How are companies meaningfully incorporating patient voices into drug development decisions?

At Incannex, we are setting new standards by embedding the patient voice at the center of every stage of drug development. We don’t view patient input as an afterthought — it is a critical driver of how we design studies, select endpoints, and evaluate success. By integrating patient-reported outcomes, collaborating with advocacy groups, and engaging directly with patient communities, we ensure that our programs deliver therapies that address both clinical efficacy and the real-world needs of patients. This leadership approach is shaping a development model that is more responsive, more effective, and ultimately more impactful for global healthcare.

Pharma, CROs, and regulators are well aware that patient voices often provide key elements for efficient, safer, and faster drug development execution with the valuable insights they provide on unmet needs, experiences, fears, doubts, and priorities when deciding to be included in a clinical trial. It began with asking for patient feedback on difficulties understanding the ICF, and progressed to the point of supporting protocol design, recruitment, and retention plans and advising on better ways of serving patients once new drugs come into the market. Incorporating this crucial patient feedback can be easily achieved with patient advisory boards and focus groups, where individuals living with specific conditions share symptoms, treatment burdens, and daily challenges.

Regulators like the FDA and EMA also encourage patient-focused drug development (PFDD) frameworks, where even trial endpoints should align more closely with what matters to patients. Digital platforms and real-world evidence tools allow continuous feedback collection. In the medical devices development, the FDA’s Center for Devices and Radiological Health (CDRH) actively listens to patients’ experiences using patient-reported outcomes (PROs).

Implementing these and many other initiatives, doesn’t mean that science is not driving the way we bring medicines to cure, improve survival, or provide better quality of life. It is about listening the voices of those that are directly affected by the conditions we are working to improve and partnering with individuals suffering diseases, patient associations, regulators, and payers to ensure that society and the public and private sectors are investing where it can effectively improve long-term quality of life for patients.

Perhaps the most meaningful way pharmaceutical companies can do this is by addressing real-world barriers to medication adherence. Noncompliance can be attributable to unpleasant sensory profiles, inconvenient dosing schedules or other delivery challenges, and is a key indicator of the lack of patient input into drug design.¹ To close this gap, pharmaceutical manufacturers are prioritizing patient experiences. For children, this means replacing “off-label” adaptations of adult drugs with tailored dosage forms, such as mini-tablets or 3D-printed chewables, that allow for customizable dosing and better palatability. Elsewhere, the encapsulating properties of excipients like hydroxypropyl-β-cyclodextrin (HPβCD) are being used to mask unpleasant-tasting APIs, such as clarithromycin, helping reducing side effects, such as nausea and gagging.² Controlled or extended release versions of drugs, such as metformin for type 2 diabetes, are similarly improving convenience by reducing pill fatigue for patients managing chronic disease.³ Together, these innovations reflect a broader shift toward patient-centric design, where pharmaceutical companies emphasize clinical efficacy and safety, as well as improvements to usability, comfort, and quality of life. By embedding patient feedback into formulation choices, producers are not just improving compliance, but are also developing drugs that truly serve the people who rely on them.

1. Kim, Jennifer, et al. “Elephant in the Room.” US Pharm. 43: 30–34 (2018).

2. Jansook, P, N Ogawa, and T Loftsson. “Cyclodextrins: structure, physicochemical properties and pharmaceutical applications.” International Journal of Pharmaceutics. 535: 272–284 (2018).

3. Hanefeld, M, and J Drzewoski. “The Current and Potential Therapeutic Use of Metformin—The Good Old Drug.” Pharmaceuticals. 14: 122 (2021).

One way companies are weaving patient voices into drug development is by leveraging the power of “living datasets,” dynamic, continuously updated data flows that capture the realities of patient experiences. Rather than relying on static trial snapshots, organizations are incorporating continuous streams from wearables, lab tests, patient-reported outcomes, and other real-world sources. This ensures that data reflecting how treatments affect daily life integrates directly into decision-making.

Expanded data types, such as genomics, behavioral insights and real-world evidence, provide a more complete picture of patient needs. By then applying advanced analytics, machine learning, and AI, teams can uncover patterns in safety and efficacy that may otherwise be overlooked, elevating aspects of care that matter most to patients. These insights also inform adaptive trial designs, enabling researchers to refine patient selection and respond to emerging trends more quickly.

The potential is an approach that reduces patient burden. Remote data capture and decentralized methods allow patients to contribute without repeated site visits, broadening access and reflecting the diversity of real-world populations. With richer, near real-time feedback guiding decisions, companies can ensure that trial strategies align more closely with patient priorities, making therapies not only scientifically sound but meaningful for patients in need.

The top priority across the pharmaceutical industry has always been and will always be ensuring the safe and efficient delivery of medications to patients in need. Today, many drug developers are balancing that necessity with patient centricity, advancing efficacious therapies that integrate patients’ experiences, needs, and preferences.

The growing popularity of pre-filled syringes for GLP-1 medications, such as Ozempic and Wegovy, represents this transition to patient-friendly drug delivery methods. In recent years, many pharmaceutical companies have moved from liquid vials to pre-filled syringes, which not only helps to overcome the complexities of product storage and administration but also provides patients with easier to use, self-administered formats for these life-changing therapies.

The industry has also made exciting progress when it comes to on-body delivery systems (OBDS). These unique devices can automate delivery, are more comfortable for patients and can provide consistent and accurate dosing, every time. In fact, data shows greater patient adherence to medications administered via OBDS.

