Patient Experience and Why it Matters


You may have noticed that one of the recurring themes at Chion Foundation is that we constantly ask you to document your experience with pitolisant. Our preference is that you do that in a systemized way via TREND Community. (https://trend.community/). We ask that you record weeks of baseline data (before pitolisant) and weeks/months of your experience with pitolisant.

We ask this for an obvious reason. We want to know if pitolisant is helping our community and if it is, how it is helping our community. We also want to know if anyone is experience adverse events and, if so, what those events are. These documented experiences will be stripped of identifying data and shared with prescribers and other stakeholders.

We also anticipate sharing this information with the Food and Drug Administration (FDA).

Traditionally, drugs that are approved by the FDA must go through many layers of clinical trials in order to be approved. Typically, these trials must take place through an FDA-approved protocol in the United States or in another country. The gold standard trial is the phase III, randomized, double-blind study which is used to gather data on the safety and efficacy of medical products. Such phase III trials are very expensive and time consuming. They often occur in highly specific patient populations and in very controlled clinical settings. Unfortunately, in the rare disease world (ie., PWS and narcolepsy), such clinical trials are rare because patients are few and far between and may be difficult to recruit.

The United States government and the FDA have recognized this difficulty and are making efforts to remedy this problem. The 21st Century Cures Act was signed into law in December 2016. The Cures Act specifies that patient experience data may be submitted to the FDA for review. This data may include data on patients’ experience with a disease as well as patients’ preferences with respect to treatment.

The Cures Act also requires the FDA to develop a plan to collect and use patient experience data in drug development. The intention is for the FDA to use patient experience data in its benefit-risk assessment framework so that it can inform decision-making. The Cures Act also re-emphasizes the need to encourage treatments for rare pediatric diseases. Additionally the Orphan Drug Act has been expanded to include not just clinical and preclinical studies in rare diseases, but “prospectively planned and designed observational studies and other natural history analysis.”

Real World Data

Many clinical researchers would like to be able to successfully integrate clinical research into real world settings by accessing data from electronic health records, electronic devices, and social media. The Cures Act shepherds in a new emphasis on this real world data, with the understanding that such data have the potential to complement data obtained from traditional clinical trials. Experts note that such real world data have the potential advantages of helping researchers answer questions about drug treatment effects and outcomes in an efficient and cost-effective manner. There are challenges, of course, to using data that may be collected in less than scientifically rigorous settings and that is the reason why we believe that we must be as diligent as possible as we collect our patient experience data. The FDA is currently evaluating whether they can use real world evidence to help support the approval of new indications for an approved drug.

The New England Journal of Medicine recently published an article written by leaders at the FDA that discussed some ideas on how this real world evidence can be incorporated into drug approval. The authors wrote that, “We believe that real-world evidence can be used across a wide spectrum of research, ranging from observational studies to studies that incorporate planned interventions, whether with or without randomization at the point of care.” The article described the challenges and opportunities of real world data. In particular, it emphasized the importance of having the data address defined populations using defined methodologies. We, at Chion Foundation, do not yet know how these challenges will be resolved or the framework that the FDA will use to incorporate real world date in drug approval. We do know that we would like to position the PWS community to take advantage of these changes as they happen.

We also know that pitolisant has successfully been evaluated in phase III trials in Europe and been approved as a safe and effective treatment for narcolepsy with/without cataplexy. We understand that the manufacturer intends to bring the drug to market in the US, presumably for a similar indication. We want to do everything in our power to help this happen quickly.

The Bottom Line

Our goal is to use TREND to systematically document the effects of pitolisant in individuals with PWS. We hope that (if demonstrated effective in individuals with PWS), this data can be used in the near future to support approval of pitolisant in the United States and possibly even support a specific indication of pitolisant for Prader-Willi Syndrome. If we are able to achieve this, then every individual in the United States would have access to pitolisant. Insurance would also likely cover the cost of the medicine.

So, please, we urge you, if you think you might explore the use of pitolisant in your child, enroll in TREND Community and start collecting baseline data now. You can request an invitation by emailing: interested@trend.community


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The Chion Foundation is a nonprofit, tax-exempt charitable organization under Section 501(c)(3) of the Internal Revenue Code. Donations are tax-deductible as allowed by law (Tax ID 81-1167193).

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