It is all about the patient.

Thank goodness the whole health ecosystem is being reminded of this and is redoubling its efforts to put the patient in the center of drug development and regulatory review. However, the more meetings I participate in and hear about Patient Centricity and Patient Centered Drug Development, I’ve needed to take a moment and really think about what that means to the many parties involved. Every patient experience with disease is unique as is that person’s risk tolerances and benefit preferences for treatment options, so a great deal of care must go into defining an aggregate patient viewpoint –if innovators and regulators are going to use them effectively and efficiently.

To me, all of these patient-centric terms are different ways of saying essentially the same thing: treatments must focus on improving the quality of the life of a patient during the term of their illness and the person’s wellness beyond, if that is possible.

Though in the past we used well-educated providers to define disease improvement or slowing the advance of a disease, we are realizing that a key to improving care is to actually hear the patient and combine that insight with our understanding of the science:

  • How would the patient define what would improve their quality of life with an illness?
  • What are the risks he or she will tolerate to obtain the benefit of improving his or her quality of life?
  • How does the patient define improvement?

No matter what path an innovator or regulator takes, the patient and his or her improved health should be the focus of everything we do, and therefore patients have a key role that is increasingly front and center.

Opportunities for patients’ voices to be organized and heard are flourishing. Here are just some of the most obvious openings we see:

  • Legislative efforts:
    • R. 6, otherwise known as the 21st Century Cures legislation, recently passed the House. It includes a range of provisions aimed at enhancing the patient voice as an integral part of drug review and regulatory decision making. Now attention is turning to the Senate to see if and when the Senate HELP Committee will offer legislative drafts that will accomplish similar goals.
  • Regulatory Efforts:
    • On July 2, FDA released an updated plan on the disease states for which it will host “Voice of the Patient” meetings through FY 2017; this rounds out the agency’s PDUFA V commitment to hold 20 patient-focused drug development meetings over five years.
    • FDA published a document in early July that guides patient groups on how to petition FDA to participate in an external “Voice of Patient” meeting.
    • In May, CDRH issued a draft guidance on how to incorporate patient preference information in PMAs, HDEs and de novo requests
  • Negotiated User Fee Efforts:
    • Patient advocates are urging an expansion of FDA’s patient voice initiative in the upcoming PDUFA VI negotiations, to more effectively incorporate patient perspectives from the very start of the drug development process
  • Educational & Leveraging Efforts:
    • There is an unprecedented number of conferences focused on patient risk tolerances, patient preferences, and related topics. As just one example, the Drug Information Association (DIA) will hold a conference in mid-September on patient engagement in benefit/risk assessment across the product lifecycle.
    • FasterCures, an action think tank that works to build value add for advocacy groups by sharing learnings, started a new “Science of Patient Input” program in June; this effort is focused on expanding opportunities for patient perspectives to inform decision-making across the product lifecycle
  • Innovator Efforts: Many drug and device companies are launching their own internal and partnership efforts to focus on hearing the patient voice and incorporating it into their drug development plans

The approach of allowing a number of initiatives to seed and bloom to advance the patient voice and in development of treatments is important. The learnings gained from these various efforts will help the health care community gain insights into how to best serve individual patient needs, while also responding to the commonalities that emerge.

As I look across these initiatives, I see some common themes that may inform efforts to inject the patient voice into drug development:

  • Getting a broad swath of patient viewpoints together and finding their commonalities and differences has never been more important. No one group can do it alone.
  • Organizing the broad “community’s” insights in a way that can be effectively shared with regulators and product developers is key.
  • Finding the right mix of both qualitative and quantities methods of gathering patient input will be critical as patient voice efforts evolve.
  • Patients must find the capacity to work with regulators and industry leaders to help foster a better understanding of the patient’s experience, so that the questions being asked and the scientific and regulatory hurdles being identified are the right ones.
  • Innovators are realizing that better understanding the patient’s needs and preferences early in the pursuit of a treatment can be invaluable in researching, developing and bringing the highest value product to market. This is an opportunity that should not be squandered.
  • These will all inform efforts to develop the “science of patient input” – determining how to translate diverse and growing patient input into clinical trial endpoints, with the overall goal of developing therapies that respond to patients’ needs.

This is an exciting time to be working in the regulatory and drug development space; I think the next year or two will likely bring a lot of positive changes that will benefit patients across a lot of different disease states. So, let’s hear that unified, collaborative, and coordinated patient voice loud and clear.