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Cem Zorlular is CEO 0f Er-Kim Pharmaceuticals in Turkey, a company that works with small to large pharma and biotech companies to commercialize their medicines in markets that fall outside the United States and the Western Europe. “We work the whole gamut of getting the products registered, reimbursed, and delivering access to the patients,” Zorular says.
“So as a result of that, we have to develop a key understanding on how these payers are thinking, how they’re approaching, and how they’re planning to change the way they look at things,” Zorlular says. “So therefore, we basically as a company need to develop a very keen understanding of the way the west thinks – and by that I mean the Western markets, the U.S. and Western Europe – to better anticipate how our regulators are going to approach an issue, and also how our market sits in terms of other pharmaceutical markets. As price goes up, it becomes less expensive in our markets, whereupon relative price might go up in the future if the Western regulators change their approach to pricing as well.”
At present, when it comes to funding and accessing treatments for rare diseases, “we’re actually in a position where our regions are coming from relative lower funding to higher funding – there is increased support from the European Union, and there is increased economic development in the countries [in which] we work,” he says. “So overall, we’re actually seeing an increasingly positive environment for the patients to be able to access to rare disease medicines, in terms of reimbursement and price negotiations.”
When payers determine if the new technology offered by a rare disease medicine justifies the cost, Er-Kim works with them to build an access plan that ensures that there is enough budget, and the budget is being used properly for these patients, Zorlular told Med Ad News.
There is also an increasing focus among these payers to think more holistically about rare diseases. “A number of regulators are not only funding the rare disease treatments, but they’re also working with us or our partners or other companies like us to improve the diagnosis of rare diseases … prevention methodology such as genetic screening prior to marriage, counseling couples to make sure that if they have a higher chance of having a baby with a congenital disease, they are warned ahead of time,” Zorlular says.
Although the Eastern European payers follow EMA single market rules, other regions that Er-Kim is active in do not, and therefore are a bit more flexible when it comes to access negotiations for rare disease drugs. “But the additional flexibility comes by looking at the U.S., and they don’t look at U.S. payers, but U.S. practices,” Zorlular says. “When you’re in a negotiation discussion, [if] the product is in the guidelines in the U.S., what FDA says does come into discussion a bit more. But from a reimbursement perspective, Europe has a very high reputation of negotiating very strongly. So therefore, once there’s a price in Europe, then that becomes the main center of discussion.”
And when it comes to patient access programs for rare diseases, these regions are investing more, Zorlular says. “Overall, budgets are increasing. And there is increasing willingness to build unique programs to improve access. But one thing that’s nice to further develop is the infrastructure. You can talk about very advanced payment methods and look at results-based payments, but they need to come with a very strong infrastructure to track those outcomes. And that’s why these are not necessarily being favorites above more traditional access pathways. That doesn’t that there is lack of innovation, but usually innovation comes from the perspective of having simple but innovative access models.”
And by infrastructure, Zorlular is referring ways of tracking, storing, and processing healthcare data. “Certain payment models are specifically built around being able to properly collect data, validate the data, and analyze it in a way that makes all sides comfortable with the results. And that requires a fair bit of IT infrastructure favor of connection in terms of national databases, and this is not as uniformly available as it is in other countries,” he says.
At the end of the day, “these are growing countries, and some have very high budgets,” Zorlular says. “But overall, budget allocation might be an important consideration.”
Rare disease access programs have been benefiting in these regions as part of a general push to continuously invest in overall health, because it’s an important driver of the country’s growth, Zorlular says. For example, in Eastern Europe, programs are being started to battle antimicrobial resistance, cancer, and heart disease.
And in rare diseases, one country established a national registry of a rare disease and is trying to implement birth screening. “Turkey is actually one country that is very forward thinking when it comes to these types of measures, investing in decreasing the incidence of congenital diseases through population counseling,” Zorlular says. “Overall, we’re seeing that being changed into an investment mindset, if not maybe more than what we’re seeing in Western Europe.”