Illuminating the path for biosimilars: an Italian access story (11 min read)
For years, many patients in Italy had difficulties accessing modern biologic therapies. The lack of clear policies limited the use of biosimilars as cost-effective alternatives. Now, thanks to the persistent efforts of advocates across political and industrial sectors, these state-of-the-art therapies are available for a larger number of patients. A model story about how legislation affects health outcomes.
Oct 25, 2018
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“Difficult,” “tough,” but “ultimately successful” – these are words Michele Uda and Manlio Florenzano use to describe the negotiation process for a new law on biosimilar medicines in Italy. For two years, Uda – director of the Italian Generic and Biosimilar Italian Industry Body (“Assogenerici”) – and Florenzano - coordinator of the Italian Biosimilar Group (IBG, the Alliance of Biosimilar Producers within Assogenerici) and Country Head of Sandoz Italy – met with politicians, and representatives of medical and other pharma associations within a working table organized by the Italian Industry Minister. Their common goal was to improve access to biologic treatment options in the Italian market and support the development of new policies regulating their use. Although this sounds like a bureaucratic task, it was, indeed, a significant change that would determine the quality of many patients’ lives. “Two lights were illuminating our path – the need to grant better access for patients and the need to make the market more sustainable,” Uda says. To understand what was at stake, we need to look back several decades.
The advent of biologic medicines in the early ’80s was a breakthrough in the treatment of previously difficult to treat diseases including arthritis, anemia, multiple sclerosis and certain types of cancer. For the first time, patients could receive therapies based on disease-associated proteins that significantly improved health outcomes1. However, the complex process of producing biologics, which involves living cell cultures, makes them expensive, which resulted in limited access to these treatments. But when patents for biologics expired, a second revolution followed with the development of biosimilars. In 2006, the first biosimilar was introduced in Europe by Sandoz – and it offered a more affordable biologic treatment option. Just like their reference medicines, biosimilars are produced in living cells. And like the reference biologics, biosimilars have to undergo several tests to prove they have the same clinical benefits and match their reference medicine on quality, safety, and efficacy2.
For this purpose, the European Medicines Agency developed a dedicated regulatory pathway for biosimilars, which is recognized globally as a gold standard. For more than a decade, the framework has been installed in many countries worldwide and almost everywhere in the European Union – and subsequently, biosimilars have proven their positive effect on patients’ outcomes and healthcare sustainability.3
Two lights were illuminating our path – the need to grant better access for patients and the need to make the market more sustainable.
These outcomes were also shown in a recent report by the European Commission. The market entry of biosimilars can create healthy competition and therefore healthcare savings, as well as a significant increase in uptake, with a volume gain of more than 50% in some cases4, even doubling in others. So, with access to biosimilars, the cost of biologic therapies decreases while the number of patients who can receive those treatments increases. This results in positive effects on a healthcare system’s sustainability. In 2016, IMS Health (now IQVIA Institute for Human Data Science) estimated that biosimilars could lead to savings up to EUR 100 billion by 2020 in the United States and the five major countries in the European Union, which opens up opportunities to “free up resources for investment in new areas and bring relief to pressured healthcare budgets.”5
Fighting “undertreatment” While patients throughout Europe have benefitted from these innovative treatment options, patients in Italy didn’t have sufficient access. “Although biosimilars were available in our country,” Manlio Florenzano adds, “physicians rarely used them for therapy. Too often, healthcare professionals, patients and politicians didn’t fully accept that, just like the reference biologic, biosimilars are state-of-the-art therapies. As a result, from about 2006 onward, biosimilars accounted for only 10% of the market volumes,” he explains. “The possibilities were there, but still patients remained ‘undertreated’.” A study conducted by Ernst & Young (EY) supported him: It also showed that up to 300,000 Italian patients suffering from cancer or autoimmune diseases, such as rheumatoid arthritis, did not have access to leading biologic therapies.6 Lack of confidence among doctors and patients with these therapies, and the scarcity of specialized biologic centers, are potential explanations for this “undertreatment” cited by the EY analysts. As well, they cited the high cost of biologics as a possible cause. For these reasons, biosimilars represented an opportunity to fill this treatment gap.
