Q&A: Patient-Centric Drug Development Should Start at Product Definition

Medication nonadherence remains one of the widest gaps between how a drug performs in clinical trials and how it performs in practice. The World Health Organization has estimated that adherence to long-term therapies in developed countries averages about 50%, and a review in identified regimen complexity, adverse effects, and health system factors, not patient behavior alone, among the drivers.^1,2 The FDA has also continued to formalize patient input in development, finalizing the third installment of its four-part Patient-Focused Drug Development guidance series in late 2025.³

For formulation and manufacturing teams, the challenge is translating those priorities into dosage-form, packaging, and supply decisions. Ahead of his presentation at CPHI Worldwide 2026 (Oct. 6–8, Milan), Ron Scarborough, CEO of Azurity Pharmaceuticals, discussed why nonadherence should be examined as a design issue.

Access Part 1 of the video interview in which Scarboro explains why real-world usability belongs at product definition.

Access Part 2 of the video interview in which Scarboro discusses how drug reformulation that removes patient barriers must preserve efficacy.

Access Part 3 of the video interview in which Scarboro explains when formulation robustness justifies added cost.

Pharmtech: If Medication Nonadherence Is a Design Problem, When Should Real-World Use Be Evaluated?

Scarboro: It should start when you define the product. We must ask early who will take this medicine, where they will take it, and what might make that difficult. That means bringing patient, clinical, formulation, regulatory, and commercial perspectives together. You can't anticipate every barrier, though. You may discover after launch that a dosing instruction is hard to follow consistently. Those observations should be treated as design information, rather than assuming the patient needs more reminders. Our medical science liaisons and medical strategists work with key opinion leaders and physicians to feed that back into development.

What Is the Hardest Trade-Off When Reformulating a Proven Therapy?

The hardest part is changing the conditions of use while preserving confidence in how the medicine performs. A fasting instruction may be closely tied to how the drug is absorbed. A new formulation may remove that barrier, but it must meet the same applicable standards for exposure, safety, and efficacy. Convenience matters only if the medication continues to do its job. Getting there takes iterative formulation work, careful testing, and regulatory, clinical, and manufacturing input throughout. Every product must stand on its own evidence. If the data don't support the change, you change course.

How Can Smaller Companies Make a Reengineered Therapy Commercially Viable?

Start with a specific patient-use problem. Bring development, medical, regulatory, quality, manufacturing, supply, and commercial teams together early, and have them ask four questions. Can we make an improvement? Can we produce it reliably? Can we get it to the patient? Can we support it commercially? When these issues are examined one department at a time, the difficult decisions come late and become expensive. Smaller companies can work through partners or licensing, but someone must own the entire pathway, from the unmet need through approval, supply, and use.

When Is Added Supply Chain Robustness Worth the Cost?

It depends on where the medicine will be stored, who will administer it, and what the supply chain and care setting can support. A specialist center is different from a community clinic or a patient's home. Robustness doesn't mean claiming performance outside validated conditions. It means designing and testing for the intended environment. Extra complexity is a cost, but so are damaged product and disrupted supply.

What Metrics Should Regulatory and Quality Teams Track After Approval?

I would begin with complaints, packaging and administration issues, and whether the product meets quality standards through distribution. I would pair those with supply availability. Where data are reliable, I would add adherence and persistence. Approval is only a milestone. The real measure is whether the medicine makes a difference for the patient who depends on it every day.

References

1. World Health Organization. Adherence to Long-Term Therapies: Evidence for Action. WHO; 2003.
2. Osterberg L, Blaschke T. Adherence to medication. N Engl J Med. 2005;353(5):487-497. doi:10.1056/NEJMra050100
3. FDA. Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments. Final guidance. 2025. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-focused-drug-development-selecting-developing-or-modifying-fit-purpose-clinical-outcome