Health Technology Assessment (HTA) is a complex process that measures the added value of new medical technology compared to existing technologies. The aim of this assessment is to ensure that patients have access to the best treatment available on the market while analyzing the costs to the patient and the impact on the organization of health systems in administering the treatment. HTA can be a very important tool for Romanian patients in terms of access to new medicines, as Romania is among the EU countries that offer the fewest innovative medicines free of charge to patients.

Medical technologies are, for example, medicines, medical equipment, and methods of diagnosis, treatment, rehabilitation, and prevention.

The authorities involved in HTA assess both the therapeutic effect of the drug, potential side effects, the extent to which it affects the quality of life and how it is administered compared to existing therapeutic alternatives, and the costs of including a new drug in reimbursement. It is therefore a multidisciplinary process that synthesizes medical, economic, organizational, social, and ethical information related to the use of medical technology in a systematic way.

The main purpose of HTA is to provide evidence-based information to policy makers so that they can formulate safe, effective, patient-centered health policies that deliver maximum results at minimum cost. The HTA is also used by national authorities to decide which technologies should be reimbursed nationally.

The EU wants to strengthen EU cooperation on health technology assessment (HTA). Thus, the European Commission launched a new initiative in 2018, which has reached the final stages of the adoption process after the compromise reached during the German Presidency of the EU Council. The final vote in the EU Council is expected in November, in the European Parliament in December, and publication in the Official Journal in January 2022.

There are 3 main areas:

• Joint Clinical Assessment – clinical evaluation of medicines.
• Joint Scientific Consultation – structured dialogue with drug manufacturers to adapt the design of clinical trials to have the best possible evaluation criteria.
• Horizon scanning – prospective research to identify drugs that will enter the market in the next 2-3 years.

The actual implementation will take place in 3 phases:

• Winter 2024- early 2025: oncology drugs and ATMPs (advanced therapies, cellular, gene, etc.).
• Winter 2027- early 2028: orphan drugs.
• Winter 2029- early 2030: all medicines.

The most important structure will also be the Coordination Group, in which Member States must nominate representatives with competence in the field of health technology assessment.

Access to medicines in Romania vs. Europe

The objective of rapid, equitable and sustainable access to treatment must be shared by all key actors in the Romanian healthcare system and recognise the delays in access for patients in Romania. Romania’s gap can only be closed if our country will be directly and actively involved at European level in the decision making process on the new regulations for Health Technology Assessment (HTA).

Romania among EU countries providing free access to the fewest innovative medicines for patients

Data from the “W.A.I.T. Study” 2020 (published in 2021) – how long patients wait to access innovative therapies” shows that in Europe patients can wait between 4 months and 2.5 years to access the same new medicines depending on the country, with Romania coming last in this ranking.

Countries in north-western Europe have much faster access to the latest drugs than their neighbours in southern and eastern Europe, with patients in some countries waiting more than seven years or more, according to the new research. Access is fastest in Germany, with an average of 120 days between marketing authorisation and availability in the country, while Romania ranks last, with an average of 883 days.

Out of 152 innovative medicines approved by the European Medicines Agency between 2016 and 2019, only 39 (1 out of 4) have been included on the list of compensated and free medicines in Romania by 1 January 2021.

Germany introduced 133 medicines for compensation, Italy 114, Slovenia 78, Bulgaria 57 and Hungary 55. Thus, the availability rate of the latest generation of medicines for Romanian patients in the compensation system is only 26%, while 74% of medicines are available neither in the compensated nor in the private system.

The main causes of delays are the waiting time before submitting the reimbursement dossier (waiting for other countries to decide on reimbursement), the bureaucratic process, a Health Technology Assessment (HTA) system that is restrictive towards innovation, and an understaffed team that has a hard and arduous time dealing with dossiers submitted by pharmaceutical companies.

In terms of therapeutic areas, although access to oncology medicines appears to be improving, access to orphan drugs continues to vary considerably between EU Member States, with long delays and low availability in Central and Eastern Europe.

