Cytel Blog

At the 2019 Challenges in Rare Diseases Clinical Trials Symposium and East training, Cytel partnered with Alexion to bring together expertise from academia and industry. David Kerr, Director of DMC Services at AXIO, was among the notable speakers and his talk “Data Monitoring Committees – Behind Closed Doors” covered general considerations and options that the DMC has when reviewing the data presentations during their closed sessions. He presented four specific case studies that highlighted the data provided to the DMC from meeting to meeting and discussed how the DMC arrived at their recommendations for each meeting. We had the opportunity to sit down with David and speak about Data Monitoring Committees, understand their proceedings and talk about his presentation at the symposium.

Author: Michael S. Paas, Market Access & Commercialization Expert, Executive at AbbVie and Guest Author at Cytel

In my previous post, I established the importance of market access strategy in the clinical development process and why emerging biotechs cannot afford to overlook it.

Click on the button if you missed Part 1 or want to revisit it.

It is imperative to start developing your strategy with market access due diligence and value story creation early in the product development (at least by phase II of drug development or the proof-of-concept phase of a device development). Impactful market access planning involves designing your clinical programs so they sufficiently address the evidence needs of market access stakeholders, patients, and regulators. This means demonstrating the value of your therapy to payers and HTA agencies by generating compelling and robust clinical trial data against appropriate comparators, augmented where possible and helpful with real-world evidence, health economic arguments, economic models and/or relevant patient-reported outcomes.

Market access strategy is an integral part of the clinical development process to ensure success in global healthcare markets and vital access to patients

Author: Michael S. Paas, Market Access & Commercialization Expert, Executive at AbbVie and Guest Author at Cytel 

In clinical development, it’s easy to underestimate the critical role that market access plays in the commercial success of a product. Understandably, organizations instead focus on the regulators, and the steps needed to secure eventual approval. However, ensuring the development plan is aligned with a well-planned market access strategy is essential to help the therapy actually reach the intended patients and realize its true commercial potential following regulatory approval. This is particularly crucial considering that payers and other key stakeholders demand compelling information on a drug’s/device’s value as well as justification of its pricing.

In this four-part blog series, I will address what can be done to optimize your market access planning, and why all that effort is really worth it.

Cytel is delighted to have Kannan Natarajan speaking at the “Complex Innovative Trial Design Symposium and East User Training” on November 6 in Boston, MA. We got a chance to sit down with Kannan and talk about his career in statistics, the changing role of statisticians, his views on evolving statistical thinking, estimands and relevance of technology in the context of rare diseases.

 
October 3, 2019 was an important day for the ADaM team as it marked the release of the ADaM Implementation Guidance (Ig) 1.2. Download the new guidance from the CDISC website here.

In 2018, several previews of the new Ig were made at CDISC Interchanges all around the world and at PhUSE conferences, it was only a few weeks ago that the final version was released. At first glance, the new Ig does not seem to contain a lot of new concepts and ideas. However, a critical and in-depth review clearly shows the efforts of the entire team to release this new Ig. Creating a new standard or releasing a new version of an existing standard is not an easy job. You need to ensure that you do not introduce anything that contradicts any of the existing CDISC standards. This includes, not only new variables but also any changes in existing sentences or adding entirely new sections that may cause misinterpretations or discrepancies. Moreover, every standard team member comes with their own background, company needs, and specific indication needs. It is not always easy to propose a solution that can satisfy everyone on the team.

A disease is generally considered to be rare if it affects one patient per 200,000 people ⁽¹⁾ and most rare diseases affect far fewer than this. However, collectively rare diseases are relatively common, affecting 350 million patients worldwide ⁽²⁾. The path to diagnosis for these patients is often a long, difficult battle and even once the diagnosis is made, it is likely there will be no suitable treatment available.  For 90% of rare diseases, there is no approved therapy ⁽²⁾. There is, therefore, a pressing need to develop new, effective therapies that can bring hope to rare disease patients. However, the clinical development environment for life-threatening, rare diseases is fraught with challenges. By their very nature, rare indications have few patients and limited sample size. This scarcity of patients also results in a lack of available information and knowledge about the disease from the best endpoints, to the treatment effect size or the variability of response between subgroups.

"If you went to bed last night as an industrial company, you're going to wake up today as a software and analytics company," CEO of GE, Jeff Immelt

We are living in a new digital world which is evolving every day. Both personally and professionally, we rely on technology for many of our routine activities, and examples of digitization are prevalent across industries. Retail is a big example of how several chains have moved from physical stores to creating an online presence. In some years from now, people perhaps won’t have to learn driving as more self-driven cars will hit the road. Healthcare does not fall far behind in this race towards digitization.

In this blog, we will examine some of the different ways that digitization is set to shift the drug development paradigm.

In place of collecting data from patients recruited for a trial who have been assigned to the control or standard-of-care arm, an external control creates a comparator arm using either real-world data-sets such as electronic health records or previous clinical trials.  The external control offers a practical, effective way to leverage real-world evidence and has been applied in regulatory approvals. In this blog, we share an illustrative example of how we can help customers in this emerging area of interest.

This article was originally published as part of a series by pharmaphorum in association with Cytel and is reproduced with their permission.

Ever felt like you ended up somewhere unexpected, not quite knowing what path you took to get there? This is the situation pharma companies can face without the right input from statisticians on clinical trial design – and the results can be very costly. As part of a series of articles in association with Cytel, pharmaphorum spoke with Natasa Rajicic about the vital role statisticians play in improving trial design and preventing expensive mistakes and trial failures.