Biopharma Beat: Patient Engagement Throughout the Drug Development Lifecycle

Medical research and development, including that which is done by the biotechnology and pharmaceutical industry, is highly dependent on patients.  Historically, this dependency has centered on clinical trials, where products, after undergoing basic lab research looking into areas such as proof of principle, mechanism of disease and toxicology, then undergo formal trials with actual patients. 

Medical research and development, including that which is done by the biotechnology and pharmaceutical industry, is highly dependent on patients.  Historically, this dependency has centered on clinical trials, where products, after undergoing basic lab research looking into areas such as proof of principle, mechanism of disease and toxicology, then undergo formal trials with actual patients.  This is the only way to know whether a product has the intended effect and whether it is sufficiently safe and well tolerated to balance the risk-benefit scale.

This ‘patient centricity’ in clinical development has been evolving from mere patient participation in the study itself to patients actually being involved in the study design and data collection processes.  Furthermore, patient participation is, albeit slowly, penetrating every other lifecycle stage, from early discovery, through commercialization and access, and through post-marketing, real world clinical experience.

Patient engagement is no longer the sole domain of clinical trials. 

Recent announcements of collaboration between biopharma companies and groups such as PatientsLikeMe and 23andMe underscore this desire to tap into patient data and to engage with patients more meaningfully and very early in the epidemiology, genetic profiling, and disease understanding processes; this is in an effort to characterize the mechanisms of disease and potential drug targets.

Going to the other end of the product lifecycle, the involvement of patients in the commercialization of a product has become not only desirable but critical.  Companies now have a much better understanding of the ‘patient journey’ and therefore can better service providers, patients, and caregivers towards better patient outcomes.

If everything we do is for patient benefits, it would be arrogant to not include patients everywhere we can.

The trend towards greater patient centricity has been going on for some time with many success stories, but with much remaining to be done.  Companies live in silos and each silo has been considering patients in their own way, but collaboration among silos has been sporadic at best.  This means that, although similar, the information gleaned by each function is not as complete as it could be and sometimes may even be contradictory.  This creates deep issues and complications in discussions and decisions about: prioritization of outcomes, clinical relevance vs. scientific purity, decision and investment dynamics, and so on.  A decision that may make most sense because it is scientifically exquisite or intriguing may be a complete failure in the marketplace because of inadequate understanding of patient realities.  Conversely, a decision that is entirely based on what doctors are asking may be desirable but seem impossible to pursue and may not be tackled at all.  When managed care, marketing, clinical research, basic research, manufacturing and others start looking at patients in a coordinated and collaborative way, probably driven by a common framework, is the day this patient centricity will finally bring major benefits. 

We are not starting from scratch; there are some good efforts already.  For example, on occasion, early R&D asks Managed Care about access and reimbursement barriers so that they can design programs and shape products in ways that those barriers are overcome – decisions can be made about: indication sequencing, dosage forms, drug-delivery systems, dosage frequency, relevant end-points, product combinations, companion diagnostics, etc.

It is Important to always keep in mind that ‘patient engagement’ is a journey, not a destination. 

There are three cornerstones for biopharma companies to be truly ‘patient centric’ in an integrated way:

1. Common platform, shared education
1. Achieve a common understanding of the patient journey
2. Use common internal language and frameworks
3. If possible, have a common knowledge and insights-sharing platform.

2. Internal and External Collaboration
1. Collaborate on projects where possible and permissible
2. Share learnings from projects
3. Stay current. Paradigms can change quickly with new diagnostics and therapies
4. Include collaboration and dialogue with regulators
5. Evolve structures as well as roles and responsibilities

3. Effective Decision-making and Engagement
1. Take into account a complete view of the patient
2. Involve patients wherever possible, value the partnership
3. Take a multi-disciplinary approach
4. Apply effective decision-making rights
5. Develop and deliver relevant customer solutions and resources.