The Target Product Profile—Your Blueprint for Drug Development

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 card adjustment programs: How do they affect patients, payers, and pharma?

“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.
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Common Protocol Template—Streamlining Protocol Implementation

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

Mapping a Successful Path to Label Optimization

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.
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How To: 5 Critical Steps to a Successful Scientific Symposium

5-steps-symposium-blog-graphic-v1Scientific 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

Why Hire a Professional Medical Writer?

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.

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Biosimilars—A New Frontier: Is the US Market Ready?

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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

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Making Sense of FDA’s Expedited Drug Approval Pathways and Designations – for the Non-Regulatory Professional

blog header linkedin - FDA Approval v6 blog sizeOne of the fundamental responsibilities of the US Food and Drug Administration (FDA) is to approve effective medicines for people who need them, while upholding high standards for safety. That mission also demands that the FDA work efficiently and not delay approval of life-saving medical advances. Today, the FDA is reviewing applications for approval of new medicines faster than ever, and that’s a welcome change from the status quo 25 years ago. In the era from 1962 (immediately following the thalidomide recall) to the early 1990s, FDA review times for a New Drug Application (NDA) or Biologic License Agreement (BLA) were often measured in years rather than months. In 1993, the standard review time (from NDA or BLA submission to decision) for a new molecular or biologic entity (parlance for a drug not previously approved for any other use) was about 28 months,1 and in some cases, approvals were delayed for many years.

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Implications of ICMJE’s Data Sharing Proposal

ProEd-ICMJE-Data-Sharing-Proposal-PostOn January 20, members of the International Committee of Medical Journal Editors (ICMJE) announced a proposal that would require the authors of clinical trial publications to share the deidentified individual patient data that support their published results within 6 months of publication. Announced in an editorial published simultaneously in multiple medical journals, this proposal is based on the belief that authors have an “ethical obligation to responsibly share data generated by interventional clinical trials.” It also reflects the broader agenda of the ICMJE to foster greater transparency and reduce the potential for bias. This new requirement will likely go into effect in 2016 and will affect any clinical trial that enrolls patients beginning 1 year after ICMJE adopts the requirement.

This proposal makes a lot of sense in the interest of transparency, but what does it mean for clinical investigators involved in research and the companies that sponsor that research? To quote the ICMJE authors, “enabling responsible data sharing is a major endeavor that will affect the fabric of how clinical trials are planned and conducted and how their data are used.” Continue reading

President Obama’s Cancer Moonshot 2020 Initiative: How Big Is the Challenge?

Apollo rocket blasting offWhen President Obama’s White House Task Force meets for the first time this week, it faces a lofty charge: “Let’s make America the country that cures cancer once and for all.” His proposed Cancer Moonshot 2020 initiative will provide much needed funding for research, will encourage collaboration between industry and academia, and will undoubtedly lead to important advances in our understanding of cancer and how to treat it.

Although this initiative is noble and well intentioned, calling for a “cure for cancer” oversimplifies the challenge and is based on an outdated understanding of the disease.

The public needs to understand that cancer is not one disease; it’s hundreds of different diseases, and it’s more complex than anyone imagined back in 1971 when President Nixon first declared war on cancer. Those involved in cancer research know that conquering cancer, in all its varied forms, is truly a global endeavor that will require a sustained commitment for decades to come. While a challenge to be the “country that cures cancer” stirs our patriotic spirit and conviction that American biomedical know-how can render cancer irrelevant in our lifetime, the reality is that meaningful progress will require international collaboration.

Are we, as a nation, prepared to take on that challenge and lead such a charge? The President has taken the first step, appointing Vice President Joe Biden to lead the White House Task Force. But now the difficult work begins. Can we balance competing priorities and sustain both our political will and financial commitment for the many years that such an effort will undoubtedly take? Continue reading