Written by Jo Wallis, Head of Commercialisation and Pricing Strategy
The recent developments put forward by the Medicines and Healthcare products Regulatory Agency (MHRA) and the Food and Drug Administration (FDA) to streamline the regulatory approval processes for therapies to treat rare and ultra rare diseases sends a positive signal to patients and developers alike. The MHRA rare disease policy proposals commit to ‘a forward-looking framework that meets the needs of patients with rare diseases, while ensuring robust evidence generation, system sustainability, and international convergence. The FDA’s plausible mechanism framework proposes a smoother approval pathway for individualised therapies treating ultra rare conditions, where randomised controlled trials (RCTs) are not feasible. This is good news for patients as well as companies developing innovative treatments for rare and ultra rare diseases. It is important to keep sight of the wider access perspective and understand the commercial realities of the environment such therapies will be launched into.
During early development, the ‘launch’ can seem a long way off, with market access considerations often undertaken after regulatory strategies are finalised. To an extent, this is understandable, high uncertainties and low risk appetites focus on securing regulatory approval first, without a full characterisation of the commercial and access pathways the product will be launched into until later. The effort to get to the start line seems huge. Meanwhile, patients with rare/ultra rare diseases and their families continue to face unacceptable delays to diagnosis and access to life changing treatments.
For rare and ultra rare therapies, it is critical for the market access perspectives to be considered in parallel with the regulatory strategy to avoid any late stage surprises and delays. The access and reimbursement pathways for rare and ultra rare diseases continue to pose a challenge for developers to navigate, these are well defined, including low patient numbers in trials, biomarker surrogate endpoints, extrapolation of treatment effects, and unique evidence packages. This raises questions such as:
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- Who will pay for the innovation?
- How will the new therapy be paid for?
- Which commercial mechanisms should be explored early to ensure the full value of the innovation is realised, and patients have access to new and life changing treatments?
- What is the impact of potential expedited regulatory approval on the reimbursement process?
Often, these questions are addressed too late in the development pathway, when the stakes and expectations are high, and resources limited. The practical issues of how the therapies will reach patients need to be surfaced, discussed, and worked through in advance, in partnership with relevant clinicians, patients and their families, regulators, and health technology assessment (HTA) reimbursement bodies to ensure every barrier to patient access is removed.
The recently announced National Institute for Health and Care Excellence (NICE) – MHRA aligned pathway and integrated advice service is a clear example of how regulators and HTA authorities can work together to support companies with their early development plans.
The right support early in development can help companies align limited resource around the highest priority activities, working towards a shared goal of faster patient access to therapies for rare and ultra rare diseases.
If you are interested in finding out more about our early commercialisation and access services for rare and ultra rare diseases, please contact Jo Wallis at Source Health Economics, a HEOR consultancy specialising in evidence generation, health economics, communication, pricing and commercialisation