The EY report also lead to another question: Why was the situation in Italy worse than in other countries? For Michele Uda, the main reason was a lack of official policy for the use of biosimilars. “The Italian market remained very fragmented and very limited in terms of accessibility of biosimilars,” he explains. This unstable situation had negative impacts for Italian patients and healthcare professionals, but also for producers of biosimilars who sought to offer these novel treatment options. Insufficient and fragmented regulation had made it difficult for biosimilars to be optimally utilized. In addition, says Uda, therapeutic equivalence – a scientific principle of the same clinical effect and safety profile between a biosimilar and a biologic that is already on the market based on the marketing authorization process – was not formally accepted. As a result, “biosimilars were used almost exclusively for the treatment of patients who had not received any biological therapy before,” Uda explains. “There was simply no guidance for using biosimilars in the other patient groups who were in the middle of their therapy.”
But even when a biosimilar became available, doctors and patients couldn’t access it for a long time. Due to a restrictive tender (contract offer to supply the market) process, “biosimilar producing companies had to wait even more than six months after approval of their products in order to enter the market,” Uda continues. And once on the market, regional authorities tried to urge physicians to use the lowest-priced biosimilar medicines, he explains. However, this attempt backfired. “The price pressure thus created reduced the acceptance of biosimilars in general by healthcare professionals. As a result, biosimilars weren’t a treatment option for doctors, and, therefore, patients didn’t have access to these therapies. For the manufacturers, it was like jumping into a pool without water.”
Negotiation, mediation and communication Something needed to be done to clear up all the misconceptions and insecurities in order to improve access to more treatment options. For Florenzano, the need for an official regulation of the biosimilar market in Italy was a crucial step in order to improve the situation. In 2014, he and his colleague Enrica Tornielli, Head of Pharmaceutical Affairs of Sandoz Italy, felt compelled to tackle what turned out to be a long process. “We started working with all stakeholders to define a legislative framework that would open access to biosimilars and provide the opportunity to bring benefits to patients. Doctors, patients, financial regulators and industrial associations’ points of view were taken into consideration to try to find a common solution,” Florenzano explains.
We started working with all stakeholders to define a legislative framework that would open access to biosimilars and provide the opportunity to bring benefits to patients.
Together with Uda, they led frequent deliberations with different stakeholders and prepared briefings for members of the Italian parliament. “It was extremely important for elected officials to understand the benefits that biosimilars could bring to patients’ access to biological therapies and to the healthcare system in Italy,” explains Florenzano. “Every argument was based on summaries of scientific data.”
Overall, their passion and strategy worked hand in hand to contribute broad expertise to the legislative process. “First, we led a wide negotiation among the pharma associations to find a common baseline. After reaching consensus, we presented our shared proposal to policy bodies for consideration,” Florenzano says.
Reaching the goal
At the end of all these steps, a new law on biosimilars was approved in December 2016.7 The focus of this law is to define a standardized framework to regulate the biosimilars market and to ensure open competition that could improve access to much-needed treatment options for patients, either to existing biosimilars or to future therapies. At the same time, the law gives a standardized framework to regulate this market and ensure competition. “It strengthens the role of prescribing physicians, while introducing framework agreements that ensures physicians can choose between different products,” Florenzano explains.
Uda also welcomes the fact that the new regulations support healthcare professionals with their treatment decisions. While it is still too early for a complete assessment of all benefits, initial results are encouraging. “There is now a much better acceptance of biosimilars in Italy than back in 2006 when the first products entered the market,” Florenzano says. Initial data from IBG in March 2018 revealed an increasing trend in biosimilars uptake across Italy, with annual growth of 74% between 2016 and 2017, and reaching 19% of the market share.8 This means that physicians are starting to use biosimilars as a treatment option more often to improve their patients’ lives. Uda summarizes, “Today, we can treat patients earlier, and we can treat more patients than before.” Adds Florenzano, “These results made the long path of negotiation worth it.”
The key principles – and outcomes - of the Italian law on biosimilars
In December 2016, a new law on biosimilars was approved in Italy to improve access to biosimilar medicine in order to increase sustainability of the healthcare system and improve patients’ access to biologic therapies. The key principles of the law are:
Biosimilars and reference medicines have the same therapeutic outcome.
There is no automatic substitution between the reference biologic and its biosimilar, or between biosimilars. Once a biosimilar is available on the market (and wins the tender), a physician must decide whether to switch to the new treatment option or not.
If there are more than three competitors of the same biologic substance, there must be a multi-winner tender model. Patients can be treated with any one of the chosen brands, thus ensuring that there are treatment choices for physicians and patients.
Physicians play a central role. They are free to choose the substance that they believe is the best for their patients.v
When the patent of a reference biologic has run out and a new biosimilar is available, authorities must accept suitable new biosimilars within 60 days. This provides sustainability to the companies entering the market, and ensures a continuously growing number of treatment options for patients.