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Timely access to priority medicines in Europe

Abstract

Many tools have been developed in Europe to accelerate the access to and the availability of medicines. However, this is currently governed by the national Member State procedures for pricing and reimbursement. In many cases, this leads to procedures that often take many months or even years to be completed. This paper explores ways that would allow a more accelerated approach and thus enable a more efficient administrative procedure to be adopted. Therefore, this would favor the timely availability of medicines for severe diseases when an unmet medical need is present.

Med Access @ point care 2017; 1(1): e131 - e133

Article Type: POINT OF VIEW

DOI:10.5301/maapoc.0000021

OPEN ACCESS ARTICLE

Authors

Enrico Bosone, Giovanni Giuliani, Nicoletta Martone, Anna Ponzianelli, Valeria Viola,* SIAR Working Group on Market Access on behalf of
* See Author List tab

Article History

Disclosures

The views and opinions expressed in this article are those of the authors and SIAR WG and do not necessarily reflect the official policy or position of Companies.
Financial support: No grants or funding have been received for this study.
Conflict of interests: G.G. is an employee at Roche SpA; N.M. is an employee at Celgene Srl; A.P. is an employee at Novartis Farma SpA; V.V. is a consultant for AstraZeneca SpA, Abbvie Srl, Biogen Italia Srl, Celgene Srl.

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One of the most defining characters of the pharmaceutical industry is the accelerated scientific progress, which leads to the development of new medicines every year, which should be authorized by the European Commission (EC) before entering in the market.

In the last 20 years, the EC issued different legislations and rules to foster this innovation, focusing on medicinal products considered essential – or at least particularly useful – to treat diseases where the unmet medical need is the highest, or where the incremental benefit is significant:

Regulation 141/2000 for the Orphan Medicinal Products (OMP)

Regulation 507/2006 for the Conditional Marketing Authorization

Regulation 726/2004 for the Accelerated Procedure

Article 14(11) of Regulation 726/2004 for the extension of indication with significant benefit

PRIME new scheme, launched in 2016, to enhance support for the development of medicines that target an unmet medical need.

Moreover, in the last few years the European Medicine Agency (EMA) enhanced synergies with health technology assessment bodies and among Member States through some initiatives aiming to define relevant data for all the stakeholders (regulators, payers, and others) to successfully enable patient access to new medicines.

However, as is known, all these rules and initiatives are linked to the EC decision on the marketing authorization (MA) of medicinal products, but they are not fully considered in the national reimbursement processes, when price negotiation occurs. As a matter of fact, in many cases, these national procedures require many months – or even years – to allow access to all the patients in need.

An analysis led by a SIAR (Italian Society of Regulatory Affairs) Working Group on orphan drugs, which received an MA from June 2012 to December 2014 shows that the average time from the EC decision to the conclusion of national procedures varies from 60 days to more than 3 years (Tab. I) with an average of around 1 year (1).

Time from the European MA to the conclusion of the national reimbursement procedure (orphan drugs)

Country Average (days) Minimum (days) Maximum (days)
MA = Marketing Authorization.
Belgium Similar timing of oncological drugs: see Table II
France 196 118 330
Italy 487 200 800
Netherland 173 0 431
Spain 574 312 1073

Another publication showed similar timing to reimbursement for oncological drugs (2); similarly, the analysis of the SIAR Working Group confirmed the great time lapse from the EC decision to national reimbursement (Tab. II).

Time from the European MA to the conclusion of the national reimbursement procedure (oncological drugs)

Country Average (days) Minimum (days) Maximum (days)
The table shows the time needed to obtain the final price, even though access to the drug was granted from European MA.
MA = Marketing Authorization.
Belgium 444 248 737
France 188
Italy 446 210 810
Netherlands 117 0 543
Spain 474 199 872

It is clear from Table II that there are significant differences between Member States in terms of access to treatment, reflecting the variety of legislation and procedures to obtain national reimbursement.

To our knowledge, only Germany has specific legislation that leads to timely access: any drug which obtains a European MA is reimbursed in few weeks at a price defined by the company to enable patient treatment. In parallel, the national reimbursement procedure starts to define the benefit produced by the drug, and consequently the price level granted. Very simplistically, often at the end of first year of commercialization after the European MA, the company is called to negotiate the final price – with some difficulties underlined elsewhere (3).

Even if other European countries had followed more or less a similar pathway – involving 1 or more local authorities or specific bodies – defining the benefit produced by drugs and then the price to be reimbursed, none of these reimbursed a drug or even inserted it in the market as fast as Germany, delaying patient access to essential treatments (in some cases).

This discrepancy causes us to question the “real need” of a new medicine. It is common sense that a drug which produces a significant clinical benefit compared to current therapies for a very severe disease should be available more rapidly than a so-called “me-too” drug.

As a matter of fact, it is clear that the five legislations/rules listed at the beginning of this article, which aim to grant an accelerated evaluation, share the criteria cited above: severity of the disease and unmet medical need/additional benefit for patients.

