Accessing medicines for rare diseases workshop

Our last workshop of 2017 helped patients groups better understand the regulatory processes around medicine access.

Treatments for rare diseases are few and far between, and when they do exist, patients – understandably – want access to them. However, accessing medicines is no easy process and patients often face endless obstacles in getting drugs licensed and approved for reimbursement in the UK. With this in mind, on Wednesday 22nd November, we held our final workshop of 2017 on the topic of access to medicines. Scroll down to read summaries of the talks and watch their recordings.

Josie Godfrey - Director, JG Zebra Consulting

Our first presentation of the day was delivered by Josie Godfrey, Director of JG Zebra Consulting. Josie spoke about the challenges in accessing medicines for rare disease patients and, having previously worked at NICE, was able to provide great insight into the current route to access in the UK. She highlighted the different organisations involved, including regulators, health technology assessment agencies, and the NHS, as well as the different questions they have when making decisions about access, such as whether a treatment works and is safe to use, whether it’s effective, and whether it’s good value and affordable. Josie also spoke about some of the reasons why making a decision about access is so difficult, including the problematic method of assessing drugs for rare diseases on a system designed for common diseases, as well as the ethical considerations at play. Josie finished by addressing what patients can do to help the process such as putting forward a strong case on the impact of their condition and the difference a particular treatment will make.

Nick Meade - Director of Policy, Genetic Alliance UK

Up next was Nick Meade, Director of Policy at Genetic Alliance UK. Nick’s talk focused on how we fix the current “mess”, and what the future route to access could be. Earlier in the year, Genetic Alliance UK ran a call for evidence on the topic of access to medicines, which revealed the frustrations of rare disease patients in accessing treatments, particularly where there appears to be a drug that could benefit them, but which is unavailable within the UK. Nick spoke about the structural difficulties in the current access model, and the different systems used within the UK itself, as well as the underlying problems – touching again on the issue of using the same method to assess drugs for both common and rare conditions. Nick also highlighted some of the future challenges to access, such as gene therapy treatments, which would have high initial costs, but long term benefits both financially and medically. Finally, Nick discussed their vision for a solution to the access to medicines problem – resetting the model to create a system which is flexible to the needs of rare disease patients, transparent, effective, and quick to make decisions.

Lindsay Weaver - Chief Executive, Climb

Following a short break, we resumed with the first of our two patient group case studies. Lindsay Weaver, Chief Executive at Climb, shared their experience in gaining access to a treatment for Hypophosphatasia (HPP), an ultra-rare metabolic bone disorder. Climb’s journey took them through NICE’s Highly Specialised Technology Appraisal process and began in May 2015. Two years later, following several meetings, questionnaires, consultations and evidence submissions, they negotiated a Managed Access Agreement for the drug Strensiq. This ensures that, for a limited amount of time, those in most need of treatment receive it, and allows for further gathering of evidence on the drug’s benefit, in the hopes that it will be made available to the wider cohort of patients. Lindsay’s advice to patient groups was to make sure they are engaged with their community, work strategically and collaboratively with all stakeholders involved in the access process, and – importantly – to gather support from other patient organisations.

Vanessa Christie-Brown - Research Coordinator, SMA Trust

Our morning was rounded up by Vanessa Christie-Brown, Research Coordinator for SMA Trust and Europe Coordinator for SMA Europe. Over the last few years, SMA Trust have been involved with a pharmaceutical company, helping to licence a drug for Spinal Muscular Atrophy (SMA), to ensure that patients with the most need gain access to the treatment. They are now just weeks away from a NICE decision which will determine which access route will be taken for the treatment. Vanessa’s presentation focused on the role patients have played in this journey, particularly in addressing what they wanted from a treatment. Importantly, they overwhelmingly stated that they were less concerned about having a drug that would improve their condition, instead preferring something which would stabilise the disease and halt further degeneration.

After breaking for lunch, our speakers returned for the afternoon panel session, which included questions on how patient groups can maximise their involvement in the decision making process, how access can be improved, and the prospect of individual funding requests. Lastly, the workshop was concluded with our discussion session, which addressed some of the day’s major themes. In groups, our delegates examined the various points of access to rare disease medicines, including who is most likely to receive drugs through different routes and who controls and pays for the drug, how patient groups can help improve the process, and also their ideas for building a new and improved system.

Overall, the workshop was a great success, and we hope all of our attendees went away feeling engaged and informed about the access process. We would like to say a big thank you to our speakers for sharing their experiences and expertise, all of our delegates for attending, and White & Case for their excellent hospitality.

We would like to say thank you to our funders Amgen and Shire for providing financial support for this workshop. This workshop was solely organised by Findacure and our funders did not determine the content.