Patient Worthy attended the EveryLife Foundation’s annual forum this past Tuesday in Washington, DC.
It was a full day workshop about why incorporating the patient perspective into the various phases of developing drugs and associated programs is vital to new rare disease treatment success. There has been so much talking about increasing patient engagement, organizing patient involvement, and quantifying patient input into the rare disease drug development process.
Kim McCleary, @KimtweetsDC of FasterCures played both historian and advocate at the workshop saying, “Historically, patients have been either subjects or patrons” she said, when it comes to the drug development process. “Traditionally we have thought about patient perspective as a zoo exhibit, in a captive and somewhat unnatural environment.”
Patient Worthy agrees that this approach is timeworn. “Patients are ready and waiting to increase engagement.” They have voiced over and over again that they are willing to accept greater risks in order to gain access to new approved treatments. They are no longer content to be passive patrons or subjects. They are increasingly well equipped to be partners and leaders from creation to cure. We were happy to hear that industry is listening.
If you are a patient, here are 5 truths to consider when raising your voice:
Medical literature is lacking and often has misinformation.
Information gathering is prone to misinformation at some level. The nature of experimental design, data collection, data analysis, and data presentation is often corrupted by impropriety, naiveté, and error. Even with supercomputers like Watson, that can process millions of existing proven and unproven data in a matter of minutes, and despite increasing FDA, industry, and physician interest accurate diagnoses of rare disease is difficult. All too often patients are training their doctors and nurses about their disease. You are the real experts, so speak up!
Information is limited and not very quantitative.
Although, surveys from patient interviews are difficult to measure, even when cross sectional, this kind of direct input is critical to further industry understanding about rare disease. The EveryLife Foundations’ President and Founder, Emil Kakkis, MD, Ph.D. believes and encourages that non-direct survey studies be routinely administered throughout the patient community so that more relevant endpoints and innovative tools for assessment of these diseases can be developed. Mixed methods of analysis are proving successful and will continue to come of age with inclusion of patient perspective.
An advocate from the Sickle Cell community asked the question: What is the most effective way industry wants to hear from patient population, how do you want it packaged, to be effective and drive change?
Dr. Kakkis collectively answered, “Gather the patient voice in a very large survey, a huge sample, and publish it. We want to see it in writing. Many factors of the disease are lost somewhere in translation. We need perspective to be solid, loud, clear, and focused. This type of data is comprehensive and powerful in that it can translate into concrete guidance.”
Risk and Responsibility is imbalanced.
One of the hardest areas to resolve is determining where the risk responsibility lies. Risk-benefit analysis is tricky and approval is not and cannot be the ultimate intent. There are too many variants and unknowns. Eliciting weights for benefits and harms from the patient perspective is crucial. More test parameters are needed to test against; efficacy and safety for example. There is no standard; the meaning of safe is not explicitly defined in regulation statutes. But patient perspective has enabled therapeutic context and content.
Chronic care differs from patient to patient.
Sally Okun, the VP of Advocacy at PatientslikeMe emphasized her organization’s understanding that living with a rare disease, and caring for someone living with a rare disease are two very different things. To better understand the patient and caregiver journey and provide measurable data to industry is what her organization aims to do. Marc Boutin JD, the CEO of the National Health Council addressed access, ensuring credible information, and raising the bar for the patient engagement movement.
Marc got us thinking. He said, “we all live for something.”
Each patient’s goals and personal circumstances are different, and so they require different levels of care. The chronic care model must continue to expand to account for this truth. Patients are explaining the impacts of their disease. You are telling yourHCP’s and caregivers what really matters, what you need, and how to help.
There is no disease too rare to deserve treatment.
Industry says the basic framework for patient perspective inclusion is up and running and patients are participating, full force. Here are just four quantitative examples of federal efforts that have come to fruition:
- Federal and pharmaceutical flexible clinical development programs have been enacted for research and development because FDA regulations allow for flexibility in scientific judgement. Today, there are four expedited rare disease drug development policies in play- all targeted to serious rare diseases and the subsequent unmet needs.
- Since 2012, the Office of Health and Constituent Affairs (OHCA) has become a central point of communication and education about the FDAs public health and regulatory activity. Hundreds of people are enrolled in its services. Some patient participants even serve as appointed government employees.
- Professional affairs and stakeholder engagement facets help conduct research and manage safe medication use projects. The Clinical Outcome Assessment Qualification Program was developed to determine whether or not a drug has demonstrated to provide treatment benefit.
- The Biomarker Qualification Program has provided a framework for regulatory acceptance of the use of biomarkers in drug development. By determining appropriate use standards, the program is working to further integrate these biomarkers into the process.
For rare disease patients the road to answers is long and frustrating.
The patient perspective remains underutilized, but data is being integrated throughout the discovery, development and delivery phases of the rare disease drug development process; and so it must continue. Patient and advocacy organizations alike must believe in the power of their voice; in the incredible weight their perspective carries in advancing medicine, its regulation and approval, and their own lives.
