Francis Pang is Vice President Global Market Access at Orchard Therapeutics and was a speaker at the last ATN event on reimbursement on 17 September. Here he talks about the unique challenges for advanced therapies in this area and how dialogue is central to finding solutions.

The next Advanced Therapies Network event on reimbursement will be in partnership with Phacilitate Leaders Europe conference at the Royal Lancaster, London. Francis will join speakers from NHS England, NICE and the Cell & Gene Therapy Catapult. 

Q. Congratulations on joining Orchard. Your position as vice president of global market access is a newly created role, what have been your first steps in developing global pricing and market access strategies?

A. Thank you! I am truly privileged to have joined a company with such an advanced and robust pipeline of potentially curative medicines based on a strong heritage of over 20 years of academic clinical research. We are at an exciting juncture as a company, with three regulatory filings expected by the end of 2021. As such, my initial priorities have focused on establishing Orchard’s pricing and market access governance and thinking through the framework and approaches for the value demonstration of these extraordinary medicines. At the product level, I have built upon a solid foundation of work already underway to generate the necessary data and insights for illustrating the full and multidimensional value of the transformational benefits of the assets we are seeking to bring to market.

Q. Your experience spans the pharma and biotech industry, what would you say are the unique challenges of advanced therapies market access and reimbursement compared to the wider industry?

A. I am really fortunate to have been able to tackle market access issues for a number of different modalities at the very outset, such as the anti-TNFs; various multiple sclerosis, diabetes and oncology medicines; nutritional supplements and therapies for rare diseases, leveraging my experience from academia, the pharmaceutical industry and the health technology organisation side. I believe my academic background in genetics, health economics and business administration (a combination of skills achieved more by accident than by design!) equips me well for helping address the unique challenges related to gene therapies.

Ex vivo, autologous hematopoietic stem cell gene therapy is fundamentally different from other products that have gone through the formal drug development process. Instead of chronic treatments that address symptoms or slow disease progression, we are developing therapies with the potential to collapse a lifetime of benefit into a single intervention by correcting the underlying genetic defect in some of the most devastating, life-threatening genetic diseases.

Currently, structures within many healthcare reimbursement systems are not set up to appreciate and accommodate one-time, potentially curative types of medicines that we, and others, are seeking to bring to market for patients.

As a result, a great deal of work is underway to explore and test new ideas that may help evolve access and/or reimbursement structures and methodologies to help ensure patients have access to these transformative therapies, if approved.

We are collaborating with multi-stakeholder forums on both sides of the Atlantic – including MIT’s New Drug Development Paradigms (NEWDIGS) Financing and Reimbursement of Cures in the US (FoCUS) Project, the EURODIS’ Rare Impact Project as well as gene and cell therapy initiatives organised by the Alliance for Regenerative Medicine (ARM) and the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) – to help advance these critical efforts.

Q. There is a lot of debate and discussion around different reimbursement models, what are your thoughts on what type of approach would provide a realistic solution for Orchard’s gene therapy products?

A. We recognise that payers’ circumstances and needs can vary by geography, populations served and even by disease states and treatments. There’s no one-size-fits all approach. Mutual flexibility will be essential, and we are open to a range of reimbursement and payment options for each of our therapies, if approved.

Q. It’s been highlighted that early dialogue between industry and Health Technology Assessment (HTA) bodies is important to establish the right reimbursement approach. How much have you been involved in these types of discussions or how much are you planning to be involved?

A. I agree that it is very important to talk with HTA bodies as early and often as appropriate, and we plan to take a high-touch approach to engagement with them. We must help all stakeholders, including HTA organisations, understand and recognise the holistic scope of the benefits that these therapies, if approved, have the potential to deliver to children, families, communities, the health system and society. In that, we have been making strides by gathering scientific advice from HTA bodies in order for us to ensure that their evidence expectations are fulfilled for future pricing and reimbursement discussions.

Q. In your view, is it possible to successfully commercialise hospital-based gene-therapy treatments for rare diseases?​

A. We absolutely believe that, in the interest of patients and society, the healthcare sector can sustainably deliver one-time, potentially curative therapies for devastating genetic diseases once they’ve been approved by regulatory bodies.

We’re in the midst of a major sea change at the intersection of science, medicine and economics, spurred by recent advances in the understanding of the human genome. Gene therapies, such as the ones we are developing, have the potential to make a critical contribution to the future of medicine and we believe that the demand for and access to such therapies, if approved, will grow substantially as society understands the full benefit that one-time, potentially curative medicines could bring to patients, families, communities, health care systems, and society as a whole.

Francis Pang will be speaking at the next Advanced Therapies Network event on reimbursement on 17 September 2019. The event will be in partnership with Phacilitate Leaders Europe conference at the Royal Lancaster, Bayswater, London. Francis will join speakers from NHS England, the Cell & Gene Therapy Catapult and NICE. The event is free to ATN members and you can become a member of the network here.