Every number has a face: Are you valuing the patient voice?

Considering the needs of patients during drug development is a crucial step to guaranteeing the most inclusive treatments possible for them. Many pharma companies lose sight of the patient experience when developing treatments so, how can they improve?
Imagine having to choose which risk you are willing to take to slow the progression of your disease. Could you give up the freedom to have children in opposition to when you and your partner want? Could you risk a potentially fatal rare brain infection as a potential cost of care? Could you risk kidney or thyroid disease just for the sake of a treatment?
These are the life-changing decisions that people living with multiple sclerosis (MS) are forced to make when choosing to begin the most aggressive form of treatment. Like many living with a rare condition, these patients rely on a drug to improve their health and quality of life. MS patients who do require the most aggressive treatment place their hopes in ocrelizumab, natalizumab, and alemtuzumab.
A patient’s drug regimen is the most promising gateway back to a normal life. Patients with aggressive MS often find they must sacrifice their body and dreams to avoid disability.
My question is: why are MS patients forced to make these life-altering decisions when most are diagnosed in their 20s, 30s, and 40s? Those living with MS may have been consulted at the time, but viewed the substantial risk as one worth taking until a better treatment was developed. If so, was their voice heard throughout the drug development process when creating the new drugs for the future? I wish I had the answers.
Involving patients from the beginning of drug development may prevent treatments, such as for MS from being developed without consideration of the patients’ needs and wants. Patients and patient advocacy groups are the true experts in their condition; these individuals understand the patient experience and can shed light on the reality of living with MS. Not involving patients in the R&D process devalues their expertise and hinders the effectiveness of a treatment. To develop, manufacture, and sell a treatment to a population successfully, the voice of the patient is critical.
Understanding a disease goes beyond the science. While pharmaceutical companies must ensure that a drug is both safe and effective for patients, they need to understand how a patient population will perceive the treatment being offered. By hosting focus groups with patients and patient group leaders who are affected by the condition, pharma companies can learn exactly what the community wants, needs and expects from a treatment. That information can be used to ensure that a product or drug is delivered in a way that best suits the targeted patient population. This point rings true, whether you are developing the first treatment for a rare disease or creating a new treatment for a condition that already has a current therapeutic on the market.
‘Social listening’ is one way to gain insight into a particular patient experience. Joining an open patient support group on Facebook to see which conversations are being had by those living with the condition can prove enlightening. You’ll uncover truths that you may have never contemplated before. You’ll learn the good, the bad, and the ugly in an authentic, unfiltered, and raw way. Whether you want to learn more about what it’s like to live with that condition, or which treatments are currently available (if any) for it, these communities provide the insight you need to develop a drug that addresses their unmet needs.
To illustrate this point, a senior executive involved in the development of a treatment for progressive MS told me that her company initially wanted to use bottles with safety caps. She insisted that she needed to first sit down with a group of progressive MS patients to test the prototype bottles before proceeding with the design. It was during this focus group that she realised that progressive MS patients didn’t have the manual dexterity to open the bottles. Had she not insisted on speaking with those who would be taking the treatment, her company never would have been aware of this significant insight.
“ It is easy to get bogged down in the science of a drug and forget about the patient experience of those who are relying on your research. Rare disease patient populations may be small, but each number is a human being 
I encourage you to explore a rare disease patient group’s website for your specific condition of interest, as the insights from patient forums can be invaluable. You will find an array of first-hand information about a condition’s cause, symptoms, and quality of life. The questions, answers, concerns, and lived experiences are all there. This is particularly important for those who are developing treatments in the rare disease space.
Jason Colquitt, CEO of Across Healthcare, emphasises this critical need when he asks, “How can we appropriately help care for rare disease patients without giving them an appropriate voice? Co-production, a non-healthcare concept first described in the 1970s, has proven that those who are affected by a service are the best ones to help design it. Input from the patient should be a top priority when delivering care and facilitating research in the rare disease space.”
I am delighted that rare diseases are beginning to gather more interest from the pharma industry. Through drug repurposing and adopting a co-production, collaborative approach, the pharma industry is giving hope to rare patients who were previously overlooked due to low patient population numbers and a high cost of drug development. The pharma industry alongside the healthcare and academia sectors are now investing in rare research, and are placing a higher value on the patient voice. These sectors are actively collaborating with rare disease patients and support groups to deliver treatments that are better targeted to the patient populations they serve.
Rare disease patient groups have more than earned their seat at the table. Their efforts have proven the value of putting patients at the heart of rare disease research and care. Despite having limited time, funding, and resources, rare disease patient groups have successfully organised patient consultations at NICE, supported the delivery of highly specialised services for ultra-rare diseases, and launched their own rare disease patient registry to aid in research. Rare disease support groups have shown why it is crucial for the pharma industry to value the patient voice. They have lobbied tirelessly for patients and their loved ones to gain access to the treatments needed to improve their lives.
I agree with Donovan Quill, President and CEO of Optime Care, when he says, ‘Patient-first’ has become a buzzword for many specialty pharmacy organisations that merely focus on profits. A true patient-first approach can enable pharma companies, pharmacists, physicians and other members of the care team to better address compliance and adherence to treatment, improve outcomes and enhance quality of life for patients. From day one, patient-first experts provide ongoing support and are available to answer questions. For many with rare diseases, and especially for those starting a new medication, this level of support is comforting and effective.”
Patient-first cannot become a buzzword, nor can it be a box to check half-heartedly. Rare disease patients and their support groups are watching. They will hold you accountable and demand equitable, inclusive treatment that meets their needs. The pharma industry, rare disease patient groups and rare patients all have the same goal: to create tailored treatments that are safe, effective, and designed with patients’ needs in mind.
My challenge to the pharma industry is to never lose sight of the patient experience when developing rare disease treatments. It is easy to get bogged down in the science of a drug and forget about the patient experience of those who are relying on your research. Rare disease patient populations may be small, but each number is a human being who has dreams, hopes and fears. Rare diseases are cruel, unfair and overwhelming. Living with one can challenge, devastate and isolate. The treatments you create are what keep the fire and hope alive for rare patients and their families.
If you only remember one message from this article, I would like it to be this:
See the people, not the profits. Every number has a face. Every number has those who love them. Every number has a life to live. Every number depends on you. Listen to those you serve. Listen to the patient voice.

Blayne Baker is the Marketing and Engagement Manager at Findacure, a UK rare disease charity that is building a united rare disease community driven by patient groups. Findacure envisions a world in which all rare diseases have treatments made together with patients, for patients. If you’d like to learn more about Findacure’s work and how you can partner with them, please email Blay