As Pfizer, Moderna, and other pharma
companies prepare to seek emergency authorization for their SARS-CoV-2
vaccines, the FDA has laid out a roadmap designed to ensure appropriate
scientific rigor and help engender public trust. That plan was the subject of a
special meeting of the Vaccines and Related Biological Products Advisory
Committee (VRBPAC) on October 22, 2020, where experts discussed 2 critical FDA
guidance documents that provide a blueprint for development and approval of
SARS-CoV-2 vaccines. That blueprint is at the center of a massive government
effort to quickly and safely speed vaccines to the American public and bring
the pandemic to an end.
Members of the VRBPAC, with expertise in
infectious disease, epidemiology, and vaccine development, focused on issues
around the FDA standards for safety and effectiveness that will support
Emergency Use Authorization (EUA) of vaccine candidates. They discussed the
need to continue the phase 3, randomized, placebo-controlled trials to
completion after an EUA is granted. They considered how the vaccines will be
rolled out to the American public, and they raised concerns about whether the
public will embrace the vaccines and roll up their sleeves.
Authorship is a hot topic in the scientific and medical publishing world. Who qualifies as an author? Who is the senior author? What are the responsibilities of the corresponding author? Opinions vary across disciplines and cultures. Whereas medical publications generally follow the recommendations of the International Committee of Medical Journal Editors (ICMJE; http://www.icmje.org/icmje-recommendations.pdf),1 academic publications may follow other guidance, or none at all. Is there a way to impose universal authorship criteria and quantify the work of authors so that their actual contributions can be tracked, giving them more than just their name on an article in the modern publish-or-perish environment?
A recent article by McNutt et al2 in Proceedings of the National Academy of Sciences of the United States of America (PNAS) seeks to create a framework for doing just that. As part of the global push toward greater transparency, with the goal of increasing integrity and trust in scientific publications, this article proposes that journals develop standardized authorship requirements and reporting, documented through ORCID identifiers (https://orcid.org) and the CRediT system (http://docs.casrai.org/CRediT).
As of July 1, 2018, manuscripts submitted to International Committee of Medical Journal Editors (ICMJE)-member journals must be accompanied by a data sharing statement. What is the new requirement, how did it evolve, and what does it mean for data sharing?
In January 2016, the ICMJE proposed that authors of all clinical trial manuscripts published in member journals share de-identified individual-patient data (IPD) underlying their results within 6 months of publication.1 The proposal included data in tables, figures, appendices, and supplemental materials. The ICMJE invited comments on its proposal and a firestorm ensued. Although many individuals and groups applauded the ICMJE proposal, others raised legitimate concerns. Some were concerned about inappropriate analyses of data without statistical rigor, and authors were concerned about competition and losing control and/or credit for their work. Others voiced concerns about the practical aspects of how to share the huge amounts of data generated by some studies, particularly large, phase 3, randomized trials. Still others raised persistent concerns about patients’ right to privacy, particularly in the rare disease setting, where, despite de-identification efforts, patients still might be identifiable. Continue reading
When utilized to its full potential, the Target Product Profile (TPP) is a dynamic, living document that ensures all stakeholders—clinical, regulatory, quality and manufacturing, commercial, market access, and medical affairs—are working from the same blueprint. Unfortunately, the TPP often has a bad rap within industry because many people think it is too rigid for today’s drug development environment. But often that reflects a failure to truly collaborate or a tendency to let the TPP get stale. To be effective, the TPP must be continually updated based on changes in the data and the competitive landscape. When companies take a balanced approach to developing the TPP and have a dynamic process that allows them to monitor and adapt it, as needed, they build agility into their drug development program that allows them to make critical go/no-go decisions or course corrections when necessary.
By using the TPP to ensure everyone is on the same page, drug developers can avoid costly delays when, for example, manufacturing isn’t ready to scale up to commercial production when the phase 3 data comes in ahead of schedule. Keeping a close eye on the evolving therapeutic landscape helps the development team anticipate what data will be needed to support labeling claims that may serve as a key differentiator from the competition and provide added value in the marketplace. So let’s look at how a dynamic TPP—one that is proactively updated—can help achieve the critical success factors introduced in the last installment. Continue reading
“Co-pay cards” (or “co-pay coupons”) are financial assistance programs from drug manufacturers (pharma) that drastically reduce the out-of-pocket (OOP) costs for someone who needs an expensive medication. These programs are controversial:
- Pharma and patients believe that these programs allow sick people to afford the medications they need.
- Healthcare payers (ie, insurance companies or their pharmacy benefit managers [PBMs]), however, regard such programs as schemes that circumvent their cost-management techniques (such as formulary tiers and patient cost-sharing). This is because with no financial impact from OOP costs, doctors and patients could decide to use more expensive drugs than the ones preferred by the insurance plans (eg, generic or well-established branded drugs).
