Orphan Drugs: The Importance of Real-World Evidence

Current expert thinking on how real-world evidence can refine orphan drug research, demonstrate stakeholder value and prove clinical effectiveness.

Alarmed at the number of often highly-priced orphan drugs coming to market, regulators, HTAs and payers are demanding a higher level of evidence for clinical utility and value. With limited clinical research data available, companies are increasingly looking to persuasive real-world evidence (RWE) to bolster their product propositions. But what RWE do stakeholders want? At what stage in a products lifecycle is RWE critical, and why? How do patient groups play into RWE generation? How can RWE be used to support physicians/HCPs in making treatment decisions?

In Orphan Drugs: The Importance of Real-World Evidence experienced executives from leading orphan drug players such as Takeda, Janssen, and Zelluna Immunotherapy assess the current RWE trends in the rare disease sector and identify the stakeholder, clinical research and technological factors that are driving regulatory approval, positive HTA assessment, physician uptake and patient satisfaction.

Orphan drug experts tackle key RWE questions:

• What are the strengths and weaknesses of current RWE collection and analysis models?
• What are the main sources for RWE and how do companies ensure the credibility of data?
• What is the impact of advances in data analytics, machine learning and artificial intelligence on RWE generation?
• What in-house capabilities are important to ensure the right types of data are collected and analysed to meet objectives?
• How can RWE help pharma to better understand patient populations and improve outcomes?
• What are the benefits of partnering with patient advocacy groups at an early stage?
• How is RWE used to support orphan drug regulatory approvals?

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What our experts say…

"In today's environment, having an approved drug doesn't mean you're going to get access for those drugs. There's a lot of payer as well as physician scrutiny coming down on rare diseases, especially when it comes to pricing and their value profile. Any company that wants to position its rare disease portfolio successfully needs to understand the value components that drive these stakeholder needs, and real-world evidence is the key to that. Real-world evidence helps you understand what is happening with the patients, how the patients are connecting to the physicians and given the strength of the databases these days, it gives you a very positive and powerful picture in terms of the payer positioning and how you would be able to address market access and health technology assessment considerations."
Usman Iqbal, Chief Medical Officer

"In my experience if you engage with payers, they will provide guidance. Unfortunately, some of them are stuck in very rigid technology evaluation processes, which are not necessarily adapted very well to orphan drugs, particularly at the rarer end of the spectrum, but they will engage. I think engaging with payers about what will be appropriate, particularly longer-term real-world evidence measures that will support the value of a product, and when would you be expected to supply them and so on, is feasible. Payers are generally not unrealistic about these things and about the industry. The payers are being clearer. It can't be a nice place to be the payers who are being seen to deny patients access to the use of 'wonder drugs'. But these things always work better if people try to work in concert, rather than being confrontational."
Damian Marron, Non-Executive Director and Corporate Advisor

"You should start looking at those data when you think about starting the development of a new drug. So, if you do a Phase I study, you should ideally have looked at what is out there. For example, in terms of our registries, which describe the natural history of disease, the epidemiology, and potentially treatment patterns. Ideally, I'd say you do this in Phase I. In Phase II you start collecting safety data, but also you would start looking at efficacy and make sure you recruit the right patient profiles, so ideally, you do it in Phase I when you prepare for Phase II."
Head of Market Access and External Affairs, Rare Diseases Division of Top 30 Pharma Company, Europe

"What I think is being required more and more is comparative real-world efficacy data between competitive treatment decisions. It's not a direct head-to-head, but it's a bit like head-to-head but done in a real-world context. For many diseases there are multiple treatment options possible and there are registries for those, so you can compare. Of course, the patient characteristics between those groups are not always fully identical, but it gives an idea of efficacy, length of treatment, discontinuation, those kinds of things. Quality of life is more difficult to look at, but those are comparable, and I hear more and more that payers want to see those kinds of data. And then there's also the difference between patients on treatment or without treatment, or the difference between a monotherapy and a combination therapy; that's also something that they payers like to see."
Global Marketing Director, Janssen, Europe

