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Transforming access in rare diseases

A quiet revolution is underway that could transform access to medicines in rare diseases, according to Duchenne UK, the charity at the heart of a new project to streamline evidence-gathering in HTA


Health Technology Assessment (HTA) is an essential component of the introduction and reimbursement of new medicines - not just in the United Kingdom (with bodies such as NICE, the SMC and AWMSG) - but in many countries across the world. Within a single country, the landscape can be complex, with up to 15 different possible routes to reimbursement in the UK alone.  

From IQWiG in Germany to the HAS in France and the Pharmaceutical Benefits Advisory Committee in Australia, the reality we face is that all technology assessment organisations work in slightly different ways, sometimes reaching different conclusions about the same treatment. And that’s just not helpful to patients - particularly those with life-limiting conditions.

At the same time, innovation in medicines for rare diseases is gathering pace. In Duchenne muscular dystrophy (DMD), for example, treatments from gene therapy to stem cell transplantation are all on the horizon, and all will need to be assessed by country healthcare systems to determine whether their cost will be reimbursed.

Inevitably this will involve developing robust data packages - including systematic literature reviews, quality of life measurement, burden of disease, economic modelling and network meta-analyses - a process that incurs a significant cost in both time and money. While larger or experienced pharmaceutical companies are often able to commit considerable resources to developing the evidence needed for the HTA process, this can pose a considerable challenge for smaller, less well-resourced organisations.

This siloed approach also risks producing an inconsistent evidence base. Uncertainty in the data means that HTA bodies may not be able to make confident decisions on a treatment, resulting in further rounds of queries and additional discussions or meetings to reach a conclusion. These delays may be frustrating to the companies and administrators involved, but can be a matter of life and death to the patients waiting to access the treatments.

At Duchenne UK, we wanted to see if we could find a way to support prompt access to medicines, without compromising on the integrity of the evidence and working with the reimbursement agencies frameworks. And we think we have found a way that works, not just in the UK but in other countries too.

Project HERCULES is a unique initiative set to radically improve the way evidence is generated for HTA submissions. Launched by Duchenne UK, HERCULES will be the first time a group of pharmaceutical companies has come together to generate, share and align disease-level data across an entire condition.

Relatively small patient populations in rare conditions means that disease-specific information gathered for HTAs is likely to come from the same group of patients. This means that pharmaceutical companies, particularly the smaller, less resourced ones, are all going through a very costly process of reinventing the wheel. Collaborating on disease level evidence will have benefits which will vary from improved modelling (with less uncertainty), a broader evidence base, reduction of generating evidence which is duplicative or inconsistent, improved engagement with patients, lower costs and management time, and making better use of patient input. While we will not expect competitors to share their hard-earned treatment data, information on placebo arms of trials could prove invaluable. Comprehensively collecting the data only once will reduce the burden on patients and families caused by returning time and again for their input.

Phase one of HERCULES is working to provide a model of the entire disease pathway in DMD, including assessing the economic cost and burden of Duchenne muscular dystrophy, researching the natural history of the disease and developing a new quality of life metric, which is essential in cost-utility based HTA processes. While this type of collaborative evidence base may take longer to achieve, it means that we will arrive at a much more robust model of the disease, rather than one that is specific to a single treatment. With HERCULES, we can set the standard for an entire disease area.

Collaborative evidence gathering though HERCULES gives us the opportunity to work with patient groups in a more collaborative, less transactional way. We can begin to fully understand their lived experiences and properly articulate the true value of a treatment. In DMD, a standard outcome measure is the six-minute walk test, but what are the benefits of being able to stand and walk that little bit longer? It may be that parents feel confident to leave their son alone without fear of him falling, or a teenage boy retains his independence for a bit longer. We need to assess the true value of outcomes like these to patients, families, health and social services.

Initiated by the charity Duchenne UK, the approach proposed by HERCULES is likely to offer most benefit for rare diseases. Here, companies are often faced with an incomplete understanding of the patient pathway and data is scarce. With companies working on wildly different treatment approaches, compiling their disease-level information will produce a much broader evidence base that can be held to a higher degree of scrutiny.

Project HERCULES helps to address a long-standing problem faced by pharmaceutical companies by streamlining evidence gathering processes and reducing inconsistencies and uncertainties in the evidence base. At the heart of HERCULES is the desire to improve outcomes for all involved in medicines development and approvals, especially for the children living with Duchenne muscular dystrophy who are waiting for potentially life-changing treatments.

Article by
Emily Crossley

is co-founder and CEO of Duchenne UK

11th January 2018

From: Healthcare



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