SIAR and ACN at European Parliament


SIAR and ACN at European Parliament


Focus on timely access to innovative medicines after the European Commission approval

1. Introduction (Mariano Votta – ACN)

The meeting is hosted by MEP Brando Benifei (S&D Group) and is organized by Active Citizenship Network in partnership with SIAR, the Italian Society for Regulatory Activities and intends to be a first follow-up step of the last European Patients’ Rights Day, occurred on the 10th of May 2017. Indeed, the meeting aims to stimulate the European issue on access to medicines and sustainability of healthcare systems, promoting a reflection on all possible options to ensure a more rapid real access to innovative medicines and strengthening the specific knowledge on the topic within patient organizations, for a more effective advocacy towards national regulators, ministries of health, etc.
As stated in the 7th right of the European Charter of Patients’ Rights, each individual has the right to receive necessary treatment within a swift and predetermined period of time and this right applies at each phase of the treatment.
At this time the process for Marketing Authorization (MA) consists of a set of steps and procedures to ensure the drug safety and the safeguard of citizens’ health. The current rules tend to seek a balance between the protection of health and the need to give quick answers to patients who often are in urgent need to treat severe conditions. Unfortunately, this collides with approval and reimbursement times that sometimes are very, if not even too, long.
Almost all the innovative and important drugs follow the centralized procedure to obtain marketing authorization (necessary time: about one year). After that starts the price-setting procedure for reimbursement, which can last more than an additional year. Thus, depending on the institutional framework of each Member State, citizens can then rely on the actual availability of an authorized drug only after a quite long period.
In March 2014, the EMA has proposed a first solution to speed up patient access to new medicines, identifying the adaptive pathways. The approach, formerly known as “adaptive licensing”, is a process which provides the early authorization of an innovative drug for a targeted subgroup of patients and its subsequent expansion depending on the evidence gathered. This approach speeds up access and strengthens the collaboration between all those involved in the life cycle of the drug, but at the same time, pushes patients to accept a higher level of uncertainty and risk.
Then, in March 2016, EMA has launched the “PRIME” program (PRIority MEdicines) which provides for the acceleration of the drugs regulatory path, supporting companies (including academic start-ups) and reducing evaluation times (50 days instead of 210). This new procedure will focus on the development of drugs considered priorities, which can offer great advantages over existing treatments or provide an opportunity to patients with no other treatment options.
However, this process relates to the centralized procedure for the marketing authorization by the European Commission but not the procedures for defining pricing and reimbursement, which depends on each Member State.

2. Brando Benifei (MEP)

He agrees about the importance of the topic “timely access of important Medicines” , which is inside the foundamental rights of European Citizens, rights which are not always respected.

European Parliament’s reccomendations about this issue concern the re-launch of the Directive about Transparency, the Legislation about HTA to armonize the criteria for the assessment and to include direct patient involvement and, finally, the increase of the power of concerned Authorities for the negotiations.

He recognizes how the solution of this issue, at national level,  is difficult and the discussion at european level might be useful.

3. Enrico Bosone (SIAR)

Short presentation of SIAR, a no profit Association, in force for 30 years, always careful to the european activities and having its main mission in the educational field.

He explains that many SIAR Members deal also with the price-reimbursement procedures in addition to the procedures for the Mas also because AIFA deals also with the reimbursement.

On this basis, the analysis of the situation in Europe springs. This activity concerns 7 Countries (“big Five” plus NL and Belgium).

For the time being, just Germany makes quickly available the medicines approved by the European Commission. In the other european Countries delays of about 1 year are reported: a situation which is not acceptable in case of medicines for severe diseases and when the new therapy has an advantage for the patient, in comparison with the current best clinical practice.

He shows SIAR’s proposal to establish, at national level, procedures for the timely availability for medicines already recognized by the european procedure of MA, being for additional advantage and for severe diseases.

This proposal has the objective to magnify and take advantage by the work already performed by EMA and by the European Commission.

Finally he suggests that, if requested by the Applicant, EPAR might include a chapter dealing with these two important parameters : severity of the disease and unmet medical need.

4. Olga Solomon ( DG SANTE)

The issue of the “Access” have being discussing for many years. Recently (June 2016) documents concerning this topic have been released by the European Commission and the Council.

She recognizes the limitations of the role of the European Commission about this issue.They are working on a possible modification of the “EPARs” increasing the information regarding the severity of the disease and the comparison with the current alterrnatives.

Recently an analisys on the “Conditional” approvals has been published: in Europe the “compliance” with the obligations connected with the MA have been respected. In addition to this, no particular issues have been emerged to confirm the initial conditional MA. In any case, the number of the CMAs is limited at about 10%.

Finally she informs about the activities of the EC to support the harmonization of HTA methodologies.

5. Lucia Ruggieri (Fondazione Benzi, TEDDY Network)

The intervention has highlighted the need to involve paediatric patients in all health-related issues. In the light of the empowerment activities that involve adult patients in complex and high-impact topics, it is necessary to structure appropriate methodologies for the paediatric population.

At European level, both in the legislative and methodological fields, the interest for children’s health has considerably grown over the last 20 years. Currently, Young Persons Advisory Groups (YPAGs) are an opportunity for sharing experience and get an active involvement of children and adolescents. Expanding this perspective will allow to include discussions on complex issues and gather the views of younger patients in the debate about timely access to treatments.

6. Ri de Ridder (RIZIV/INAMI Belgium)

He points out the difficulties of the national Authorities to accept prices which are higher and higher, not in compliance with the sustainability.

He shows the activities to allow an early access, also before the European MA, but on the basis of a priority list, filled by the Belgian Authority. For example, between the priorities, the Spinal Muscolar Atrophy (SMA) is reported.

He informs about the collaboration between the Authorities for reimbursement: the initial group made by Belgium, Netherlands, Austria and Lichtestain has now included other 15 European National Authorities, in particular for the “Horizon Scanning”.

A recent joined pilot activity by Belgium and Netherlands has ended up with a joined negative opinion for the reimbursement of a new medicine for Cistic Fibrosis, because a price which has been evaluated as too high.

7. Gabriella Almberg (EFPIA)

She shows the position of the pharmaceutical Companies which work in Europe for research, development and marketing.

She starts by the increase of the age population and the increase of the health request. They create new challenges for all the system. She shows that, during the last years, the Health expenditure continues to increase,  but the expenditure for therapies is sligtly decreasing.

The Pharmaceutical Companies propose to use the parameter based on the “Real World Outcomes” when an evaluation of the investements in health care has to be performed. This methodology should be used not just for pharmacological therapies but for all the activities in Health Care.

8. Neil Bertelsen (HTAi Interest Group)

He underlines that the involvement of Patients in the evaluation for MA and also for the HTA becomes more important where the level of uncertainity is higher because the urgency in making available new therapies for severe diseases and with unmet medical need.

9. Conclusions

Of course a solution for the timely availability of “prime” Medicine cannot be found and agreed during a meeting of few hours. However there is a consensus on the usefullness of an exchange of ideas, experiences and how this activity can be useful to progress.

SIAR’s proposal will take into account the contributions emerged and the proposal could be discussed at national level, with the aim to create a pilot experience which could pave the way also for other European Member States.


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