“The Year in Review/The Year Ahead” is designed to spotlight notable topics in 2018 and assess potential impact for the coming year. The topics were suggested by The MAP Working Group, then matched with subject matter experts. Commentary and perspective clarify each topic, offer insight into the practical implications for medical publication professionals, and can serve as a guide to help us to assess, adapt, inform, and progress. This year’s topics are:
- The Emergence of Preprints in Medical Publishing
- FDAMA Section 114: A Key Guidance to Communicating HCEI to Payer Audiences
- The Evolution of Patient Involvement in Clinical Research and Publications
- Key Takeaways on Practical Implications of the EU General Data Protection Regulations (GDPR)
The opinions expressed within are the authors’ own and do not necessarily reflect the views of the authors’ employer.
The Emergence of Preprints in Medical Publishing
Tracy T. Cao, PhD, ISMPP CMPP™, Janssen Global Services, LLC., with Eric Y. Wong, PhD, MBA, ISMPP CMPP™, Janssen Therapeutics
Medical publication professionals understand that broad data transparency and prompt availability of data are important considerations for medical/clinical research because those factors allow stakeholders (such as healthcare providers, public health agencies, government payors, peers, and the public) to make informed, up-to-date, evidence-based decisions for patients. Prompt and transparent publication of data also naturally leads to greater collaboration and innovation, as peers will be able to engage in scientific exchange sooner and research fields can progress more quickly. As medical publishing continues to evolve to meet these needs, another channel for medical publishing is gaining prominence: the preprint.
What are preprints and why are they used?
Preprints are manuscripts posted by the author(s) in an open forum either before or in parallel with submission to a peer-review journal. Preprints allow authors to quickly share data and ideas with their peers and the public, to foster collaboration and receive early feedback on their work. Preprints are typically uploaded, at no cost, to a preprint server, and each preprint receives a digital object identifier (DOI), which allows the work to be cited. The major preprint servers include arXiv,1 which is run by Cornell University and predominantly hosts physical science manuscripts, and bioRXiv,2 which is run by Cold Spring Harbor Laboratory and hosts mainly biology manuscripts. A growing number of journals are allowing submitted manuscripts to be published as preprints in parallel with submission for peer review, and some journals will solicit open peer reviews of preprints. The adoption of preprints in the biology field has been slow compared with those in the physical sciences.
What is the impact of preprints on medical publications as it pertains to the pharmaceutical healthcare industry?
As preprints become more common in medical publishing, pharmaceutical and healthcare companies should have policies in place to manage how preprints based on company-sponsored research or investigator-initiated studies should be handled. This not only applies to whether company-sponsored research should be published as a preprint, but also whether data published as a preprint should be considered in company research projects. Many companies restrict the public dissemination of data in the preprint prior to peer-review publication. While early research and development work may carry risks, the legal, governmental, and regulatory implications concerning publishing medical research data as preprints are unknown, especially where conflicts of interest are concerned.
With the proposed initiation of MedRxiv3 to host medicine and health science preprints and mainstream journals offering preprints as part of the submission process, it will be interesting to see over the next one-two years whether pharmaceutical and healthcare companies embrace this medium to disseminate their research. Because preprints are not peer-reviewed, in contrast to academic basic science or theoretical research, there are additional concerns for the heavily regulated pharmaceutical industry that poor-quality, incomplete, or even wrong late-stage clinical trial data may get into the public domain and be misused or overblown to influence patients, consumers, or the market. Thus, there may be a “wait and see” approach for companies considering the publication of preprints, but publication professionals should be prepared for discussions with legal and compliance to determine what will be the path forward with preprint publishing.
FDAMA Section 114: A Key Guidance to Communicating HCEI to Payer Audiences
Robert J. Matheis, PhD, MA, and Nicholas J. Combates, PhD, ISMPP CMPP™, Celgene Corporation, with Paul A. Petruzzi, DLitt, ISMPP CMPP™; The Lockwood Group
Food and Drug Administration Modernization Act of 1997 – Section 114 (FDAMA 114) provides guidance on proactively communicating health care economic information (HCEI) to payers, formulary committees, and similar entities. An update to the guidance issued in June 2018, clarified how HCEI can be provided to payers and expanded the definition of who can receive this information. HCEI often includes reports of health economics and outcomes research (HEOR) and data based on real-world evidence (RWE) vs. results from randomized clinical trials (RCTs). An important consideration for medical publication professionals is the guidance on what information is to be included in the methodology (eg, meaningful analyses to payer audiences). FDAMA 114 had an immediate impact for medical publication professionals in three key areas:
- Renewed the need for, and interest in, HCEI publications and activities, as there are now expanded opportunities to communicate HEOR and RWE data.
