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Treatment availability for rare disease patients under fire in Europe.
In principle, the European Union offers rich options to its citizens for obtaining high-quality healthcare and the most innovative treatments—and this has been borne out, to some extent, by the wide-scale provision of vaccines during the COVID pandemic. But the system is still imperfect, and major gaps persist, particularly for rare disease patients who might benefit from advanced therapy medicinal products (ATMPs)—as the manufacturers of these drugs have been emphasizing during the summer.
Many of these companies, predominantly smaller or mid-size firms in Europe, argue that they are hit by a double barrier to the use of their products. In the first place, launch depends not only on the already arduous EU market authorization requirements, but also on agreement by individual member countries to reimburse—a notoriously idiosyncratic procedure with varying results. And, secondly, although EU arrangements exist for patients to overcome the consequent patchwork of availability across Europe, these are not functioning adequately, with the result that take-up is reduced.
The challenges of winning reimbursement status for such products are well-documented and have been widely discussed, but ATMP makers are now calling for EU action to ease patient access when a much-needed drug is not available in the member country where the patient resides, but is in another member country. The theory is that a patient can travel across Europe to get that drug where it is available. This concept of cross-border healthcare is inscribed in EU law, as a mechanism to offer equality to EU citizens irrespective of which country they live in. The process of relocating or crossing borders often represents the only solution for many patients. But it is complex in design and onerous in operation, and—say companies—the result is that patients are subject to a zip code lottery: if you are lucky you live in a country where the product is available, but if it isn’t, and you want to exercise your rights to seek treatment elsewhere in the EU, the theoretical right to cross-border care is not delivering, and patients are left out in the cold.
The challenge is intensified by the fact that many ATMPs can require highly specialized clinical expertise and infrastructures. Due to their specificities and the technology used, some ATMPs may require specialist manufacturing and patient treatment processes and not all can be administered in all hospital settings. Highly specialized or qualified treatment centers may be needed, and administration by specialist doctors and nurses with specific training will be required. These circumstances currently do not pertain in all EU countries—so even where a product has been authorized and reimbursed, local patients will still be deprived if the skills and apparatus to administer it are absent.
Another major obstacle is that patients are expected to pay in advance for treatments, with only the uncertain prospect of recovering the outlay from their domestic healthcare insurance agency. Prepayment by patients is not a viable option in the case of ATMPs, where both the cost of the treatment and all the associated costs can be high, covering travel, accommodation, multiple visits to the center of treatment, and perhaps hospital overnights. Nor is it appropriate that member states should be allowed to arbitrate on which planned treatment is required for their patients seeking care abroad, or to engage in lengthy deliberations on whether and when to provide reimbursement in such cases. “Time delays can be a major obstacle to potentially life-saving and life-transforming treatments, with potentially significant consequences for patients,” argues EUCOPE, the organization representing many ATMP companies in Europe. Even a report by the EU’s own internal watchdog, the European Court of Auditors, recently concluded that “it would not be reasonable” to require an upfront payment by patients and oblige them to seek subsequently reimbursement.
The current EU framework for accessing treatment abroad “is not suitable for most rare disease patients,” judges EUCOPE. It says that 10 years after the adoption of the key legislation to provide this right, “the directive has fallen short of its primary objective.” So it is urging closer collaboration between the EU institutions, member states, and others to come up with practical solutions.
Some of those could come from greater use of the Europe’s growing cross-border networks of reference centers that specialize in some 25 rare diseases, with patient pathways connecting each network with the national healthcare systems across the EU. EUCOPE also argues for improved awareness among patients and physicians of the opportunities and rights that are theoretically available. Manufacturers also want to see a reduction in the discretionary nature of the national approval process for cross-border care.
Reflector is Pharmaceutical Executive’s correspondent in Brussels