Tuberous Sclerosis Complex (TSC) is a rare multi-system genetic disorder in which tumours can grow in any organ of the body but most commonly the brain, skin, heart, lungs and kidneys. TSC can also cause epilepsy, developmental delay, kidney disease and other health complications.

Tuberous Sclerosis Australia (TSA), which was founded in 1981, is the only not-for-profit organisation supporting families living with TSC in Australia and New Zealand. It is a small consumer health organisation supported by volunteers.

The Working Together Guide in action

In 2010, Novartis Oncology was in the process of developing the first medicine for TSC when it approached TSA, which had never collaborated with a medicines company.

From the first meeting, the Working Together Guide was used to create the framework supporting the partnership between TSA and Novartis.

A successful collaboration

TSA has held several educational events for families living with TSC. These were partially supported by grants from Novartis. Another grant from Novartis helped fund a large project that vastly improved the technology capabilities of TSA.

TSA reviewed information Novartis produced for clinicians to better reflect the patient experience. TSA and Novartis held regular meetings to share knowledge about developments in TSC. TSA also presented an independent submission to the Pharmaceutical Benefits Advisory Committee (PBAC), which supported the listing of a medicine for TSC sponsored by Novartis.

Addressing key issues and challenges

The TSA volunteer committee created a policy document for working with medicines companies. This process helped TSA identify the key issues and challenges in collaborating with a pharmaceutical company.

For example, a major consideration for a small organisation such as TSA is sustainability: how to ensure that the organisation does not become reliant on funding that may not be available in the future. To address this, TSA decided to not accept funding from the pharmaceutical company for ongoing expenses such as regular publications or patient support staff, but to accept grants from Novartis for capability improvements such as implementation of new technologies that will enable future growth of the organisation.

Respect for independence

A small organisation with limited capacity, TSA wanted to ensure that a grant from Novartis didn’t shift its priorities. TSA made the decision to seek financial assistance from Novartis only for projects nominated as priorities in its strategic plan.

Additional benefits

As a consequence of regular meetings between TSA volunteers and Novartis staff, each partner has an increased understanding of the other. Novartis has more appreciation of the challenges of living with TSC, including symptoms not previously targeted by its medicine. TSA volunteers are now much more familiar with the Therapeutic Goods Administration (TGA) and Pharmaceutical Benefits Scheme (PBS) processes, and are able to update families on the availability of new medicines for TSC.

Areas for improvement

The collaboration began with a Novartis research project conducted by a third party, which needed to contact TSA for help identifying potential participants in the project.

The third party refused to disclose that it had been commissioned by Novartis, an unfortunate oversight. As TSA volunteers knew that Novartis was the only company trialling a medicine for TSC, the secrecy was unwarranted. It could have been avoided with standard confidentiality practices.

The establishment of a patient registry for TSC was also challenging, with TSA raising concerns that it had not been involved in the project’s planning or governance. There were two reasons for this. The project was driven by the Novartis global team, so it required some adaptation to suit the local circumstances. Meanwhile, Novartis’ Australian medical team, which was involved in the project, underwent several staff changes, and new staff took time to get up to speed with the company’s relationship with TSA.

For its part, TSA struggled to follow one of its policy rulings: to seek funding from sources other than medicines companies also. TSA revised its plans and delayed some projects, allowing other funding sources to be explored.