It is no secret that much money is involved in the pharmaceutical industry. Many types of medicine are expensive to develop and manufacture. The financing of healthcare is very complex. The pharmaceutical industry is exciting, but sometimes rightly, sometimes wrongly, too much money is made. Especially if there is a shortage of medicine because of inaccessibility and availability, treatment can be costly for patients.
Nevertheless, there are possibilities to bridge the gaps: it is why Vincent van der Wel founded Orfenix. This company brings doctors, patients, and social and private investors together, providing business cases to develop profitable and affordable remedies for the patient. Their goal is to spend money in the healthcare industry wisely and not make medicine expensive if it doesn’t have to be.
The beginning – Patient One
‘The idea of Orfenix came after I started two public-private partnerships (PPPs). The first PPP I founded was Patient One. I met a group led by the Fair Medicine foundation during my studies. I then learned about a medicine for cystinosis, a rare disease caused by a build-up of the amino acid cysteine affecting the kidneys and eyes. Patients originally needed to take cysteamine every six hours to prevent further body damage.’
‘An American pharmaceutical company developed a slow-release solution so patients can take it every 12 hours. Unfortunately, this medicine is so expensive that it is still unavailable in most European countries. A group of experts came together in the Centre of Human Drug Research (CHDR) in Leiden, wanting to develop a similar slow-release product for the European market but make it cheaper and thus more accessible to patients. To achieve this, we brought all stakeholders together in Patient One. The company is currently in the second phase of its clinical trials.’
‘In hindsight, I maybe didn’t know enough when starting patient One. However, enthusiasm and good mentorship made up for a lot, and the success of the first clinical trial of Patient One made me realize that I wanted to pursue such endeavors more often.’
From outdated remedy to suitable medicine for rare diseases – Haltex Therapeutics
‘Reading a newspaper, I stumbled upon an article titled: Finally, there is a new medicine for a rare disease, but now it goes off the market. The drug etidronate was traditionally a medicine for osteoporosis. In the University Medical Centre Utrecht, scientists discovered that the medicine could help treat Pseudoxanthoma Elasticum (PXE). In this disease, calcifications occur in various connective tissues, affecting the elastic tissues in the skin, eyes, and blood vessels. However, when the first clinical trial in the UMCU showed promising results, no one made the drug anymore since other medicines were available for osteoporosis. So, we decided to create another public-private partnership to reinstate etidronate, this time not for osteoporosis but for PXE. Of course, with a healthy business case for both the pharmaceutical companies and the patients.’
‘This proved a challenge. The raw material for the drug was hard to find. However, eventually, I met a family-owned pharmaceutical company in Buenos Aires, Argentina. So I went to Argentina to invite the company to join our journey to develop this old drug for the new indication. They have joined the journey and have already produced the raw material. In the Netherlands, a subgroup of patients with PXE now has controlled access to etidronate again because we brought doctors, representatives, the pharmaceutical company, and social investors into one company, Haltex Therapeutics. In the next years, we will do more research to investigate the efficacy and safety of the drug to register the drug for new indication(s)’.
Orfenix: no charity, but solid business cases
‘I didn’t want to stop with what I was doing. It is why I founded Orfenix. The Orfenix team is responsible for the day-to-day management of Patient One and Haltex Therapeutics. We focus primarily on tasks related to business development, regulatory and clinical development within the companies. Additionally, we are actively contributing to the Platform Medicijn voor de Maatschappij in the Amsterdam UMC, where we support the development of new therapies from within the academic center. Sometimes the platform helps with small things such as an import permit, but often more work needs to be done to get the right drugs to the patients.’
‘Producing medication takes time, effort, and money. To continue research and development, pharmaceutical companies need a healthy profit. Many processes are complicated and expensive, and that’s okay. But, some medicine shouldn’t be as expensive as they are now, and processes can change. I was positively surprised at the number of companies and investors willing to join our cause for achieving accessibility. Though the more shareholders, the more difficult it becomes. It is where Orfenix comes in: with our backgrounds, we understand the patients and doctors and what it takes to make a sustainable profit. Let’s try to find more innovative ways for all parties to make medicine accessible and profitable for all parties.’
‘What we do is by no means charity. I recognize that the pharmaceutical industry needs to make profits and develop new therapies. Not every medicine can be cheap, but there should be many considerations to make the process as efficient and affordable as possible.’
There needs to be more than a solid business case
‘Of course, it isn’t always easy to set up a business to create affordable medicine. Goodwill isn’t always enough for agreements. A deal isn’t a deal until it’s signed. Also, financing can take much longer than you originally hoped. Therefore, expectation management is one of the most important things as an entrepreneur, especially if many stakeholders count on you. I learned this by just diving in and doing it. However, it did present me with many challenges. For my next projects, I will ensure we have solidified funding. One of the reasons is that I have a growing team: people depend on Orfenix for their salary, which changes your risk profile.’
Three takeaways for future founders
1. Find mentors
‘Find mentors and learn as much as you can from them. If you don’t know the answer, there is always someone out there who knows the answer, and many people have gone through the same struggle. Especially at the beginning of your entrepreneurial journey, having a good mentor is vital.’
2. Social impact
‘More companies and investors are willing to do things for a fair price than you think. So it’s not all about money. You need a solid business case, but social impact is becoming increasingly important. If you don’t ask, you don’t know.’
3. Ask good questions
‘Though some say there are no bad questions, people with knowledge always like it when you ask them a good question. So, please do your homework on this person and their field of expertise. You will find that even the most inaccessible people will be available for a really good question in their field. In the end, everyone likes to talk about their expertise.’
