharper focus. Shortage has now also become a pressing problem upstream: the enormous capacity that is required for the vaccine development and production, may lead to a shortage in available manufacturing capacity for other (lifesaving) medicines on a worldwide-wide basis, including a lack of available tooling, equipment and raw materials. This may cause a run on available capacity and pharmaceutical companies having to take considerable risks in reserving capacity many years ahead of the actual manufacturing date. Evidently, such race may have a substantial ripple effect on available medicines on the market. Innovation may come with a delay and pricing may be impacted.  The current geopolitical challenges may throw even more oil on the fire. 

There are a number of legislative initiatives that were taken in the field of shortage of medicines. These legislative initiatives include:

• the Law of 13 June 2021 containing measures to control the COVID-19 pandemic and other urgent measures in the field of healthcare (providing certain minimum stock and stock disclosure requirements);
• the Ministerial Decree on various urgent measures concerning specific medicines, to combat shortages of medicines in the context of the SARS-CoV-2 pandemic of 30 August 2021 (including mandatory quotas for marketing authorisation holders and wholesalers of certain medicinal products); and
• the Royal Decree of 26 November 2021 implementing Article 6, paragraph 1sexies of the Law of 25 March 1964 governing medicines with regard to the transparency of the stock of medicines dedicated for public service obligations (introducing information obligations, including a stock monitoring system, for wholesaler-distributors).

However, one could definitely argue whether these legislative initiatives will be sufficient to successfully address the above-described issue that lurks around the corner. In the Ministry of Health’s policy statement 2021–22, it is rightfully stated that Belgium, in collaboration with all stakeholders and other governments, should urgently develop a strategic safety stock. Also, the intention is mentioned in said statement to revisit the massive destruction of medicines that reach the end of their shelf life. Furthermore, what some expect to see in the near future, as part of the mitigation efforts for unavailability risks, is the development (eventually on an EU level) of a collective purchase policy.

Affordability

A well-performing healthcare system is also able to bring innovation at an affordable price. Since many years now healthcare budgets are under pressure. The growing – but very expensive – field of personalised medicines, including, ATMPs, will further fuel the discussion on affordability and willingness to pay. 

Considering the challenges ahead, as described above, it is probably not the appropriate time to heavily hit the brakes and not to invest in innovative medicines that prove to be safe and effective. It will all the more important to make the right decision, making sure money is spent efficiently and effectively. RWE will play a crucial role in this.

It is expected that Belgian policymakers will boost its EBP approach, including through the increased use of nationally available HTA expertise, international co-operation (including with EU and WHO) and collaboration with all stakeholders. This will probably be an important axe in the new pact on the future of the pharmaceutical industry that will soon be released. 

Furthermore, Belgian government has commissioned relevant bodies to re-evaluate the current regulatory framework on pricing of medicines. The Ministry of Health’s policy statement 2021–22 also mentions the intent to re-evaluate the decision-making processes (and the role and responsibility of all actors involved in these processes) around the reimbursement of medicines. This re-evaluation should lead to a redesign of the regulatory framework, reportedly also including a reform of the Commission for the Reimbursement of Medicines (CTG) and the adjustment of the so-called "managed entry agreements".

Conclusion

Clearly, the life sciences industry and healthcare system are under pressure. This has put both the EU and national policymakers in hyperactivity mode. An avalanche of new regulations is about to see the light to mitigate the challenges ahead. These legislative initiatives all stem from the idea that we should have a more resilient life sciences industry and healthcare system, capable of coping with crisis and securing at all times the continued access of patients to qualitative, effective and affordable treatments.

On the basis of EU’s pharma strategy for Europe, a more resilient EU pharma sector seems to be understood as a sector that is less dependent on other regions in the world. The current geopolitical challenges will further drive that process of rethinking certain aspects of the life sciences industry and the healthcare system.

There surely is never a dull moment in the pharmaceutical industry.