- Created education and training opportunities as HCEI data often originate from a variety of sources (eg, clinical outcome assessments [COAs], study tables, and reports) that are different than working from a typical clinical study report. Further, the development of HEOR and RWE manuscripts must be tailored to the needs of the payer audience, which requires a writing style and approach that is different from publications that are aimed toward health care providers (eg, analyses, context, graphics, methodologies). “Consolidated health economic evaluation reporting standards” (CHEERS) may be a helpful resource for HCEI publication development.
- Provided additional opportunity for partner functions, such as outcomes liaisons, to interact with payers, thereby increasing the need for publication plans to include HEOR and RWE publications that consider this context. Emerging role of field-based outcomes liaisons also increased the need for cross-collaboration among medical publication and HCEI professionals.
The continuing impact of FDAMA 114 may also renew interest in “big data.” FDAMA 114 allows for big data analyses to inform treatment decisions. This will also prompt increased interest in personalized medicine considering that big data analyses may allow for identification of patient characteristics where interventions may have enhanced benefits. An additional implication from FDAMA 114 is that HEOR and RWE data must be generated early to allow payers to assess the value of a new regimen to make formulary and reimbursement decisions prior to product approval and, thereby, provide assurance that patients have access to the therapy. This contrasts with the traditional sequence of presenting phase 3 data, waiting for approval, and then developing HCEI data and publications.
As a result of the revisions to FDAMA 114, medical publication professionals need to be prepared for the resurgence of RWE and big data analyses to address the growing interest in HCEI publications. In addition, proactive training will ensure that medical publication professionals can partner effectively with authors and sponsors to develop HEOR and RWE publications.
The thoughts, views, and opinions expressed within this article, either explicit or implicit, are those of the authors and are not reflective of the perspectives represented by their employer, Celgene Corporation.
The Evolution of Patient Involvement in Clinical Research and Publications
Prof. Karen Woolley, PhD, ISMPP CMPPTM, Envision Pharma Group, with Jason Gardner, PhD, ISMPP CMPPTM, Complete Medical Communications [CMC]
The year in review
One of the most exciting developments during 2018 has been the broad-scale recognition that patients need to play an important role, not just in clinical research but also in medical publications and other communications. As tangible evidence of this, just look at the majority show of hands when audiences were asked, “Who thinks patients should be involved in medical publications?” at this year’s ISMPP European Meeting and Annual Meeting.
The driver for this momentum comes from a growing appreciation of why patients should be involved; patients bring a unique value to medical publications as authors, contributors, and reviewers that can no longer be overlooked. Along with this recognition has come a positive change to the medical publications environment; ie, the how. An increasing number of journals, such as Research Involvement and Engagement, require or encourage open-access plain language summaries. Journals are also updating their guidelines to reflect and encourage patient involvement. An example is The BMJ, which now requires authors to disclose how they have involved patients in all aspects of a study, including dissemination of results.1 Disclosing the involvement of patients in this way will enhance the relevance, credibility, and reach of a study, and the associated publication. As part of its “Patient and Public Partnership” initiative, The BMJ now also involves patients in both journal editor and peer-reviewer roles.
This was also a year where the need to enhance the science of patient involvement, particularly in publications, has been recognized. For example, patients can provide valuable insights on patient information needs, publication content, communication vehicles to enhance publications, and assessment of the impact of publications. Emerging evidence indicates that the benefits of involving patients in publications outweigh the potential harms.2 In today’s world, as patient leaders have reinforced, sponsors should not have to justify why they have involved patients in publications; they should have to justify why they have not!
The year ahead
If 2018 was the catalyst year for solidifying the why and how of patient involvement, 2019 will be the year of implementation. We need to evolve from the early adoption period to a time in which patient involvement is the mindset of the majority, and we need the tools to help us. A tool that is ready to use in 2019 is the Patient Engagement Quality Guidance tool from the not-for-profit organization, Patient Focused Medicines Development (Figure).3 But, we need more guidance and more tools that focus specifically on publications and which have been co-created with key stakeholders (including patients!). One widely used guidance tool, GPP3,4 did not address patient involvement in publications, but with opinions being gathered openly (#GPP4),5 the GPP4 authors will now have further evidence and insights to use.