Going forward, the industry will continue to innovate with patient experience at the forefront of the entire drug development journey. These innovations will lead to improved patient outcomes and increase likelihood of market success.

A patient-centric approach fosters trust, transparency, and shared ownership, transforming drug development into a collaborative ecosystem guided by lived experience. True innovation in rare disease therapeutics depends on the ability of drug developers to efficiently collaborate with patient advocacy organizations and disease foundations. These groups can provide invaluable vital resources, such as natural history data, patient registries, biobanks, and are instrumental for patient recruitment to clinical trials.

Engaging patients early ensures therapies reflect real-world needs. Patients and caregivers offer unique perspectives on disease burden, symptom variability, and treatment priorities often missed by traditional endpoints. Patient-reported outcomes and natural history studies, facilitated by advocacy groups, inform target selection, biomarkers, and endpoint validation.

Organizations like the Cystic Fibrosis Foundation and CureDuchenne have accelerated transformative therapies and helped companies navigate regulatory pathways as agencies prioritize patient-focused frameworks. Incorporating patient perspectives strengthens trial design, recruitment, and retention. Flexible protocols, decentralized models, and remote monitoring, often shaped by patient feedback, make participation feasible and meaningful for the target population.

Pharmaceutical companies are increasingly adopting patient-centric approaches by integrating patient input throughout the drug development life cycle. This includes establishing patient advisory boards and conducting surveys or listening sessions to inform clinical trial design, endpoint selection, and protocol development, reducing delays and improving relevance. This can be considered as a dynamic approach. Early involvement in drug discovery helps align research with patient needs, such as through capacity-building initiatives that foster an organizational mindset focused on patient engagement. Partnerships with advocacy groups are especially effective to amplify patient voices in preclinical research, ensuring priorities match lived experiences. Regulatory frameworks like the FDA's Patient-Focused Drug Development program encourage incorporating patient perspectives in evaluation and decision-making. Additionally, patient narratives are woven into medical affairs communications to make data more relatable while maintaining compliance. Overall, this shift drives more impactful outcomes by treating patients as partners rather than subjects

Companies are increasingly integrating patient voices into drug development in ways that directly support regulatory decision-making. Early engagement through advisory boards, focus groups, and surveys helps sponsors identify meaningful endpoints and prioritize outcomes, such as quality of life or daily functioning, that regulators now expect to see in trial designs. Patient input is also shaping eligibility criteria and simplifying protocols to improve trial accessibility and retention, elements that health authorities view as critical to generating robust evidence. Importantly, patient advocacy groups today are stronger and more powerful than ever, wielding significant influence and driving meaningful change across development programs. Their involvement not only amplifies individual patient perspectives but also helps companies and regulators align on benefit–risk assessments and long-term commitments. Patient representatives increasingly review protocols and contribute to plain-language summaries that accompany regulatory submissions, ensuring clarity and transparency. By embedding patient perspectives, strengthened by advocacy group leadership, into both evidence generation and regulatory dialogue, companies enhance compliance, accelerate approvals, and deliver therapies that truly reflect patient needs.

Incorporating patient voices into drug development decisions means more than asking for feedback, it’s about creating tangible ways for patients to shape the process. At donor centers and clinical sites, we see this firsthand. By working with both healthy individuals and those living with disease, we’re able to provide opportunities for people to contribute, often through something as simple as a blood donation. Those samples are incredibly powerful in early-stage research, where access is limited, and they allow scientists to explore new biomarker targets and better understand differences across patient populations. When developers combine these biological insights with direct patient perspectives, they’re not just advancing science, they’re ensuring that therapies are developed with patient needs and realities at the center. 

At IMUNON, we believe that meaningfully incorporating patient voices begins with building trusted partnerships with patients, caregivers, and advocacy groups. These relationships are especially vital in areas like ovarian cancer, where unmet needs remain high and lived experiences can vary widely. By collaborating with patients, families and advocacy organizations, we gain critical insights that shape trial design, endpoint selection, and overall development strategy — ensuring our therapies reflect what truly matters to the ovarian cancer community. In our ovarian cancer development program, we integrate patient-reported outcomes to capture feedback on symptoms, treatment burden, and quality of life. These data are not only scientifically rigorous but will ensure that our phase 3 trial is patient centered Our engagement with patient advocacy organizations provides additional channels of communication and transparency from, and to, the ovarian cancer patient community. Patient input also guides our broader strategy, from anticipating access challenges to ensuring diverse representation in clinical trials. By listening closely and engaging meaningfully, we aim to develop therapies that are not only innovative but also aligned with the values and priorities of the communities we serve. 

Companies are increasingly recognizing that successful drug development isn’t only about extending survival but also about improving overall quality of life. Patient voices are shaping decisions around tolerability, ease of use, and day-to-day impact. For example, oral therapies are often preferred because they reduce treatment burdens compared to hospital-based infusions. Minimizing toxicity is another major priority; if treatment is less toxic, patients are more likely to complete it and integrate it into their lives. This balance of efficacy, tolerability, and convenience is becoming central to how companies evaluate new therapies. Incorporating patient perspectives helps researchers understand what matters most: patients not only living longer but also living better. In cancer treatment, especially, the calculus has shifted from focusing solely on tumor shrinkage or eradication to considering how therapies affect a patient’s body and mind. By bringing patients into advisory boards, focus groups, and discussions on trial design, companies can ensure that the result is a therapy that truly addresses patient needs.