Towards Regulatory Standards for Exploiting Real-world Data from Social Media in Patient-focused Drug Development

Owned by miffy wilson (Unlicensed)
Last updated: Feb 18, 2022
4 min read

Draft Title: Towards Regulatory Standards for Exploiting Real-world Data from Social Media in Patient-focused Drug Development

Date: December 2021

Who to Contact: projectenquiries@pistoiaalliance.org

Patient-focused drug development (PFDD) is defined as „a systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation“1. Consequently, in addition to clinical evidence from randomized controlled trials, decision making in PFDD is increasingly fueled by real-world evidence (RWE) in order to understand the burdens and needs patients are facing in their real-life context, rather than under clinical or laboratory conditions.

In current practice, real-world evidence is usually gathered from data sources such as electronic health records, claims databases, as well as surveys or interviews with medical practitioners or patient focus groups. From our perspective, these approaches still do not unfold the full potential to integrate the views of patients „as experts in what it is like to live with their condition“2 into PFDD, as they are either limited to capturing the patients’ experience only indirectly through medical practitioners as mediators, or they are hardly applicable at large scale, and if so, only at the cost of high expenditures in terms of time and money.

Against this background, social media poses an interesting and promising alternative to listen to the patients’ voice directly and in real time, thus capturing first-hand patient experience data in large volumes and in a fully unsolicited manner. The potential of social media data for carrying out online patient experience research has recently been acknowledged by the FDA in their guidance on „Collecting Comprehensive and Representative Input“ for PFDD3. In this guidance, the FDA is taking a balanced assessment by explicitly emphasizing the potential usefulness of social media data to be included in regulatory submissions, while also pointing out some potential limitations.

The potential of online patient experience data as a source for RWE generation is also increasingly recognized in the industry, as observed in a preliminary literature review study: Since 2015, more than 30 publications are listed in PubMed which describe social media listening approaches in the context of patient-focused quality-of-life research. These studies address various phases in the pharmaceutical value chain, thus corroborating the potential of online patient experience research from social media sources as a strategic instrument to be applied at various stages along the PFDD process:

  • Contribute to understanding the value of a drug (incl. disease burdens, unmet needs, patient-reported outcomes) from the perspectives of patients, HCPs, insurances and regulatory agencies (Cook et al., 2018; Cotté et al., 2020; Wolffsohn et al., 2020; DelesetreLevai et al., 2021)

  • Monitor postmarket safety and effectiveness (Kalf et al., 2018)

  • Generate data to understand and support treatment decisions and to develop guidelines and decision support tools for use in clinical practice (Booth et al., 2019; Reuter & Lee, 2021)

  • Support the design of clinical trials and/or PRO instrument development (Patalano et al., 2020)

These studies still rely on substantial amounts of manual work and/or disease-specific settings; therefore, they are not readily scalable to other diseases or other online populations. In order to leverage the huge potentials of online patient experience research, automated solutions should be pursued and integrated as a flexible strategic tool into the PFDD chain. As prerequisites of such investments into solution development and integration, remaining uncertainties regarding regulatory validity and acceptability of the results from online patient experience research need to be mitigated (among others):

  • How can patients be validated based on their self-reported online narratives?

  • How can relevant patient characteristics be reliably confirmed from self-reported online patient narratives?

  • What are sufficient methodological criteria in order to avoid biases in data selection and enable representativeness as much as possible?

  • To what extent can online patient experience research yield comparable or complementary results, relative to established research instruments?

  • How can data saturation or other criteria from qualitative research be applied in online patient experience research?

Therefore, we propose to collaborate on strategies to resolve (some of) the above issues. The goal would be to translate these resolution strategies into common industry-wide standards and propose them to regulatory authorities (FDA and EMA) for approval. This will create trust and reliability to an extent that all industry players need in order to implement online patient experience research as an integral anytime tool into PFDD chains. The benefit for participating members would be that a highly innovative and inherently scalable approach to make the patient voice actionable in PFDD would be facilitated for broad strategic use.

References

Booth, Alison et al. (2019): Using Social Media to Uncover Treatment Experiences and Decisions in Patients with Acute Myeloid Leukemia or Myelodysplastic Syndrome Who Are Ineligible for Intensive Chemotherapy. Patient-centric Qualitative Data Analysis. J Med Internet Res 21(11): e14285.

Cook, Nigel S. et al. (2018): Patients’ Perspectives on COPD. Findings from a Social Media Listening Study. ERJ Open Res 5: 00128-2018.

Cotté, Francois-Emery et al. (2020): Exploring the Health-related Quality of Life of Patients Treated with Immune Checkpoint Inhibitors. Social Media Study. J Med Internet Res 22(9): e19649.

Delestre-Levai, Irisz et al. (2021): Patients’ Perspectives on Bronchiectasis. Findings from a Social Media Listening Study. ERJ Open Res: in press.

Wolffsohn, James S. et al. (2020): Social Media Listening to Understand the Lived Experience of Presbyopia. Systematic Search and Content Analysis Study. J Med Internet Res 22(9): e18306.

Kalf; Rachel R.J. et al. (2018): Use of Social Media in the Assessment of Relative Effectiveness. Explorative Review with Examples from Oncology. JMIR Cancer 4(1): e11.

Patalano, Francesco et al. (2020): Gathering Structured Patient Insight to Drive the PRO Strategy in COPD. Patient-centric Drug Development from Theory to Practice. Adv Ther 37: 17-26.

Reuter, Katja & Lee, Delphine (2021): Perspectives toward Seeking Treatment among Patients with Psoriasis. Protocol for a Twitter Content A