Because of these opposing points of view, payers have tried (with limited success) to disallow co-pay cards if possible, and pharma is developing new ways to circumvent payer controls. This “cat-and-mouse” game has antagonized payer-pharma relationships as each are pursuing different goals: payers are trying to manage expensive medication use, whereas pharma is trying to maximize their sales and prescription volumes.
Study protocols are required for every clinical trial. Approximately 20,000 are submitted and posted to www.clinicaltrials.gov every year1—each one different. The format and core content can vary from sponsor to sponsor, costing the US Food and Drug Administration (FDA) time and resources to interpret, review, and ultimately, approve each uniquely complex protocol. This process, as it stands, slows down progress for new drug development. Clearly, there is a need to accelerate the pace at which protocols are approved so that new clinical studies can be initiated. In a world where technology continues to offer a platform for efficiency and accuracy, the development of the Common Protocol Template (CPT) is a welcome addition to the medical field. Common Protocol Templates can lead to faster review time, simplified trial startup, and prompt execution of clinical trials. Although the use of a CPT is not required for all new clinical trials, it is only a matter of time before its use becomes commonplace in drug development. Continue reading
Bringing a drug to market is a long and expensive process. An analysis by the Tufts Center for the Study of Drug Development estimated the total cost of development from discovery to commercialization at $2.6 billion over the course of about 10 years (based primarily on big pharma companies). This represents more than a 10‑fold increase since the 1970s, when a drug could be developed from bench to bedside for less than $200 million (Figure 1).1 Others estimate the cost to commercialize a drug to be much lower (< $1 billion when they consider small biotech companies),2 yet it is generally accepted that the cost of drug development is on the rise. A major driver of those rising costs is the money spent on drug candidates that never make it to market because of safety concerns or lack of efficacy. The bottom line is that there is no room for costly mistakes, miscalculations, or inefficiencies in the drug development process.
Scientific symposia at medical conferences are a great way to educate physicians on the current treatment landscape and on how new agents can improve patient care. But your symposium is often competing with many others for attendees’ limited time and attention. If you want your content to be seen, you need to find creative ways to draw an audience and offer a meaningful educational experience. Since 1991, ProEd Communications has helped medical affairs teams create novel and engaging scientific symposia. Success requires creativity, diligent preparation, and outstanding organizational skills. Below are my top 5 steps to creating a successful symposium.
1. Develop a theme that will capture attention
A symposium can only be successful if it reaches its target audience, and that requires capturing the interest of congress attendees. So the number one success factor is to come up with a creative theme that will grab the attention of potential attendees. Continue reading
The updated Good Publication Practice guideline (GPP3) acknowledges the legitimate role of medical writers in helping authors with compliant, complete, and timely development of publications, “particularly when authors have limited time or lack knowledge of publication ethics and current publication and reporting guidelines.”1 Indeed, most authors (>84%) recently surveyed value the assistance provided by professional medical writers, particularly in editing manuscripts and ensuring conformity with reporting guidelines.2,3 In addition, emerging evidence suggests that the use of professional medical writers may enhance publication quality.1 So what impact does the medical writer really have on the quality of the publication? That is the question asked by William Gattrell and colleagues in their paper recently published in BMJ Open.4
Their cross-sectional study examined the relationship between medical writing support and the quality and timeliness of randomized controlled trial (RCT) reports. Completeness of the manuscript was assessed based on a predefined subset of 12 typically underreported items from the Consolidated Standards of Reporting Trials (CONSORT) checklist. Time from manuscript submission to editorial acceptance was also measured, as was the overall quality of written English as assessed by peer reviewers.
This post has been updated based on FDA approval of Celltrion’s Inflectra™, a biosimilar Remicade® (infliximab) for treating arthritis and other inflammatory diseases, on April 5, 2016. Inflextra is the second biosimilar approved by the FDA and the first to be granted multiple indications. This is an important milestone for biosimilars in the United States.
Since the first FDA approval of a biosimilar in March 2015, biosimilars have quickly become one of the hottest topics in the US pharmaceutical industry. Why is this such a big deal, and is the US market ready?
Biosimilars are alternatives to innovative biologic therapies (known as “originator” products) such as Humira® (adalimumab) and Enbrel® (etanercept) for treating rheumatoid arthritis (RA) and psoriasis, or Herceptin® (trastuzumab) and Avastin® (bevacizumab) for treating cancer.
Biosimilars promise to increase access to these life-changing biologic therapies by reducing costs, analogous to the cost savings of generic drugs over brand-named drugs. In fact, biosimilars could save American consumers about $250 billion over the next 10 years.1