Gauchers Association

Gauchers Association is a charity which provides support and information to families affected by Gauchers disease, an inherited lysosomal storage disease which can be life-threatening. As part of their patient advocacy efforts, Gauchers Association encourages and supports the availability of new drugs to treat the disease. When the Association was founded, there was only one drug for Gauchers disease in development. In recent years, this has increased to five treatments which for a rare disease is a crowded market! Maintaining independence and dealing with multiple companies has therefore been key to the organisation’s success.

Maintaining Independence

Gauchers Association’s independence has been important for promoting confidence in their organisation and maintaining trust with other stakeholders. A lack of trust can create communication difficulties and be a barrier to collaborating with other stakeholders. To achieve this independence, Gauchers Association only takes funding from industry for very specific and defined projects.

Dealing with Multiple Companies

With several different companies now developing or marketing drugs for Gauchers disease, there are some key challenges that have had to be navigated such as:

• Resources: Choosing which meetings to attend, in terms of time and travel costs, as well as deciding where it is most important for the organisation’s voice to be heard.
• Crowded Market: Working in a crowded market means that there are additional legal matters to navigate. Gauchers Association now enters into confidentiality agreements with the companies they work with, which can limit the openness of discussions.

Key Successes

Over the years, Gauchers Association has successfully advocated for patients in industry in many ways such as:

• Working to develop an access programme with drug manufacturer Genzyme, to provide access to treatment for patients in Eastern European countries.
• Finding a solution to a drug shortage caused by manufacturing problems. Along with other patient groups, Gauchers Association arranged a meeting with the CEOs of manufacturers after it was found that a problem at a manufacturing plant would limit the supply of a drug. The groups successfully persuaded the company to allow patients in need to use a stockpile of the drug that was going to be used for a clinical trial. This delayed the trial but made sure patients didn’t have to stop treatment, which for some would have been life-threatening.
• Gauchers Association is now participating in the development of a gene therapy for the treatment of Gauchers disease. Gauchers Association has successfully gained Orphan Drug Designation for their gene therapy and is working with industry to make the treatment a reality.

Gauchers Association has also collaborated with industry in clinical trial recruitment, research design, educating staff, lobbying Government and representing the patient community on steering groups and panels.

Niemann-Pick UK (NPUK) and IntraBio

Niemann-Pick disease is an inherited, neurological, progressive disorder which occurs at a young age and can lead to a comparatively short life expectancy. NPUK is a small charity dedicated to making a difference to patients living with Niemann-Pick disease in the UK.

IntraBio is a biopharmaceutical company based in the US. They focus on developing novel treatments for rare and common neurological disorders.

The partnership between NPUK and IntraBio began following the publication of a paper about a drug candidate for Niemann-Pick disease by a group who went on to form IntraBio. Upon seeing this article, NPUK were impressed with the many potential benefits of the drug and wanted to explore the possibility of the drug being made accessible to patients in the UK. However, there were still questions remaining about how the drug could be accessible, its price and effectiveness. NPUK reached out to IntraBio to explore opportunities for taking the drug forwards; this led to the start of a successful collaboration.

IntraBio treats the patient advocated as key stakeholders and has always been very active in seeking patient group insights into every aspect of clinical trial design, including patient recruitment, what is important to patients and gaining advice. This has led to the design of an innovative clinical trial protocol for the drug in collaboration with NPUK and other patient groups.

NPUK’s Key Insights from Working with IntraBio

• Reach out: Interacting with IntraBio has allowed a great partnership to develop; it wouldn’t have happened if NPUK hadn’t actively reached out to IntraBio.
• Develop relationships: Develop relationships with key people in the organisation you work with.
• Respect the people you are working with: Just because your contact works in industry it doesn’t mean they don’t understand your experiences.
• Understand your value: People are often more willing to give their insights to patient groups than to a pharmaceutical company and may trust your opinion and judgement more.
• Be clear about who you speak for: Be clear about when you are speaking on behalf of your patient group, a subset of the patient community or voicing your personal experiences or opinions. Remember that a patient group is different from a company and you will be rarely speaking on behalf of the whole patient community.
• Patients should be valued: Don’t be afraid to speak up and express your opinions and concerns.
• ABPI Code of Practice is in place to protect patients, it and should not be considered as a barrier to working together.