Read also: Romania has one of the worst performances in the EU when it comes to access to treatments for rare diseases

According to the CRA‘s „Report on root causes of market access delays”, there are 10 interrelated factors that explain the lack of medicines in local markets and access delays.

For Romania, the 3 main factors negatively influencing access are :

1. Failure to meet deadlines for assessment and lack of predictability in updating the list of compensated medicines.
2. Lack of multi-annual budget projections and insufficient budget for innovative medicines.
3. Dysfunctions related to the procurement process of medicines in hospitals.

Strengthening EU cooperation on ETM after 2021

The new EU regulation establishes a working method, an implementation timetable, and decision-making structures for evaluations at the EU level. Evaluation reports will have to be taken into account in national processes, but the extent to which this is done is left to the Member States.

Implementation of the evaluation process is expected to start in winter 2024, with the evaluation of cancer drugs and advanced therapies (gene, cellular, etc). It is very important for Romania to define its position regarding the adoption of the European reports, to prepare its representatives for the working groups, and to analyze what and how much of the national legislation will be amended so that Romanian patients can benefit from innovative medicines as soon as possible.

Read also: Romania to negotiate for the first time, at European level, new regulations on patient access to treatments for rare and pediatric diseases

According to national and European experts, access to innovative medicines for Romanian patients suffering from rare diseases can be improved in the coming years, as Romania has for the first time the chance, as a member state of the European Union, to contribute to the revision of the Orphan Medicinal Regulation, which was implemented 20 years ago.

National authorities, European decision-makers, representatives of the pharmaceutical industry responded to the initiative launched by the Media Platform www.caleaeuropeana.ro and the Romanian Association of International Pharmaceutical Manufacturers (ARPIM) and held an open dialogue on the access of Romanian patients to orphan and paediatric medicines, as well as on Romania’s role in the forthcoming negotiations at European level for the revision of the Orphan Medicinal Products Regulation.

To ensure that patients suffering from rare diseases have access to medication, the “Regulation on Orphan Medicinal Products (OMP)” was adopted 20 years ago, introducing specific legislation, a definition of OMP, and a specific committee responsible for OMP at the European Medicines Agency (EMA), as well as incentives to promote the development of treatments for rare diseases.

Read also: Romania has one of the worst performances in the EU when it comes to access to treatments for rare diseases

The role of Romania and Romanian experts in the negotiations held by the European institutions

This more than 20-year-old regulation will be re-evaluated, and this is the first time when Romania, as an EU Member State, will participate in the legislative process in the negotiations at the Council level and subsequently in the negotiations between Council and Parliament by submitting successive positions in the negotiations on the legislative act, according to national interests in the matter.

The review of EU legislation addresses the gaps identified by the evaluation with regard to products and their development for the specific needs of children and patients with rare diseases; early access to treatment for these groups and improvement of approval procedures and reduction of inequalities in access.

The European Commission launched another public consultation on 28 September, which will close on 21 December 2021, on the reform of the overall medicines policy framework as part of the resilience of the EU pharmaceutical sector. Thus, the Permanent Representation of Romania to the EU invites key stakeholders in Romania to participate and get involved and, most importantly, make their voices heard at European level.

What happens after the European Commission presents the legislation

Once the European Commission presents the legislative act to the public, the EU Council and the Parliament will start debating it (co-decision). The French Presidency, which will be at the helm of the Council in the first half of next year, will put the proposal on the agenda with a view to finding a compromise with a qualified majority of the EU 27, and the Parliament in parallel will vote on its position at first reading, initially in the ENVI committee and then in the plenary of the European Parliament.

When the Council reaches an agreement it will start negotiations with the European Parliament, the so-called trilogues, and after several rounds of negotiations, a political agreement will be reached between the two co-legislators. Once agreement is reached, the regulation will be adopted and will enter into force immediately after publication in the Official Journal of the EU with immediate legal effect, provided there are no transitional periods and it is a regulation and not a directive. From the moment the public consultation is closed until the publication of the act, Member States, the Council and the Parliament cannot influence the legislative act, as it is pending and in preparation for adoption by the Commission.

