Patients and their families not only wish to be involved in decisions about their own healthcare. They also increasingly strive to be involved more directly in the research and development of therapies. Indeed, regulators and payers are increasingly mandating patient engagement in drug development.
After all, patients and their families/caregivers know best about their disease, particularly when we think of rare diseases, about the unmet need, which aspects and symptoms are most in need of therapeutic intervention to improve their well-being and quality of life. They are the ones living with the disease on a daily basis. They know what needs to change to make a difference to their daily lives and what their expectations are. Depending on the disease, these can range from a cure to stabilization, slowing down disease progression or simply managing some of the worst symptoms to enhance quality of life.
But: Patients and their families/caregivers do not want to be a mere tick box exercise!
They both deserve to and should be involved throughout all stages of drug development. Nothing ab0ut us without us!
Involving patients/patient representatives is vital:
- To ensure that researchers and pharmaceutical companies address the issues most important to the patients
- To enable the design of optimal, patient-friendly clinical trials that better meet the needs and preferences of the participants and minimize the (often considerable) burden of participation in these trials
- To increase the chance of success for the company and the patients
There are various ways to involve patients in the process of drug development:
Pharmaceutical companies can organize focus groups or workshops or set up so-called Ad-boards. However, these can be biased, as the company chooses who should represent the patients, the location, organizes travel, accommodation and hospitality, sets the agenda and establishes the results.
After experiencing a number of such Ad-boards as well as encountering a number of failed clinical trials for various reasons (wrong population, endpoints not sensitive enough to change, and other factors), the World Duchenne Organization (WDO) made the decision to initiate its own disease specific Duchenne Community Advisory Board (CAB) in 2018 in alignment with the EUROCAB programme set up by EURORDIS.
The advantage of this approach is that the Duchenne CAB is independent, led by patient representatives with expertise, and is non-biased. Its members are recruited from WDO member organizations and trained in all aspects of drug development, from pre-clinical research through to access and reimbursement. It is an international group from twelve different countries, covering all ages/stages of this life-limiting disease, and the aim is to represent neither our own children nor our own country, but all patients with DMD all over the world.
The Duchenne CAB
The express goal of the Duchenne CAB is to provide its accumulated experience and knowledge in the global endeavour to accelerate the development of safe and effective treatments for DMD patients worldwide.
WE invite companies to OUR table; set up a collaborative agenda that integrates the needs of the company as well as those of the community we represent; WE compile the notes and determine the outcomes of the discussion in our letter of recommendations. WE organize the venue and provide a safe space for confidential and honest discussion. Confidentiality and trust are vital for these interactions, as is the assurance that we are taken seriously. Follow-up after a meeting/in between meetings gives us this confidence and informs us on what is being done with our advice, what changes to the protocol/formulation/policies have been made. This ensures that the Duchenne CAB is definitely NOT just a tick box.
Since its initiation in 2018, the Duchenne CAB has organized 12 3-4 day sessions (bi-annually) comprising 64 individual meetings with 18 companies. It is not (generally) a one-and-done exercise but a collaborative partnership over the development life-cycle.
Some of the advice implemented and topics discussed are:
- Patient preferences on duration and ratio of placebo randomization
- Benefit-risk preferences and managing expectations
- Biopsies – necessity/number/method
- In-/exclusion criteria and meaningful and relevant outcome measures/endpoints in the population to be studied
- Policies: cross-border/sibling (for families with more than one child with DMD)/expanded access/compassionate use
- Reducing the burden of clinical trials: e.g. home/local hospital visits and blood draws where feasible; home infusions
- Access to drug during the gap between market authorization and commercial access
- Commitment to sharing both aggregate and individual clinical trial results with patients
What is the added value of this approach for the pharmaceutical companies (quotes taken from surveys following meetings)?
- The opportunity to discuss with the Duchenne CAB provides a reality check to how industry may think about clinical studies, and the discussion will certainly be included regarding the clinical development plan
- The Duchenne CAB has provided great insights into: study design parameters, outcomes, ICFs and education required for patients/ families
- Demonstrating we have worked with those living with the disease is becoming a critical component of the discussion with regulators and may help shape their thoughts on clinical trial requirements
- Engaging individuals living with disease in drug development and regulatory processes leads to more thoughtful and sensitive trial designs, drives more informative and meaningful outcomes from clinical studies, and builds trust between the public, government, and industry stakeholders. This engagement is especially important in the case of rare diseases, where affected individuals and their families face many difficulties getting information, treatment, and support
- Partnering with individuals and families living with DMD (…) helped (…) improve clinical trial design and reduce the difficulties affected individuals and their families face when participating in these clinical trials
The pharmaceutical industry can only benefit from well-organised interactions with disease-specific community advisory boards, and the disease community also benefits: With optimal, patient-friendly clinical trials, recruitment and retention rates are much higher, results can be established faster and the therapy potentially reaches patients sooner.
