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Sophie Schmitz attended world orphan drug congress

Many rare disease patients lay their hope in gene-therapy development. However, recent experience with Glybera (uniQure) has shown difficulties faced in the launch and access. During the World Orphan Drug Congress in Washington this month, Partners4Access had the opportunity to get latest insights from a German sickness fund that reimbursed Glybera and the CEO of UniQure.

Glybera is one of the few approved gene-therapies, it is approved for familial lipoprotein lipase deficiency (LPLD), and nicknamed “the world’s most expensive drug” with a 1-million-dollar price tag. Since its approval in 2012, it has only been prescribed to one patient. Partners4Access listened to the insurer that paid for the treatment, the German sickness fund DAK and the UniQure CEO Matthew Kapusta.

Detlev Parow head of care management and development at the DAK, discussed his experiences with Glybera and solutions for future products. “Glybera was a case where we had more doubt than optimism, the patient’s situation improved, but the patient isn’t cured. Paying $1million for the treatment with 28 injections is what we consider excessive”

Asked about his plans for Glybera, Matthew Kapusta answered “[it was] one of the hardest decisions of my career not reviewing the license for Glybera 5 years after it was approved in Europe. With only 1 patient treated, the regulatory, compliance, manufacturing and registry costs were too high. We want to be more focused on areas that have greater impact to patients at large”

Discussing the price and the future, Parow continues “we need to think about different approaches to evaluate and pay for such products” he adds “cost benefit assessment instead of early benefit assessment could save us time and disappointment later in the process. Germany is very generous with its current free pricing but the Glybera case shows how this is going overboard, I am in favour of a free price threshold, it might support uptake” Partners4Access asked about his vision for future product launches in the gene-therapy space, looking at best practices in other countries. “The UK has the cancer drug fund for drugs with a high level of uncertainty, setting up a risk fund for highly expensive drugs with a limited certainty, as sickness funds we have a limited budget available and need to ensure sustainability” he continues “we are committed to provide patients access to treatment and cures, however, this will need flexibility from all parties, the current financial model of sickness funds is not prepared for high upfront payments, models as payment by instalments, risk sharing and price caps have to be openly discussed.”

At Partners4Access we are focusing on the support of companies that develop specialised therapies, and are working in collaboration with involved stakeholders across countries to find the optimal and fair solution for patients, companies, and payers.



At Partners4Access, we believe patients with rare diseases deserve the right to access the most appropriate treatment. Our vision is ensuring fair access for all key stakeholders. We partner with our clients to think carefully, plan smartly and implement effectively to successfully secure access for orphan drugs. To receive more information please follow us on linkedin

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