Romania will, for the first time, participate in the legislative process in the negotiations at the Council level and subsequently in the negotiations between the Council and Parliament by submitting successive positions in the negotiations on the legislative act, depending on national interests in the matter.

Negotiations between the Member States at Council level take place between the Member States at three levels: at technical level, by Member States’ experts in the working groups on pharmaceuticals and medical devices; at the level of the Permanent Representatives of the Member States and at ministerial level, the EPSCO Council (health component).

In addition to participating in public consultations, Romania will have a new opportunity to participate, contribute and influence the EU legislative process in order to create optimal conditions for ensuring access to treatment for rare and pediatric diseases for the benefit of Romanian patients and to create the incentive framework necessary for the development of orphan and pediatric products for the pharmaceutical industry.

Romania will also contribute to the initiative “a European Pharmaceutical Strategy” and will support ensuring the availability of innovative and affordable medicines for patients, as well as supporting the competitiveness, innovation capacity and sustainability of the pharmaceutical industry in Romania and in the European Union.

Opinion article signed by Ion I. Jinga, Ambassador, Romania’s Permanent Representative to the UN

Motto: “Our problems are man-made, therefore they may be solved by man. And man can be as big as he wants. No problem of human destiny is beyond human beings” – John F. Kennedy

In September 2020 the world leaders committed to upgrading the United Nations and tasked the Secretary-General to produce a report on how to respond to current and future challenges. One year later, António Guterres responded with his report on “Our Common Agenda” (General Assembly document A/75/982), presenting proposals on twelve areas of action: “leave no one behind, protect the planet, promote peace, abide by international law, place women and girls at the centre, build trust, improve digital cooperation, upgrade the United Nations, ensure sustainable financing, boost partnerships, work with youth, and be prepared”.

Our Common Agenda report is one of the most far-reaching and comprehensive strategies ever produced by the UN. It was crafted on the basis of consultations involving over 1.5 million people, including national and local governments, business community, young people and civil society, from 147 countries. It looks ahead to the next decades and represents a vision on the future of global cooperation based on inclusive, networked, and effective multilateralism.  

In a nutshell, Our Common Agenda is set under four headings: strengthening global governance, focusing on the future, renewing the social contract between governments and people, and ensuring a UN fit for a new era. It tackles “the triple planetary emergency” (climate disruption, biodiversity loss and pollution destroying our planet), social equity, human rights and implementation of the sustainable development goals.

Our Common Agenda is not focused on the United Nations, and the States are not the only actors in the picture; it is primality about people, partnerships and results, and provides a 360 degrees analysis of the state of the world, with 90 specific recommendations. For those who are familiar with the Secretary-General’s priorities during his first term, this approach is not a surprise, being anticipated by the report “Shifting the management paradigm at the UN:  ensuring a better future for all” (document A/72/492) presented to the General Assembly in September 2017, which encapsulates António Guterres’ concept of “networked multilateralism” : “The UN works hand in hand with regional organizations, international financial institutions, development banks, specialized agencies and civil society, in order to bring multilateralism closer to people”.

It is also worth to note that during his first term a key-word was “prevention”. Now, Our Common Agenda refers to a key-triangle: “prevention – adaptation – resilience”.

Maintaining peace and security is at the core of the UN Charter. But today peace and security is more than avoiding war. It implies safeguarding the global commons, mitigating climate change, managing public health and the global economy, making the internet affordable to all, and ensuring sustaining peace – a new concept mentioned for the first time in the second review of the UN peacebuilding architecture (twin resolutions A/RES/70/262 and S/RES/2282 (2016)): “Sustaining peace should be broadly understood as a goal and a process to build a common vision of a society, aimed at preventing the outbreak, escalation, continuation and recurrence of conflict, addressing root causes, assisting parties to conflict to end hostilities, ensuring national reconciliation, and moving towards recovery, reconstruction and development.”

