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EF: If 2020 was the year of diagnostics and 2021 the year of vaccines, what do you think will be the key healthcare talking points for 2022?

RS: The war in Ukraine has precipitated a severe recession. The global compounded inflation is reaching a level we have not seen in decades and becoming the most significant economic threat. There are exacerbated supply chain shortages in Europe, and, particularly in Germany energy shortages that are a danger to the very existence of many businesses. Our industry is affected by all of the above.  

EF: What are the lessons learned from the pandemic?

RS: The pandemic has given us lessons for the future. The speed with which vaccines were developed is a remarkable aspect, showcasing the public the substantial and positive impact of the pharmaceutical industry. During the pandemic, I found it noteworthy how science, particularly virology, was suddenly the focus of public attention. On TV, people debated different mechanisms of action and how the virus worked, spiking an increased interest in life sciences. It also addressed the serious future issue of accessibility of talent, and how our industry is dependent on talent and research. Another positive aspect of the pandemic was the general acceptance and widespread use of telemedicine, which was for a long time prohibited in Germany. It was later encouraged to prevent vulnerable people from entering hospitals and private practices, thus reducing the risk of getting Covid-19. Like many other European countries, Germany has an aging population, particularly in rural areas, where young physicians want to live the least. Furthering development of telemedicine will help to better address the medical needs of an aging population. From an industry perspective, the pharmaceutical field force was not allowed to visit physicians in hospitals, and virtual channels for interacting had to be developed. Despite face-to-face interaction still playing an important role, digital channels are here to stay. They will play a more important role than pre-2020, saving time and unnecessary greenhouse gas emissions. At a macro-socioeconomic level, we will need to evaluate the efficiency of the measures taken by the government in response to the pandemic. Money was spent, which is now debt and a burden to future generations. A fact-based conclusion would serve as a lesson for future pandemics.

EF: Could you elaborate on your footprint in Germany, six years from startup?

RS: In 2016, we have established a legal entity in Germany and in 2018 we got marketing authorization for the European Union for an enzyme replacement therapy for a rare lysosomal storage disorder. This year, we transferred the European Marketing Authorization of a medicine to treat a severe inherited form of dyslipidemia, from Regeneron Pharmaceuticals to Ultragenyx Germany GmbH. We also have a wholesale trade and export permission resident at our office.  

EF: How do you see virtual interactions and physician education evolving in rare diseases?

RS: Virtual will certainly play an important role in educating physicians with the purpose of earlier diagnosis. According to the alliance of patient organizations for rare diseases, ACHSE, it takes seven years for a rare disease patient to receive the correct diagnosis. In the case of even rarer diseases, it can take longer. These diseases are rare, and we cannot expect a pediatrician or general practitioner to be familiar with all thousands of rare diseases described in the scientific literature. Primary care physicians must recognize signs and symptoms of rare diseases and should be encouraged to refer the patients to a center for rare diseases in Germany. These centers’ purpose is to have a cross-functional approach across multiple medical disciplines and speeding up the diagnosis process and recommending the right treatment. There are still several unmet medical needs in rare diseases for diagnosis and treatment. Online education and artificial intelligence will play a role in accelerating diagnosis. Many physicians are interested in the whole spectrum of rare diseases. A win-win scenario would be creating a joint effort between companies developing orphan drugs to deliver broader and more relevant medical information to a wider audience of health care providers. Communicating about multiple rare diseases will increase the relevance of the information for the recipient and reduce the cost for companies.

EF: How can we increase the importance of rare diseases and orphan drug awareness in Europe? How do you see the Draft Healthcare bill impacting?

RS: The current Bill is an effort to contain costs for the statutory health insurances, and it will very likely reduce the attractivity of the German pharmaceutical market for innovation in general. The effect is not just limited to orphan drugs. We have already seen the trend in Germany that more and more innovative medicines are pulled from the market and are no longer generally reimbursed.  

This is a warning sign that accessibility to innovative medication –even before this Bill- is not as good as the German healthcare system prides itself on being. The German healthcare system is one of the most expensive systems in the world without producing above-average results. There are healthcare systems that have equal results at a lower cost, such as Denmark. The underlying reasons must be analyzed. One of the most striking differences is that in Denmark, there are less hospital beds per capita than in Germany. The hospital sector consumes the most resources for statutory health insurance revenues. I would encourage healthcare policymakers to review the entire German healthcare system and address their structural problems: Are hospitals delivering the desired quality? Could interventions performed in hospitals in Germany, with equal quality, be done in an outpatient setting thus optimizing resources? There is a need for restructuring and substantial reforms.

Another room for improvement in the sector is that Healthcare-related data is not shared between the different healthcare systems stakeholders, and we do not leverage the synergies that sharing data could enable. As a result, there are double interventions, analyses, and unnecessary imaging because data is not shared. Of course, there must be a balance to protect people’s privacy.

EF: Shifting to Talent Acquisition now. How do you attract the best and the brightest to the organization?

RS: Being a highly specialized organization that caters to small patient populations does not make it a challenge to hire the best. I have recently recruited for several vacant positions without using external service providers. A company with a future that develops innovation will always be considered an attractive workplace. It is always exciting to build something new. Ultragenyx is perceived as an attractive employer in the labor market, and we reinforce that by offering flexibility to our employees.

EF: What is the level of access to innovation in Germany compared to other markets?

RS: Historically, access to innovation has always been very good in Germany, mainly because new products are reimbursed before price negotiations with payers are concluded. The Bill that entered into effect recently may negatively affect this. A future scenario may be that Germany would not always be the first country to launch a new product in a European context. Germany is the biggest market within the EU, and a negative scenario has repercussions on the attractiveness of the EU overall as a pharmaceutical market for innovation.  

EF: In 4 years from now, on your tenth anniversary, what would you like to celebrate?

RS: On our tenth anniversary, I would like to celebrate having made more innovative medicines accessible to rare disease patients. We have multiple drugs in development that address inherited errors of metabolism that have a high burden of disease and a severe impact on the patient’s quality of life. Patients with rare diseases still have high unmet needs, and it is our objective at Ultragenyx to address those needs in severe genetic diseases. We have a broad pipeline, which is remarkable for a company our age. I would like Ultragenyx to be widely recognized as an innovation leader in rare diseases and as a trustworthy partner for stakeholders in healthcare by taking care of patients. Finally, I hope we get inflation under control. The consequences of an uncontrolled inflation are that it will take away purchasing power from people; the rising energy cost can ruin industries and create unemployment, eroding the income of statutory health insurance and taking away from investment in healthcare.

November 2022