Power to the patients: Patient organisations enabling drug development
Drug development is a complex process and there are many stakeholders involved in bringing treatments to patients. Ben Hargreaves finds that patient organisations are becoming an increasingly important link in this network, providing an invaluable line to patients and their needs.
Patient organisations have emerged as a powerful tool to convey the voice of the patient to other stakeholders in the healthcare industry. This represents a rapid development in purpose for the organisations, which were originally created to share the experience of living with certain diseases, but now involves many aspects of care, such as providing patients with information on treatment options and also assisting them in finding clinical trials. This even extends into the drug development space, where patient organisations can work with pharma companies to understand research priorities, fund research, and have input into clinical trial design and recruitment.
For the pharma industry, the role patient organisations play is an essential part of the drug development ecosystem. There are gaps in pharma companies’ ability to interact with patients and to engage with regulatory authorities on the needs of patients in specific areas. As a result of these and other factors, the importance of patient organisations is only increasing and there are more calls for greater links between the groups and those developing treatments.
Suzie-Ann Bakker, communications coordinator at the World Duchenne Organization, explained to pharmaphorum why the voice of the patient is integral to successful drug development: “Patients are the people who know best about living with their disease and what symptoms most need addressing. They also know the best form for medicines, according to the specific disease – for some, pills are acceptable; for others, only if they are really small or crushable; for some, liquid form is more appropriate/tolerable.”
Building the skills
The pharma industry has its own list of issues that it faces when it comes to successfully developing drug candidates, making the aid patient organisations can bring invaluable. However, just having a patient organisation on board is not enough. An article by the European Federation of Pharmaceutical Industries and Associations (EFPIA) outlined the key components required for individuals within patient organisations and industry to be an effective patient advocate, as well as to cooperate effectively with stakeholders:
- Persuasive in his/her communication, assertive as an expert in a specific disease area, and able to build coalitions with different stakeholders
- Equipped with knowledge about his/her disease area and about medicines research & development, the regulatory environment, and the health technology assessment process
- Aware of the patient movement, of the key players in it, of the trends, and of what may be coming next
- Familiar with industry’s compliance and legal rules of engagement, and able to advocate towards industry partners with compliant proposals
The EFPIA noted that possessing these skills naturally is rare and is instead made possible through ‘capacity building efforts’. It noted that this can be best achieved by building stronger links between not just patient organisations and pharma companies, but also with academia, patient-orientated initiatives, payers, and regulatory bodies.
Facing down challenges
One of the major areas where stakeholders are linking is at the clinical trial stage. A challenge facing clinical research is securing enough patients and retaining them once enrolled, and this is an area that can be aided by patient organisations. The organisations have good access to patients within their disease area and are able to disseminate information about available trials. This serves a purpose for all stakeholders, by encouraging patients to take up potential treatment options and allowing companies to fill trial slots to move drugs through clinical phases.
When applied to rare disease, the challenge facing pharma companies is even higher, with a limited pool of patients and a greater need to retain those that join trials. IQVIA recently published a report on the development of a treatment for an ultra-rare disease and identified the crucial role that a network of patient organisations played in the successful development of a treatment. On recruitment, the authors stated that, “[t]hese organisations were able to contact patients, provide information and education about the upcoming trial, and provide support and motivation throughout its duration.”
When it came to retention, the authors outlined that patient motivation was a major factor behind them staying through the course of clinical trial treatment or otherwise. As it can be evident when a patient is taking medication or a placebo, the patient organisation also played a role in conveying to patients how important the trials were to developing a treatment for their condition and the authors concluded that altruism became an integral element in patient retention, which could be aided by the help of a patient organisation.
Effective action
Bakker explained that, rather than relying on other stakeholders, notable action can come from within patient organisations to drive change and help create new treatments. She explained that, after a number of clinical trials were discontinued in the area, the World Duchenne Organization formed its own community advisory board from members from various countries to provide voluntary input to drug developers working in the area.
The board functions by offering meetings to drug developers starting from early stage development, “in order to give input on unmet need, clinical trial design, patient preferences, population to be investigated, outcome measures relevant to patients and their caregivers, education materials, ICFs etc.,” Bakker stated.
Once initial meetings have taken place, the discussions continue twice per year and the two parties keep in touch during the course of drug development, even convening via ad-hoc virtual meetings, Bakker added.
“The aim is to accelerate the development, approval of, and access to safe and effective drugs for our patient community by contributing to an optimal trial design and lessening the burden for patients and their families,” Bakker concluded.