Under this paradigm, peacebuilding is no longer a post-conflict phase but a part of the new concept of “peace continuum”: it happens before, during and after a conflict. Our Common Agenda confirms this view: “To protect and manage the global public good of peace, we need a peace continuum based on a better understanding of the underlying drivers that sustain conflict, a renewed effort to agree on more effective collective security responses and a meaningful set of steps to manage emerging risks.”

In order to achieve these aims, a New Agenda for Peace is envisaged and the Member States are called to consider “allocating a dedicated amount to the Peacebuilding Fund from assessed contributions, initially through the peacekeeping budget and later through the regular budget”. This is in line with the proposal the Secretary-General made his 2020 report on peacebuilding and sustaining peace (twin resolutions A/74/976 and S/773 (2020)): “Member States commit the equivalent of 15% of the final full-year budget of a closing peacekeeping mission to be contributed to peacebuilding activities each year for a period of two years following the end of a mission mandate.”

On December 21^st, 2020 the third review of the UN peacebuilding architecture (twin resolutions A/RES/75/201 and S/RES/2558 (2020)) reconfirmed that peacebuilding financing remains a critical challenge and decided to convene a high-level meeting of the UN General Assembly at the 76^th session: “to advance, explore and consider options for ensuring adequate, predictable and sustained financing for peacebuilding”. Undoubtedly, the peace continuum and the appropriate funding for the Peacebuilding Fund are top peacebuilding priorities.

Therefore, the New Agenda for Peace might encourage Member States to commit more resources for prevention, including by tailoring development assistance to address root causes of conflict, upholding human rights and linking disarmament to development opportunities. As Rosemary DiCarlo, the UN Under-Secretary-General for Political and Peacebuilding Affairs, pointed out at the 10^th Ministerial Conference of the Communities of Democracies, chaired by Romania, on September 22^nd, 2021: “Conflict prevention is less costly in human lives and resources than picking up pieces after war. Prevention also means helping to build peaceful and resilient societies – the fundamental goal of the Sustainable Development Agenda.”

Our Common Agenda mentions the Peacebuilding Commission contribution to reshaping responses by the UN to multidimensional threats, and suggests to expanding its role to addressing the cross-cutting issues of security, climate change, health, gender equality, development and human rights, from a prevention perspective. Peacebuilding should also focus on placing women, girls and youth at the heart of peace and security policy, and on the UN transition missions.

Women and youth empowerment, cross-border and regional peacebuilding and support to the transition of UN peace operations are the three priority-windows assigned to the Secretary-General’s Peacebuilding Fund. These have been reconfirmed in September 2021 by the Security Council first-ever stand-alone resolution on the transition that follows deployment of United Nations peacekeeping missions (S/2594 (2021)), which: “underlines that the transition of UN peace operations should support peacebuilding objectives and the development of a sustainable peace, in a manner that supports and reinforces national ownership, national priorities and needs of the host State and its population, and includes engagement with the local community and civil society, with the full, equal and meaningful participation of women, youth and persons with disabilities.”

Interdependence is the logic of the 21^st century. In order to remain effective, the UN needs to become a platform to foster the networked multilateralism envisaged by the Secretary-General. On October 24^th, the Organization celebrates its 76 anniversary; the day after, Member States will gather in the General Assembly to discuss Our Common Agenda report and to agree on the follow up. Our Common Agenda will only be achieved if all Member States are genuinely on board. The adoption of a procedural resolution to provide the framework for continuing inclusive consultations would certainly help to keep the momentum.

Post scriptum:

When he presented “Our Common Agenda” to the General Assembly on September 10^th, 2021, António Guterres confessed: “I am an engineer. I believe in the infinite capacity of the human mind to solve problems. When we work together, there is no limit to what we can achieve”. I share the belief that problems we have created are problems we can solve. Like him, a long time ago, I was an engineer in Physics.

Note: The opinions expressed in this article do not bind the official position of the author nor do they necessarily reflect the editorial views of CaleaEuropenă.ro.