ExCEEd Orphan
Heralding Innovative Medicines into CEE Countries

About 68 percent of children afflicted with Type 1 Spinal Muscular Atrophy (SMA) die before their second birthday, 82 percent of them cannot make it to their fourth. Those who do survive are rarely able to stand or walk unaided. Yet, if a child from an impoverished family is born with this rare disease, their chances of survival without the right treatment are slim to none. As unfortunate as it is, many healthcare organizations fail to successfully treat similar rare illnesses (orphan diseases) due to either absence of any effective treatment or insufficient funding from the government. But with over 100 years of cumulative experience in the field of rare diseases, ExCEEd Orphan was established to tackle that very problem—ensuring that such innovative medications reach the maximum possible number of patients suffering from rare diseases in Central and Eastern European (CEE) countries as soon as possible after EMA approval.

Unlike consultancies that only offer advice to orphan drug companies but do not actively help them improve outreach, ExCEEd Orphan provides insights, formulates and most importantly executes multiple reimbursement strategies to deliver rare disease medicines in each country. Additionally, in accordance with national regulations, the company can provide distribution of orphan drugs as it coordinates with selected specialized distribution partners in each CEE country. ExCEEd Orphan also helps BioTech and Pharma companies create an accurate representation of the patient demographic across these regions. This extends to analyzing the total number of patients, how many are diagnosed, and how many are eligible for specific treatments. “From identifying patients and organizing premarketing strategies to coordinating marketing and distributing medicines, we offer a comprehensive business solution where clients need only one partner for a successful market entry,” says Jiri Hermanek, CEO and Founding Partner.

The strength of ExCEEd Orphan lies in the company’s deep understanding of local market environments in each individual country within the CEE region combined with the team’s extensive experience of launching multiple rare disease medicines (including gene therapy) in those countries.“Providing ample market access for both patients and clinics is the cornerstone of our company,” Hermanek adds.

Providing ample market access for both patients and clinics is the cornerstone of our company

By working closely with key stakeholders in CEE countries and fostering relationships among regulators, payers, patient advocacy groups, and decision-makers, ExCEEd Orphan offers a discernible advantage to BioTech and Pharma companies who aim to maximize their business opportunity in Europe. In efforts to ensure the success of orphan drug programs, ExCEEd Orphan also educates KOLs (Key Opinion Leaders) such that they become familiar with the treatments. Recognized KOLs who spread unbiased information about innovative treatments are essential for an optimal product launch and can also advocate government reimbursements for new treatment options. ExCEEd Orphan has stayed true to this method of operation, and it has resulted in the company orchestrating multiple successful projects for its clients.

One such case study involved the premarketing of Zolgensma –a gene therapy medication used to treat SMA. ExCEEd Orphan was hired by AveXis, a company that developed this gene therapy, to strategise the premarketing operations and create market access in CEE countries. Firstly, ExCEEd Orphan mapped the market and found centres in each country that could treat SMA. The company then worked with KOLs, who were instrumental in devising unique alternatives to the treatment and creating peer-to-peer discussions with the scientific community. Finally, since the treatment was available only to children of a specific weight category, ExCEEd Orphan also gathered data of patients who met the criteria. The project was immensely successful allowing numerous children suffering from SMA to benefit from gene therapy treatment even before EMA approval. “Allowing a child with a debilitating illness to crawl and walk and slowly turn their life around after their therapy gives us one of the highest levels of job satisfaction,” says a proud Hermanek.

For BioTech and Pharma companies, achieving early market access simultaneously in multiple countries helps them bring innovative therapies to patients in need and also expedites early revenue generation. And this is precisely what ExCEEd Orphan provides – starting from market mapping and continuing with pre-approval medical communication with selected key stakeholders to recommending optimal market access strategy, negotiating reimbursement, and finishing with focused marketing and distribution. All these services result in clients gaining a highly cost-effective, impactful, and low-risk entry into the usually not easy to penetrate CEE markets.