A rare partnership: Collaborating with patient advocacy organisations for rare disease registries

When facing the challenges of developing a treatment for a rare disease, it can be helpful for sponsors to partner with groups, such as patient advocacy organisations (PAOs), that share similar goals. PAOs are non-profit organisations that aim to improve the lives of those impacted by a given disease, with missions that can range from providing social support to influencing public policymakers — or supporting clinical research. The value of collaborating with PAOs in research has become increasingly recognised among those working to develop treatments for rare diseases.

One of the areas in which it can be particularly beneficial for sponsors in the rare disease space to partner with a PAO is in creating a patient registry. Here, we’ll explore some of the benefits of creating such a registry, as well as considerations for partnering with a PAO.

Advantages of a rare disease registry

Creating a rare disease registry can serve a number of purposes. Because there are limited numbers of patients with a given rare disease, information and data on rare diseases are often incomplete or inadequate. Patient registries, which are organised systems to collect information on a given disease, can help alleviate these difficulties. For example, registries can be used to gather data for a natural history study to gain a better understanding of the progression of a disease.

A registry can also be used as an external control arm for clinical research. This is of particular interest to patients with rare diseases, as it can help avoid the use of a placebo group in clinical trials. As a number of rare diseases do not have many options for effective treatment, participation in a clinical trial offers a source of hope for some patients — but the possibility of being included in an untreated arm may discourage them from participation.¹ Using a registry to reduce the need for placebo comparison and increase the likelihood that a patient will receive the treatment may also increase their willingness to take part in a trial.

Another challenge faced in rare disease clinical studies is the difficulty finding eligible patients, given the smaller numbers of people living with a given rare disease, as well as the fact that it may be harder to identify them through electronic health records. In some cases, a registry can offer a way to find potential trial participants.

There are many other ways in which rare disease registries can be beneficial to sponsors and PAOs. Some of these include informing trial design, generating real-world data after patients have participated in a trial, and identifying diagnostic and treatment pathways to help establish best practices among physicians.

Working with a PAO

Partnering with a PAO is different from a typical business arrangement that sponsors may be familiar with. Instead of a simple business transaction, engaging in a lasting, meaningful relationship with a PAO can help to build trust with the patient community, and create lines of communication to ensure that the needs of both parties are being met. In fact, if a robust relationship is formed, the PAO can offer invaluable input beyond the patient registry alone, such as insight regarding clinical trial design that can improve recruitment and retention.

Additionally, PAOs are often run by volunteers, who in many cases are patients or caregivers of those who are affected by a disease. These individuals may be under a great deal of stress, and may not have experience in undertakings such as building a registry or working with large for-profit organisations and clinical sites. As a result, working together may require education, patience and understanding.

More specific to collaboration on registries, there may be complications in regard to data ownership. In most cases, the sponsor cannot claim sole ownership to data from a registry created in partnership with a PAO — as might otherwise be possible if the sponsor set up its own registry. Instead, it is typical for a sponsor to have exclusive rights to that data for only a set period of time before the registry becomes a community resource. However, sponsors and PAOs can be innovative in how they structure their partnership, and consider non-traditional approaches.

A complex endeavour

Collaborations between rare disease PAOs and sponsors to create registries have great potential to further the shared goals of both parties. While such partnerships may seem to be simple undertakings, there are many facets to take into consideration, such as navigating the complexities of a relationship with a PAO, choosing the correct technology and future-proofing the registry’s design. A successful rare disease registry requires careful planning and thoughtful relationship building.

References:

1. C. M. W. Gaasterland,  M. C. Jansen – van der Weide, et al., The patient’s view on rare disease trial design – a qualitative study. Orphanet Journal of Rare Diseases. Published February 07, 2019. Accessed November 14, 2022. https://ojrd.biomedcentral.com/articles/10.1186/s13023-019-1002-z#Sec6