Developing new medications includes several steps where a drug candidate’s protection and usefulness face close review. Preclinical and clinical research, plus an in-depth inspection by the Food and Drug Administration (FDA), are integral portions of this progression.
As this course nears its end, an FDA Advisory Committee may gather to tackle specific questions. Here, a Patient Representative is invited to participate on the panel. However, patients have yet to contribute to creating new pharmaceuticals up to this point.
Leen Kawas, Ph., emphasized it is time to amend this tradition. She fervently thinks patient perspectives should integrate into various phases of the drug development cycle. Dr. Kawas previously employed this tenet while occupying the Chief Executive Officer (CEO) role at Athira Pharma, Inc.
Now, as Propel Bio Partners’ Managing General Partner, Dr. Kawas promotes patient engagement while aiding the venture capital firm’s biotech entrepreneurs. She motivates the company’s stakeholders to welcome patient input during drug development.
Five Steps to Developing Pharmaceuticals
The FDA oversees the drug development procedure mandated for all U.S. drug candidates. Applicants wash out for varied rationales, with only a few obtaining FDA authorization.
Step 1: Discovery and Development
This two-part phase starts when experts gain an understanding of a health condition. Alternatively, academics may seek positive impacts from molecular compounds. Current treatments with unintended effects and new technologies that enable targeted remedies may also spark discovery.
After researchers identify a prospective drug development candidate, they conduct experiments focused on the drug’s performance. Academics analyze optimal dosage, side effects, interactions, and comparative efficacy here.
Step 2: Preclinical Research
Before human trials can launch, experts must decide if the drug could potentially damage volunteers. Throughout this preclinical research phase, scholars perform either In Vitro (in a test tube or lab dish) or In Vivo (in a living organism) investigations. Next, academics determine whether to proceed with human experimentation.
Step 3: Clinical Research
Clinical research reveals how the drug affects the human body. Carefully designed clinical studies form the core of this phase. However, first, developers must finish the Investigational New Drug Process.
Then, academics evaluate existing data before outlining the drug’s clinical trial stages. Clinical studies usually match a three-phase format. Small early Phase 1 trials differ enormously from significant late Phase 3 experiments.
Step 4: FDA Drug Review Process
Presume the drug maker now has proof a medication is safe and effective during intended use. The company can then apply to market the drug via a New Drug Application. The FDA’s experts thoroughly analyze all submitted data before approving or denying the request. Multiple critiques and exchanges often unfold between the FDA and drug developers.
Step 5: FDA Post-Market Drug Safety Monitoring
The FDA evaluates adverse reports on prescription and over-the-counter medicines because no one can predict a drug’s future performance. The Agency may append certain precautions on a drug’s use and take added steps for severe events.
FDA Realizes Patients’ Value in Drug Advancement
The FDA has recognized patients’ vital position during pharmaceutical progression and evaluation. The FDA created the Patient-Focused Drug Development (PFDD) Program to bring patients into the process.
This progressive tactic incorporates patients’ requirements, priorities, and experiences into the intricate drug development cycle. From the FDA’s perspective, each patient’s first-hand knowledge of their condition can prove invaluable when creating targeted treatments. The FDA wants patients involved in four main ways:
- Pinpoint Best Practices to Streamline Patient Clinical Trial Enrollment
- Improve Patient Decision Making Via More Effective Communication on Treatment Risks, Benefits, and Burden
- Use Appropriate Techniques to Gather Data on Patient Preferences and Perspectives on Treatment Benefit vs. Risk
- Develop Structured Patient and Caregiver Opinions to Better Inform Drug Development and Regulatory Judgments
Dr. Leen Kawas Advises Patients’ Voices in Clinical Studies
Dr. Leen Kawas strongly supports the FDA’s Patient-Focused Drug Development program. As an esteemed bioscience expert and Propel Bio Partners’ Managing General Partner, Dr. Kawas integrates this approach into her work with biotech entrepreneurs.
Propel’s venture capital leadership blends financial backing and biotech expertise. Once an owner joins, Dr. Kawas guides the company’s drug development cycle, including clinical trials. She takes concrete actions to make patients vital to this strict progression.
Dr. Leen Kawas’ Patient-Focused Clinical Trial History
Dr. Leen Kawas cultivated her patient-centric attitude while occupying the CEO role at Athira Pharma, Inc. While steering a high-performing drug development group, Dr. Kawas kept patients’ aims at the lead.
“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 will follow,” Dr. Leen Kawas commented.
How Patients Can Bolster Clinical Trial Participation
Clinical trials only advance when enough patients volunteer. However, patients may hesitate to enroll if trials conflict with other duties and require strict criteria. Dr. Leen Kawas realized insufficient patient involvement halts a drug development agenda.
Dr. Kawas identified what matters to patients to encourage higher clinical trial participation. Put simply, she wanted patients’ voices to be evident in the clinical trial design, an unconventional tactic in biotech.
“If you design clinical trials that have the patient’s voice, patient retention will increase (which is a problem in our industry),” Dr. Kawas remarked. She also made clinical trial participants’ health a priority. During one study, she mandated onsite meals for participating Alzheimer’s patients and their caregivers.
How 21st-Century Patients Engage in Drug Advancement
Today’s tech-smart patients are more active in their care and wellness. This expanding involvement reaches patients’ contribution to drug development. The Pharma Times’ December 2021 issue outlined two ways patients make their voices heard:
Dynamic Information Exchange
While many patients once heavily relied on their provider, Internet-fluent patients now dive into reliable sources like peer-reviewed studies and credible medical content. Patients also contribute personal experiences. Progressive healthcare professionals actively listen to patients’ perspectives.
Patients also share information on social platforms, patient forums, and advocacy groups. This collective knowledge exchange helps clinicians better detect and manage rare disease risks.
Furthermore, some pharma firms now have closed patient communities. These communication-rich trades often encourage patients to follow medication regimens better.
Active Patient Organizations
Patient groups bridge the gap between pharma corporations and patients. Patients can share experiences in these associations and feel assured the companies are listening.
Moreover, patient groups can partner with pharma firms to tackle patient priorities and drive patient-focused drug advancement.
Patient Groups and Drug Development
Optimally, pharma companies will collaborate with patient groups in three ways. First, patient groups can recommend improvements to clinical trial recruitment and retention.
Additionally, patient groups can indicate barriers to recruitment, like perplexing legal and technical wording in clinical trial details. Patients needing help to grasp these specifics may avoid participating.
Finally, patient groups can tackle logistical worries about clinical trial enrollment. Hurdles may entail work duties, family responsibilities, and transportation gaps. Settling these matters can facilitate patient involvement.
Dr. Leen Kawas Reinforces Ongoing Patient Recognition Efforts
In Dr. Leen Kawas’ influential Propel Bio Partners position, she leads partner companies through the multifaceted drug development process. Hence, Dr. Kawas can prod patient engagement in every relevant phase. She anticipates more chances to amplify patients’ collective voices.
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