Written by Tim Irfan
Business Lead, Rare Disease & Biotech
The World Orphan Drug Congress (WODC)
The World Orphan Drug Congress (WODC) is one of the largest of its kind
This year, it has attracted attendees and speakers from the industry (Alexion, UCB, Pfizer, Takeda, Sanofi, to name a few), from regulatory bodies such as the EMA and German ministry of health, as well as patient organizations and policymakers from the EU. The congress had a strong focus on research, development, and innovation of orphan drugs. A goal that everyone would like to achieve is to bring 1,000 new drugs for rare diseases to the market by 2030 – annually. Undoubtedly, there is momentum and interest from pharma companies to advance treatments for patients living with rare diseases. However, right now, 1,000 new drugs seem like a stretch goal with 251 submissions (175 positive COMP opinions) in 2021. On the bright side, around 20% of the submissions in 2021 were for new conditions. Thus, bringing hope to patients who previously had no approved treatments available. Among the key speakers and panelists, there was a consensus that this ambitious goal for 2030 is reachable. The technology exists, the experience is there, and the unmet need surely is huge. (Policymakers will be key in supporting interest in investing in orphan drugs). What is needed to reach this goal is collaboration.
Collaboration
Everybody, from industry to policymakers to patient organizations and regulatory bodies agreed that collaboration is the way forward to accelerate Research and Development (R&D) and stoke up innovation. As a prime example, Project HERCULES was given. Duchenne UK, UK’s leading Duchenne muscular dystrophy (DMD) charity, has generated and provided access to evidence needed for HTA dossiers. These data were utilized by patient organizations, clinicians, academics, leading pharmaceutical companies, Health Technology Assessment agencies, and other advisers. 1 This collaboration has led to a quicker and more streamlined approval process. And especially in a setting where the generation of viable data is difficult due to its scarcity, such collaboration is prone to accelerate the approval process. It is also great to see that pharma companies are willing to collaborate. Too often, they act in silos. And still, many companies are siloed within, not having strong connections between R&D and Commercial. Collaboration was the buzzword at WODC this year.
What does this mean for Commercial?
Obviously, this newly found collaboration is a great development to observe. It definitely makes the lofty goal mentioned above more reachable. However, this is collaboration on the R&D side only. And that brings me to the elephant in the room. How far can the collaboration go? Is there a willingness for pharma companies to collaborate beyond R&D and generate data needed for submissions? Is there a willingness to collaborate in the commercialization of the drugs once approved? I am posing this question because right now each company is on its own when it comes to the commercialization of its drugs. Each brand team works tirelessly to create a USP and carves out the slightest differentiation possible to become the brand of choice for the prescribing physician. On the receiving side, physicians are flooded with sales visits and information, each brand demonstrating how it is superior and best for a specific patient group. I doubt that decision-making in such an environment is accelerated. Considering the need for collaboration, we have to ask whether pharma companies’ current business model is outdated regarding rare diseases. Currently, collaborations exist in the form of co-marketing agreements. Usually these only divide the world into different territories while creating quite the overhead through added complexity. There is a constant need to align and get buy-in from all partners, which can easily slow down marketing activities and that might not be in the best interest of the patients.
Imagine how different a truly collaborative approach would be.
What if...
An approach where patients’ needs are at the center of all marketing activities. Where pharmaceutical professionals work with the HCPs to determine which treatments yield the best results for specific patients – irrespectively of the company behind them. How realistic is this going to be? OK, let’s shift our perspective away from the pharmaceutical company. Let’s imagine a single, global database that holds all health-related information for a patient. That database would be a true representation of the patient with records coming from GPs, specialists, and hospitals (private & public). All medication, past and present, all concomitant diseases, biomarkers, and doctor visits would be documented here. And the data structure would allow for analysis and insights creation. We could explore thousands of patient years in an instant and expand our knowledge of disease treatment and management significantly. It would be an incredibly powerful tool that could guide physicians’ decision-making when it comes to treatment choices and guide policymaking based on real-world evidence on a scale never seen before.
What will happen
Granted, while this image holds a huge promise it does also sound a little alarming and dystopian, having all health-related information in one place and accessible by someone else. The fact is, while we are far away from this unified database today, electronic health records, machine learning, and artificial intelligence are pointing in that direction already. And efforts are made to apply advanced technologies to analyze large sets of data to advance healthcare. Almost every larger pharmaceutical company has its own data lake and is working with its big data. And they are not alone. Giants like Google, Amazon, and others already offer their cloud based healthcare-focused solutions to other companies. There is a change coming and I am curious to see how pharma companies will adapt – and how quickly – once prescribers can receive guidance through these advanced analytics. Either, because the data can be utilized by them directly or the (inter)national indication guidelines prescribers are following anyways are influenced by these analytics.
Are you ready?
At the World Orphan Drug Conference, collaboration was the buzzword. Great examples of successful collaboration were shared that led to better patient care and outcomes. Right now, each pharma company only has a fraction of the truth. It is only a matter of time before a third party (Google, Amazon, etc.) can see the full picture while the big players in pharma struggle to see the forest for the trees. Only time will tell but this conference has given me hope for a future where we stop talking about unmet needs and try to address them siloed, and instead start addressing them quickly, evidence-based, and collaboratively. The first steps of collaboration have been made in R&D. The next steps will need to lead us into commercialization, and it will be an interesting journey to take.
Conducting market research in rare indications requires careful consideration across all stages of the process, engagement, methods, data collection, analysis & reporting. Our team is dedicated to providing remarkable clarity to marketing and general business questions at all stages of our engagements throughout a drug’s lifecycle. Our dedicated rare disease team of senior consultants would be happy to provide you with the answers you need to your business questions. Whether you are considering a licensing deal / M&A, developing a molecule in your pipeline, or if you are considering different lifecycle management strategies for an in-market product in a rare disease, we can provide the answers you are looking for.
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