Patient Advocacy Groups as a Strategy for Orphan Drug Access in the UK

By Remy Denzler, Ph.D. (Senior Analyst) and Kelly E. Cockerill (Director)
Health Advances GmbH

A recent example illustrates how patient advocacy groups can have increased impact on market access for orphan drugs

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Summary

  • Although the first drug for Duchenne Muscular Dystrophy (DMD) received European Medicines Agency (EMA) approval in August 2014, it took nearly two years until the national health technology assessment (HTA) was completed and cleared the way for reimbursement.
  • In response to the lengthy approval process, the patient advocacy organization, Duchenne UK, initiated Project HERCULES (HEalth Research Collaboration United in Leading Evidence Synthesis) together with seven pharmaceutical companies, with the goal to support HTA processes and achieve faster access to novel, innovative treatments for DMD in the future.
  • Since patient advocacy groups are consulted as part of the National Institute for Health and Care Excellence (NICE) highly specialized technology (HST) evaluation, pharmaceutical companies commercializing orphan drugs in the UK benefit from assessing strategic partnership opportunities with patient advocacy groups in order to facilitate evidence collection and accelerate drug access.

Background
Orphan drugs may benefit from separate reimbursement evaluation pathways that seek to improve access to drugs that address high unmet needs. Demonstrating clear improvement in patient outcomes in small patient populations, where approved standard of care therapies are lacking, remains however a significant challenge. One popular topic of discussion in guidelines for assessing rare disease therapies, such as the recently published guidelines from a European multi-stakeholder working group called ORPH-VAL, is the importance of including the patient perspective into HTAs. The UK, in particular, has a formal process for incorporating the patient voice into its HTA process – as part of NICE HST evaluation, patient groups are given the opportunity to submit their opinion and sit on the advisory panel during the evaluation. The recent case of DMD illustrates how patient organizations may facilitate faster access – a trend expected to play an important role in future orphan drug HTAs.

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Figure 1: Overview of pharmaceutical companies forming Project HERCULES; this initiative was created by patient advocacy group, Duchenne UK

Duchenne Muscular Dystrophy (DMD)
In August 2014, the first small molecule drug for DMD (Translarna, PTC Therapeutics) received conditional EMA approval in Europe. The National Health Service (NHS) in the UKhowever refused to reimburse the drug until NICE completed the HTA. Although the patient advocacy group, Duchenne UK, was deeply involved in the HTA process by engaging patients to collect supporting data, it took nearly two years until NICE recommended the use of the drug.

Driven by the delayed reimbursement decision, Duchenne UK along with seven pharmaceutical companies, initiated Project HERCULES in November 2017. HERCULES aims to support HTA processes and enable smoother and faster payer reviews for innovative treatments in DMD in the future. Specifically, the consortium plans to: promote fast-track designation for DMD, develop economic model templates, standardize quality of life determination and other clinical trial endpoint analyses for DMD, and educate stakeholders during the HTA process.

Implications for Orphan Drug Manufacturers
The example of Duchenne UK demonstrates the important role that patient advocacy groups play in ensuring assessments incorporate the patient perspective as well as in enabling easier access to data required for HTA submissions. In addition, advocacy organizations enable pharmaceutical companies to engage with patients, offering a compliant platform to establish meaningful pharma-patient interactions.

Companies preparing for the commercialization of orphan drugs in the UK benefit from evaluating strategic partnership opportunities with patient advocacy groups to facilitate evidence collection and accelerate drug access.


About The Authors
The authors are members of Health Advances European Practice Area which helps clients navigate through the diversity of European healthcare systems to optimize commercialization strategies of pipeline and in-market products.

References
NHS, NICE, Duchenne UK

iUK and England are used interchangeably

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