The 2019 budget law introduced important innovations in the pharmaceutical sector, bringing an EQUITY mechanism that did not exist before, in the law regulating expenditure ceilings on pharmaceuticals. The compensation (so-called payback) in the event of exceeding the programmed spending ceilings will take place on the basis of one's share of turnover and not through abstruse methods which have led over time to over 350 appeals and a lack of collection for the regions of over 2.5 billion euros only for the period 2013-2017.

A deductible has been introduced for ALL companies (principle of progressiveness) and therefore the first 3 million euros of turnover do not participate in the shelf mechanism. An advantage that will help many SMEs in the sector that represent more than 90% of our economic system. This will mean greater investment opportunities in research and development for many more companies than before with positive effects for the whole sector. In short, by applying the principle of progressivity, THE MORE INVOICES THE MORE PAYS, I am not giving any "gift to big pharma" as someone MISLEADINGLY AND INCORRECTLY wrote, but a decidedly fairer system will apply than in the past.

It is worth recalling that the new law provides for the exclusion from the sales shelf of orphan drugs included in the list of the European Medicines Agency (EMA). A further facilitation compared to those already provided for by the European regulation.

This is to say that those who tell inaccuracies about these new rules do not actually do it for the benefit of patients, but do it for the super big names in Pharma, those with the biggest turnovers. Yesterday, for example, the news that one of the largest companies holding patents for orphan drugs (including a single orphan drug that had a turnover of 193 million euros in 2017) was acquired for 74 billion euros . So in the new system there is no punitive intent for the industry (God forbid!!!), but a fairer system of monitoring and payments.
No rights or facilities for rare patients have been affected. Patients come first.

Giulia Grillo – 4 January 2019 facebook

Related news: Maneuver. Payback for orphan drugs: 200 million removed

The clash between Giulia Grillo and the Observatory for rare diseases on orphan drugs and the budget law

OMaR. Orphan drugs: the controversy over the budget law breaks out

EU Regulation 141/2000

LAW 30 December 2018, n. 145. State budget forecast for the 2019 financial year and multi-year budget for the three-year period 2019-2021.

What was the situation before the Budget law for 2019

The fundamental point of the question is to understand what the situation was before the last budget law, and what instead has changed with the intervention of the Lega-M5s government. The central measure is the so-called pay-back mechanism, with which pharmaceutical companies participate in the expenditure on public sector drugs.

Until this year, the system created by the Budget law was in force for pharmaceutical expenditure on orphan drugs for 2014. This excluded (see art. 1 co. 228) from the pay-back mechanism of pharmaceutical expenditure the turnover of orphan drugs, of "orphan-like" drugs - i.e. "de facto" orphan drugs but without the wording because they were approved before of the European regulation of 1999 which introduced the definition – and those identified in a specific circular of the European Medicines Agency.

The pay-back mechanism for drug expenditure (see art. 5 of Legislative Decree 159/2007 And art. 15 co. legislative decree 95/2012), In summary, it works like this: State law establishes the maximum ceiling for public pharmaceutical expenditure. If this ceiling is breached by the various Italian regions, the excess spending is compensated by companies in the pharmaceutical sector. The companies competed in the settlement based on a complex calculation, operated by Aifa (Italian Medicines Agency) and based on the purchases of medicines by public structures.

As explained by the Parliamentary Budget Office (UPB) in a focus of the June 2017, this system has not worked well in recent years, and has generated a dispute brought by the pharmaceutical industries before the Tar for 1.5 billion for the three-year period 2013-2015 alone. Of this money, moreover, 600 million have not been paid precisely because of the pending appeals before the administrative judges.

According to the Upb, "the difficulties in applying the pay-back are to be found in the complexity of the institutional design, in a constantly changing legislative framework, in the poor quality of the data used, in the insufficient solidity and transparency of the calculation procedures adopted by AIFA ».

As we have said, orphan drugs - and the like - were completely excluded from this sharing of costs deriving from the overrun of the ceiling on public pharmaceutical expenditure. Therefore, the manufacturers of these drugs did not have to pay any portion of the pay-back for them.

What changes with the Budget law for 2019

There budget law for 2019 (art. 1 co. 575-584) has modified and simplified the criterion with which the pharmaceutical industries contribute to the pay-back mechanism.

In particular, it is envisaged (art. 1 co. 578-580) that AIFA determines the market share of each pharmaceutical company on the basis of turnover: the companies cover any exceeding of the pharmaceutical expenditure ceiling pro rata. Companies under three million in turnover gross of VAT are not required to participate in the pay-back mechanism (art. 1 co. 579 letter a)).

What happens to orphan drugs? The Budget Law provides (art. 1 co. 578) that «drugs included in the register of orphan medicinal products for human use of the European Union» are excluded from the turnover calculations.

This register is expected from European regulation of 1999 already mentioned, the one that also gave the definition of orphan drug. The problem is precisely that the register does not include "orphan-like" drugs and orphan drugs that have lost market exclusivity.