What to expect

• A detailed report revealing the insights of orphan drug experts for leveraging RWE in support of orphan drug development and commercialisation
• An examination of 13 key issues which orphan drug developers need to understand and respond to
• 40 targeted questions put to industry experts
• Their perceptive responses that provide 74 insights supported by 84 directly quoted comments

Expert payer contributors

This report harnesses the critical insights of senior pharma executives directly engaged in the collection and interpretation of RWE for orphan drugs. In some cases, their identities have been kept anonymous to ensure free and frank expression of views.

• Miguel Forte, Chief Executive Officer of Zelluna Immunotherapy and currently Chief Commercialisation Officer and Chair of the Commercialisation Committee, International Society of Cellular Therapy. Miguel has extensive expertise in the regenerative medicine and cell therapy industries, ranging from leading early and late stage clinical trials to market authorisation and the launch of new biologic products for various indications. Held various senior positions at the European Medicines Agency, Bristol-Myers Squibb, Abbott and Wellcome Laboratories (now part of GSK), International Medical, Nabi Pharmaceuticals and UCB.
• Claudio Marelli, Global Medical Lead, Medical Affairs, Takeda Pharmaceuticals in Zug, Switzerland. His focus is on orphan drugs for rare diseases at different stages of development from Phase II to post-MAA. Claudio is an MD specialist in cardiology with a long-standing global expertise in the pharmaceutical industry. During his professional career, he has held senior positions in R&D at local and global levels. He has worked in complex matrix organisations, providing medical affairs support to pre-launch and post-approval drugs. Claudio is also conversant with the discovery and preclinical phases of drug development. As an inventor of patents for MRI-based diagnostic methods in heart failure, he has been honoured with Fellowship of the American College of Cardiology.
• Damian Marron is currently Non-Executive Chair of Board at PepGen Ltd, an OSI funded, Oxford University spin-out developing drugs from their innovative delivery platform, Non-Executive Director at Bone Therapeutics, a clinical stage, regenerative medicine company and Resolys Bio, a private, late pre-clinical US startup and Head of Biopharma with Treehill Partners, a pure-play healthcare advisory boutique. Damian is an experienced life sciences executive with successful track record leading public and private biotechnology companies. He is a four-time CEO, including two companies acquired (Trophos by Roche; Agalimmune by BioLine Rx), and one IPO (TxCell, EuroNext). He has a strong track record of value creation through public and VC financing, deal making, strategic and portfolio planning and company development.
• Usman Iqbal is a medical executive with a 15 years portfolio career spanning R&D, medical affairs and end-to-end evidence and value development, across both large and small cap biopharmaceuticals. His experience spans a number of therapeutic areas including neuroscience, as the Senior Medical Affairs Leader at AstraZeneca Innovative Medicines Group, and oncology, as former Head of Sanofi Oncology, Global Evidence & Value Development. Usman's area of expertise also includes harnessing big data science, real-world evidence, advanced analytics and digital medicine to optimise R&D portfolios, trial designs, medical affairs delivery, and patient outcomes.
• Emma Harvey, Global head of medical affairs at F2G Ltd, a UK biotech focussed on rare fungal diseases, as well as a medical affairs consultant with expertise in rare diseases (including metabolic bone disease, lysosomal storage disorders and Duchenne muscular dystrophy), specialised medicines and infectious diseases. Formerly Senior Medical Director at Alexion Pharma, UK, Emma's experience includes managing pre- and post- launch strategy, lifecycle management at national, regional and global level. She has significant experience with NICE Highly Specialised Technology Appraisals including optimisation of clinical dossiers and the creation of managed access agreements.
• Global Marketing Director, Janssen (Johnson & Johnson) based in Europe.
• Head of Market Access and External Affairs, rare diseases and oncology specialist, Europe.
• Senior Vice President, Development, specialty subsidiary of a top 10 pharma company, US.

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