Dr. Leen Kawas Explains Why The Patient’s Voice Must Be Integral To The Drug Development Cycle

Leen Kawas
5 min readJan 25, 2024

The United States’ drug development cycle involves a five-step progression during which the drug candidate’s safety and efficacy undergo close evaluation. Pre-clinical research, clinical research, and a detailed Food and Drug Administration (or FDA) review are key parts of the process.

Near the development cycle’s conclusion, an FDA Advisory Committee may be convened to address specific issues. A Patient Representative is often invited to join this panel. Until this phase, however, the patient has historically not been included in the drug development process.

Leen Kawas, Ph.D. emphasized that it’s time to change this practice. She strongly believes that the patient’s voice should be integrated into multiple phases of the drug development cycle. In fact, Dr. Kawas previously put this principle into practice while serving as Chief Executive Officer (or CEO) of Athira Pharma, Inc.

Today, as Propel Bio Partners’ Managing General Partner , Dr. Kawas advocates for patient involvement while assisting the venture capital firm’s biotechnology entrepreneurs. She encourages the firm’s partners to include patient contributions in drug development.

The United States Food & Drug Administration oversees the drug development process, required for all U.S.-based drug candidates. Candidates are weeded out for varied reasons, with only a small percentage receiving FDA approval.

This two-step phase begins when researchers gain insights into a disease process. Alternatively, researchers may look for molecular compounds’ favorable effects. Current treatments with unplanned results, and new technologies that may facilitate targeted treatments, may also be discovery sources.

After researchers pinpoint a potential drug development candidate, they perform experiments centered around the drug’s performance. During this phase, researchers analyze the drug’s optimal dosage, side effects, interactions, and comparative efficacy.

Before the drug’s human trials can begin, researchers must determine whether the drug can potentially harm those taking it. During this preclinical research phase, researchers either perform In Vitro research (done in a test tube or lab dish) or In Vivo research (performed on a living organism). Following this phase, researchers determine whether to pursue human testing.

Clinical research demonstrates how the drug will interact with the human body. Carefully designed clinical trials are the foundation of this phase. Before that begins, however, developers must complete the Investigational New Drug Process.

Next, researchers review available information before designing the drug’s clinical trial phases. Clinical trials typically conform to a three-phase study format. Small-scale early Phase 1 studies contrast with large-scale late Phase 3 studies.

Assume the drug developer now has evidence that a drug is safe and effective during its intended use. The firm can now apply to market the drug via a New Drug Application.

The FDA’s review team carefully evaluates all provided data before deciding whether to approve or deny the application. This phase can involve multiple reviews and exchanges between the FDA and the drug developer.

Because no one can predict a drug’s future performance, the FDA analyzes reports of issues with prescription medications and over-the-counter drugs. The FDA may add certain cautions for a drug’s use and may take additional action for serious occurrences.

The Drug Administration has acknowledged patients’ key role in the drug development and analysis cycle. To integrate patients into the process, the FDA has launched the Patient-Focused Drug Development (or PFDD) Program.

This forward-thinking approach seeks to bring patients’ needs, priorities, and experiences into the complex drug development cycle. In the FDA’s view, each patient’s firsthand knowledge of their medical condition can be invaluable in developing targeted therapies. The FDA desires to incorporate the patient’s voice in four significant ways:

  • Identify Best Practices to Enable Smoother, Less Burdensome Patient Clinical Trial Enrollment
  • Enable Better Patient Decision Making Through More Effective Communication Regarding Treatment Risks, Benefits, and Burden
  • Engage Appropriate Methods to Obtain Information on Patient Preferences and Their Attitudes Toward Treatment Benefit vs Risk
  • Develop Structured Patient and Caregiver Feedback to Better Inform Drug Development and Regulatory Decision Processes

Propel’s venture capital leadership combines financial support and biotechnology expertise. Once a founder is invited aboard, Dr. Kawas guides the company’s drug development cycle, including its clinical trial phase. She takes concrete actions to make patients an integral part of this rigorous process.

Dr. Leen Kawas developed her patient-centric focus while serving as Chief Executive Officer of Athira Pharma, Inc. While managing a high-achieving drug development team, Dr. Kawas kept patients’ goals at the forefront.

“We built a team that did not think about barriers. We only thought about solutions and how we can do things better, differently, with a mindset that we are serving the key stakeholders, which are the patients.

“Track the success and satisfaction of your customers (in life sciences, the patients). That’s going to drive value. You are developing therapies. You are changing people’s lives. Once you achieve that, the financial value is going to follow,” Dr. Leen Kawas remarked.

Clinical trials can only proceed when a certain number of patients agree to participate. However, patients may be hesitant to enroll if trials conflict with other commitments and/or involve stringent requirements. Dr. Leen Kawas understood that insufficient patient participation means a drug development program grinds to a halt.

To encourage higher clinical trial participation, Dr. Kawas pinpointed the factors important to the patients. In other words, she wanted to give the patients a voice in the clinical trial design, a somewhat unusual practice in biotechnology circles.

“If you design clinical trials that have the patient’s voice in them, patient retention will increase (which is a problem in our industry).” Dr. Kawas also made clinical trial participants’ well-being a priority. During one clinical trial, she ordered onsite meals for participating Alzheimer’s patients along with their caregivers.

Today’s technology-savvy patients are taking a more active role in their healthcare and physical well-being. This growing participation extends to patients’ involvement in the drug development cycle. The December 2021 edition detailed two ways in which patients are making their voices heard.

Historically, many patients depended heavily on their healthcare provider for medical condition management. Today, however, Internet-savvy patients regularly delve into credible sources such as peer-reviewed studies and other reliable medical content. The patient also brings their firsthand experiences into the mix. Forward-thinking healthcare providers are actively listening to patients’ voices.

Patients are also sharing their information on social media, in patient forums, and in patient advocacy groups. This collective knowledge exchange is helping clinicians better identify and manage rare disease risks.

Finally, certain pharmaceutical companies now operate closed patient groups. These communication-rich exchanges often spur patients to better adhere to their medication regimens.

Patient organizations bridge the gap between pharmaceutical firms and patients. In these associations, patients can share experiences and feel confident the pharma companies are listening.

Taking it a step further, patient organizations can enter into collaborations with pharma companies. The organization’s goal: to address patient priorities and spur the growth of experience-driven drug development.

Ideally, pharmaceutical firms will work with patient organizations in three ways. First, patient organizations can offer recommendations on improving patient clinical trial recruitment (and retention).

Next, patient organizations can point out barriers to recruitment such as unclear legal and technical jargon in clinical trial information. Patients’ difficulties in understanding these details could deter them from trial participation.

Finally, patient organizations can address patient logistical concerns about clinical trial enrollment. Issues may include work commitments, family responsibilities, and lack of transportation. Resolving these issues can help drive better patient trial participation.

Originally published at https://washingtonindependent.com.

--

--