So, at least for medicines included in the “PRIME” initiative by the EMA, it would be useful if every country allowed a quick administrative procedure to enable patient access as soon as MA is granted by the EC, as Germany does. The request for this special procedure should be initiated by the local authorities or a stakeholder different from the Sponsor (i.e., Patient Association, Scientific Community, and so on), at a price defined by the pharmaceutical company. At the same time, the “usual” reimbursement process should start to negotiate the price.

In order to hypothesize how many new medicines could be inserted into the market with the quick administrative procedure mentioned above, we conducted an analysis on all the medicines approved by EC from 1995 to 2015, and obtained the following results (Tab. III).

Total number of European MA and percentages of MA that have been evaluated according to regulations and legislations to foster innovation

EC = European Commission; CMA = Conditional Marketing Authorization; MA = Marketing Authorization; OMPs = Orphan Medicinal Products.
Total number of MA by EC (1995-2015) 878
 OMP 92 (10.5%)
 Extensions with significant benefit 17 (1.9%)
 Conditional MA (excluding OMPs) 6 (0.7%)
 Accelerated procedures (excluding OMPs and CMA) 18 (2.1%)

Over the last 20 years, the total number of “PRIME” medicines has been 133 (15.2% of the total number of the medicines approved by the EC), which is less than 7 medicines per year.

We can hypothesize that this number may increase during upcoming years, since the number of approved OMPs has been increasing, but we believe that it will not exceed 20% of the total number of approvals (i.e., if we calculate an average of 43 medicines approved per year, the total number of “PRIME” will be <10 medicines/year).

However, a number of business models can be employed to ensure that a fair and reasonable reimbursement/price is ultimately established.

To avoid economic burdens that cannot be supported by the national healthcare system, one approach is to apply this automatic procedure only if the forecasted budget impact for the first year does not exceed a certain threshold (e.g., 0.5% of the total drug expenditure). This is in line with the nationwide budget threshold approach described by Messori (4).

Moreover, after the conclusion of the “usual” national reimbursement procedure, the sponsor and the local authority could agree upon a percentage of payback (up to 100%) of the difference between the first temporary price proposed by the company and the final price negotiated.

Another approach might be to control the appropriate use and to monitor the outcomes of the new therapies by using national registers, which already happens in some Member States (5).

In conclusion, the proposal reported in this article would enable timely patient access in European countries for essential medicines for severe disease in which there is a strong unmet medical need, included by EMA in the “PRIME” initiative.

Of course, bold action at a national level in each Member State is needed to promote this solution, but we believe that – through the appropriate tools – it could be sustainable in most European countries (6, 7).

Disclosures

The views and opinions expressed in this article are those of the authors and SIAR WG and do not necessarily reflect the official policy or position of Companies.
Financial support: No grants or funding have been received for this study.
Conflict of interests: G.G. is an employee at Roche SpA; N.M. is an employee at Celgene Srl; A.P. is an employee at Novartis Farma SpA; V.V. is a consultant for AstraZeneca SpA, Abbvie Srl, Biogen Italia Srl, Celgene Srl.
References
  • 1. SIAR news 69,70,71 (Journal of the Italian Society of Regulatory Affairs). http://www.siar.it/Pages/SiarNews.aspx. Google Scholar
  • 2. Martinalbo J Bowen D Camarero J et al. Early market access of cancer drugs in the EU. Ann Oncol 2016 27 1 96 105 Google Scholar
  • 3. Cassel D Ulrich V AMNOG under economic scrutiny. Final report http://www.bpi.de/fileadmin/media/bpi/Downloads/Internet/Presse/Pressekonferenzen/2015/2015-06-08_Cassel-Ulrich-AMNOG-Gutachten_SCHlussfassung.pdf. Accessed October 30, 2017. Google Scholar
  • 4. Messori A Application of the price-volume approach in case of innovative drugs where value-based pricing is inadequate: description of real experience in Italy. Clin Drug Investig 2016 36 8 599 603 Google Scholar
  • 5. Prada M Ruggeri M Sansone C et al. Timeline of authorization and reimbursement for oncology drugs in Italy in the last 3 years. Med Access @ point care 2017 1 1 e29 e36 Google Scholar
  • 6. Curto A van de Vooren K Garattini L Market approval for drugs in the EU: time to change direction? Eur J Health Econ 2017 18 8 933 936 Google Scholar
  • 7. Pani L Montilla S Németh G Russo P Viceconte G Vogler S Balancing access to medicines and sustainability in Europe: An analysis from the network of competent authorities on pricing and reimbursement (CAPR). Pharmacol Res 2016 111 247 250 Google Scholar

Authors

SIAR Working Group on Market Access on behalf of *

Affiliations

  • SIAR President, SIAR, Pavia - Italy
  • SIAR member, SIAR, Pavia - Italy
  • SIAR board member, SIAR, Pavia - Italy

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