The involvement of patients in medical publications and communications is bringing some unexpected benefits. Many of us embarked on careers in the pharmaceutical and medical communications industries to become closer to the patients who benefit from medical intervention; to make a difference. Now, with the growing association between medical publication professionals and the patient community, our roles are becoming more in touch with our ultimate customer – the patient! Helping patients through our work is important and motivating. Furthermore, it cements the patient–medical publication professional partnership in the publication ecosystem.
With the greater involvement of patients, medical publication professionals have an opportunity to build trust with patients, to work with patients (as both contributors and authors), and to empower the broader patient and public community through clear and credible communications. 2019 is going to be a great year for realizing the promise of patient involvement in publications, and it is our responsibility to do so ethically, effectively, and efficiently.
- The BMJ. Patient and public partnership. Available at: https://www.bmj.com/campaign/patient-partnership. Date accessed, 26 November 2018.
- Woolley KL, et al. Patient involvement in preparing clinical research peer-reviewed publications or results summaries: A systematic review. Poster presented at ISMPP 14th Annual Meeting 2018. Available at: http://bit.ly/2H8j2iM. Date accessed, 26 November 2018.
- Patient Focused Medicines Development. Patient engagement quality guidance. Available at: http://patientfocusedmedicine.org/wp-content/uploads/2017/11/PFMD_PE_Quality_Guidance.pdf. Date accessed, 26 November 2018
- Battisti WP, et al. Good publication practice for communicating company-sponsored medical research: GPP3. Ann Intern Med 2015;163(6):461–464.
- GPP4 Twitter hashtag. Available at: https://twitter.com/hashtag/GPP4. Date accessed, 26 November 2018.
Key Takeaways on Practical Implications of the EU General Data Protection Regulations (GDPR)
Karen Mittleman, PhD, ISMPP Global Transparency Committee Member (previously with Sanofi), with Erica Chevalier-Larsen, PhD, ISMPP CMPPTM, Cello Health Communications
The European Union (EU) General Data Protection Regulations (GDPR), which went into effect May 25, 2018, establish a wide-ranging set of rules protecting the use and storage of personal data. The regulation applies to companies that process data on people living in or working in the EU, regardless of whether that company has employees or offices based in the EU. Violation of GDPR carries substantial fines: €20 million or 4% of the company’s global turnover, whichever is greater.
The primary premise of GDPR is that an individual should have, and retain, control over his or her personal data. In certain cases, individuals must actively affirm that their data may be used or shared; passive or assumed consent may not be acceptable. When consent is required, the language must be easily understood and should not be buried within other terms of an agreement. Additionally, individuals have the right to be “forgotten,” that is, to request that their data be erased or not be used or distributed in the future.
Personal data from authors are frequently used to support medical publications activities. For instance, an author’s contact information accompanies abstract and manuscript submissions. Under GDPR, it is necessary to obtain an author’s consent for this information to be stored and shared. The implications of even simple activities, such as including an author’s email in a distribution list, should be considered. Questions remain about how to reconcile an author’s right to erase their data with retention of documents that are required for regulatory purposes.
While most patient information in publications is anonymized (ie, summary data) and, thus, does not fall under GDPR, in instances where the number of patients in a study is very small or the medical condition is very rare, patient information may be identifiable. In these situations, express permission from the patient to use or store these data would be required.
While publication professionals do not have to be experts on GDPR, they do need to be vigilant about safeguarding personal information. Over time, more experience with the activities impacted by GDPR will suggest more concrete guidance. In the meantime, the best approach is to take a proactive role, asking questions and, where needed, requesting permission for use and/or storage of personal data.
We realize that these are not the only topics of note from 2018, and that the impact of these topics will evolve in the coming year. The MAP Working Group hopes that this year-end review encourages assessment of 2018 accomplishments and offers insights to support 2019 planning. We look forward to your continued readership, comments, and support of The MAP newsletter.
The MAP Working Group
Eric Wong, chair; Erica Chevalier-Larsen, Jonathan Druhan, Jason Gardner, Anna Geraci, Paul Petruzzi, Lana Vegman