Author: Flóra Raffai-Executive Director at Findacure
The Value of Patient Group Engagement in Drug Development

The Value of Patient Group Engagement in Drug Development

The Value of Patient Group Engagement in Drug Development Date:11 October 2016
In recent years, there has been a growing movement among researchers and the pharmaceutical industry to involve patient groups in their work developing novel treatments, especially in the field of rare diseases. But many query the value of doing so, questioning what patient groups can add to the drug development process. The answer is, a lot.
Patient groups can be vital partners in drug development as they can ensure treatments are appropriate to patients’ needs. Patient groups are, in essence, a community of patients, their families, and their carers. Through frequent and personal contact, they are best placed to understand the needs and priorities of this community. They appreciate patients as people with lives beyond their condition, not just consumers of medical products. As a result, they can survey and build a wider picture of their patients, and more specifically, work with them to create a list of priorities for medical research. By working with patient groups before investing in an expensive R&D process, researchers and the industry can ensure they do not waste money developing treatments patients do not want.
A stark example of the value of consulting with patients can be found in achondroplasia (ACH). ACH is a rare genetic bone growth disorder which causes short limb dwarfism, macrocephaly, apnoea, and recurrent ear infections. Traditionally, research has focused on short stature, with treatments being developed to increase height in children affected by ACH. However, when this research was presented to the patient community, they found this focus on stature highly inappropriate. For the patients and their families, their dwarfism was part of their identity, it was not something that needed to be ‘cured’. Instead, the community requested a larger focus on the neurological impairment associated with the condition, and other symptoms which affect their daily lives. The treatment developed was not one the patient community wanted or would be interested in using.
Beyond directing research based on patient priorities, patient groups can provide funding to ensure research takes place. Small biotechs with new technologies or therapy ideas are often seen as too high risk investments for most venture capitalists, especially as rare diseases are not seen as financially attractive due to small population sizes. As a result, founders find it difficult to even raise enough capital to set up the company and cover core costs. Patient groups can, and have, changed this dynamic.
For example, in 2014, three charities (Duchenne Children’s Trust, Duchenne Research Fund, and Joining Jack) collectively invested $5 million to establish Solid GT, a gene therapy platform focused on developing novel therapeutics for Duchenne muscular dystrophy. Over the course of 12 months, this initial investment was leveraged to raise an additional $42.5 million from industry investors, one of the most successful rounds of financing in private biotech. Therefore, patient groups can fund necessary high risk research, basic and translational, to address critical gaps, de-risk the research and leverage millions.
Furthermore, patient groups can act as an invaluable partner during clinical research. Firstly, due to the wide patient network patient groups have established, they are well positioned to set up registries and other natural history studies. Indeed, an increasing number of rare disease patient groups are setting up their own independent registries, thanks in part to the rise of free/cheap registry software. These studies can reveal unknown patterns in disease progression and links between genetic mutations and phenotypic presentation of conditions.
This extensive patient network also means patient groups can be a critical partner in recruiting for trials. Patient groups are often in contact with a large portion of the patient population in a given country or region, and therefore can quickly reach out to potential trial participants, speeding up recruitment. For instance, the AKU Society led all patient recruitment for the DevelopAKUre trial, recruiting almost half of all eligible adults in Europe in just nine months.
On the point of assisting with clinical trials, patient groups can use their experience of the condition’s impact on patient lives to ensure trials are designed with patient needs in mind, reducing the burden on patients to participate. For example, patients with mobility issues can schedule Skype consultations (where appropriate) instead of travelling hours to trial sites. This minimises the health costs of lengthy travel, reduces time missed from work (for both patients and their carers), and lessens the impact of the trial on the day-to-day life of the participant. This can increase recruitment rates and reduce drop-out rates.
Finally, patient groups can also increase retention in trials through effective communication and dissemination of the research’s progress and results among their patient community. By seeing the fruits of the labour of being involved in a trial, participants are more likely to remain in trials as they feel part of something bigger than themselves, and increases the likelihood other members of the patient community will feel inclined to become involved in research in the future.
In sum, it is clear that patient groups have an important role to play in developing novel treatments for rare diseases. But the question arises, are patient groups prepared to take on this role? Especially in the rare disease field, most patient groups are ‘kitchen table’ organisations, set up by either patients themselves or family members. They usually are volunteer-run, with limited time, experience, and resources. As a result, they may not necessarily have the knowledge needed to take on this valuable partner role, or even be aware of the possibilities open to them in supporting research. Due to this, it is critical patient group empowerment projects exist to support patient groups to achieve their full potential.
Fortunately, several such projects already exist. EUPATI and the AMRC have guidance available through their websites for all patient groups, not limited to rare diseases. These can provide patient groups with background knowledge of medical research and the processes needed to establish effective research partnerships. Specific to rare diseases, EURORDIS organise an annual summer school for European patient advocates to deepen their understanding of medicinal development, equal access to treatments, and regulation around orphan products. Findacure also offer UK-specific training for patient advocates and groups in the form of training workshops, webinars, peer mentoring, and an online resource portal. In order to ensure patient groups can take on their critical role in drug development, it is crucial such projects are supported and allowed to continue to empower groups to take on the mantle of research partners.