The lengthy drug development process in the United States progresses through five key stages, including pre-clinical and clinical research trials, before undergoing regulatory review by the Food and Drug Administration (FDA). Historically, patient input has only been sought at the conclusion during FDA Advisory Committee hearings. Dr. Leen Kawas advocates that it’s time for change – patients’ perspectives should help shape multiple phases of advancement.
Dr. Kawas has firsthand expertise in actively involving patient priorities while previously leading Athira Pharma, Inc. as Chief Executive Officer. As Managing General Partner at Propel Bio Partners, Dr. Kawas urges biotech entrepreneurs within the venture capital firm to integrate patient insights. She encourages Propel’s partners to gather regular patient contributions when creating new therapies.
The five steps in the standard drug development cycle are discovery, preclinical testing, clinical trials, regulatory review, and post-approval monitoring. The discovery phase identifies disease processes to target and test molecular compounds. The preclinical phase analyzes toxicity before human trials commence. Clinical trials establish safety and efficacy in people. Regulatory bodies like the FDA then rigorously evaluate all data and decide on approval. Post-approval, the FDA continues tracking prescription medications to maintain admaintain adequate standards a Patient-Focused Drug Development initiative acknowledging patients’ meaningful involvement can strengthen therapies. Focus areas include easing burdensome trial enrollment, communicating clearly about treatment trade-offs, capturing patient priorities, and funneling insights directly into decision-making. As a seasoned scientist and investor guiding new companies, Dr. Kawas wholly supports integrating patient voices, especially in trial design.
Drawing from her CEO tenure, Dr. Kawas prioritized patient-friendly programs that boosted retention and made participation more convenient and rewarding. For one Alzheimer’s disease trial, she had meals catered on-site for caregivers and patients. Dr. Kawas emphasizes that when trials directly address patient requirements, they elicit greater meaning, adherence, and better scientific outcomes for all.
Today’s tech-adept patients increasingly seek information and share experiences online through forums and advocacy networks. Some even participate in pharma-run closed groups centered on conditions and medications. This two-way knowledge exchange both empowers patients and provides data to improve care. Patient organizations partner directly with drug companies to pinpoint priorities and generate patient-driven solutions.
Ideally, patient organizations should collaborate with ceutical companiespharmaceutical companies across three domains:: boosting clinical trial recruitment and retention, clarifying complex legal terminology in study materials, and resolving logistical barriers for patients around participation. As Dr. Kawas steers startups through company-building, she advocates for patient input through all facets of development. With extensive drug innovation expertise, Dr. Kawas aims to open more avenues for patients’ collective voices to help shape future therapies.