The point of contention is therefore the fact that the list has shrunk: according to the calculations made byRare Disease Observatory, an information agency dedicated to rare diseases and cancers, it deals with overall of "39 medicines intended for rare patients".

As for the 200 million euros cited by the Observatory, the latter informed us that it is an approximate calculation (180-200 million, to be precise): it has been simulated how much more pharmaceutical companies producing orphan drugs will have to pay with the new system, based on the numbers of 2017. In short, by increasing the participation of orphan drug producers, the share that everyone else must share (including the large pharmaceutical multinationals) decreases.

Of course, even large companies in the sector deal with orphan drugs: in fact, Minister Grillo had answered that "one of the largest companies holding orphan drug patents (including just one orphan drug that had a turnover of 193 million euros in 2017) was acquired for 74 billion euros".

Having used the previous system necessarily makes the Observatory's quantification inaccurate, given that the system, as we have seen, has now changed. But the imprecision, adds the Observatory, should in any case not affect the order of magnitude of the damage for orphan drug manufacturers. In any case, the Observatory has informed us that, within the next week, the exact estimate calculated on the basis of the new system created by the Budget law for 2019 should be released.

We pull the strings

It is true, as claimed by the Observatory for rare diseases, that with the Budget law for 2019, pharmaceutical companies that produce some particular types of drugs in the so-called "orphan" sector - the so-called orphan-like and those that have lost market exclusivity – will now be included in the pay-back mechanism, for which they will have to contribute to the settlement of the overrun of the limits established for pharmaceutical spending, thus suffering economic damage.

At the same time it is true, as Minister Grillo maintains, that the new system simplifies the previous situation, recognized by official sources as not very functional and at the basis of numerous judicial appeals, and provides for a deductible of 3 million euros which will benefit especially smaller companies.

Conclusion

Overall, it therefore seems exaggerated to speak of a "gift to multinationals", with reference to the new discipline envisaged by the Budget law for 2019, even if it is true that the producers of some orphan drugs are penalized.

from AGI Fact Checking – January 9, 2019

Budget law 2019

Published last December 31st in the Official Gazette the Law of December 30th 2018, n. 145, State Budget for the 2019 financial year and multi-year budget for the three-year period 2019-2021 (Ordinary Supplement no. 62).

Satisfaction of Minister Grillo

Minister Grillo in a post immediately after the final approval of the Parliament expressed his satisfaction because the maneuver "contains many important innovations for health, which will bring better services for citizens" starting "from the commitment to find adequate resources for face decisively the difficult issue of waiting lists. But not only. The national healthcare requirement will grow by 4.5 billion over the next three years and we have foreseen the definition of a new and pragmatic Health Pact in collaboration with the regions and autonomous provinces of Trento and Bolzano. I also want to underline the extra 4 billion euros to implement a real Marshall Plan for healthcare construction. Finally, a consideration for pharmaceuticals and medical devices: with respect to the latter, we have implemented some measures to make health surveillance registers effective and mandatory; on pharmaceuticals, thanks also to the work of the Governance table, the Budget Law includes rules for the adaptation of the price definition system - stopped in 2001 - as well as for updating dynamic of the national pharmaceutical handbook. Finally, the paragraphs relating to the provisions on pharmaceutical expenditure limits deserve a separate note."

The main loans

• 350 million over three years for the reduction of waiting lists; 150 million euros for the year 2019 and 100 million euros for each of the years 2020 and 2021 (paragraphs 510-511-512)
• 4.5 billion for national health needs in three years. For the year 2019, the level of funding is set at 114 billion and 451 million euros, increased by 2 billion for the year 2020 and by a further 1.5 billion for the year 2021. For the years 2020 and 2021, the access of the regions to the increase in the level of funding is subject to the stipulation, by 31 March 2019, of the new Pact for health (paragraphs 514-516)
• 4 billion euros more to modernize and restructure hospitals. The dedicated fund goes from 24 to 28 billion (paragraph 555)
• 10 million to increase scholarships in general medicine (paragraph 518)
• 22.5 million euros for the year 2019 for specialist training contracts for doctors, which increase to 45 million euros for the year 2020, 68.4 million euros for the year 2021, 91.8 million euros for the year 2022 and 100 million euros per year starting from the year 2023 ( paragraph 521). Furthermore (paragraphs 547-548) doctors in specialist training enrolled in the last year of the related course are admitted to competitions for healthcare management (paragraphs 547-548)
• 5 million euros for the IRCCS of the Oncology Network engaged in the development of new CAR-T anticancer technologies (paragraph 523)
• 5 million euros for the IRCCS of the Cardiovascular Network engaged in primary cardiovascular prevention programs (paragraph 523)
• 25 million Euros in three years for hadrontherapy, an innovative therapy for the treatment of tumours, 5 million for 2019 and 10 million for each of the years 2020 and 2021 in favor of the National Center for Oncological Hadrontherapy - Cnao (paragraph 559).

Consult the text of the Budget law 2019.

Ministry of Health