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Value and Market Access Considerations Often Missing From Trial Design

Meadow Green and Madelyn Hanson, PhD
Published: Tuesday, Jan 26, 2016
Most manufacturers examine the competitive landscape from both a clinical and a market access perspective when designing clinical trials to optimize a product’s commercial potential. A thorough understanding of efficacy and safety benchmarks within the current and future clinical landscape is absolutely necessary to assess a product’s potential to benefit patients. As the product progresses through clinical development, standard pricing and value research with a “set” target product profile will usually be undertaken. However, in the urgency to speed the drug to market, thoroughly integrating value and market access considerations into a product’s clinical program design is often neglected. To truly convey a product’s commercial potential, careful consideration and planning must be given to payers’ value measures. Having an evidence package at launch that satisfies payers’ value criteria will increase the likelihood that payers will reimburse the drug at a desirable price and enable patient access to innovative treatments.

Contributors of Pricing and Reimbursement Risk in Oncology

The number of agents in development for the treatment of cancer is double what it was 10 years ago. Simultaneously, agents are moving through development at an ever increasing pace. The availability of targeted therapies and now the dawn of immunotherapies have altered treatment pathways in multiple tumor types, and this is highly likely to continue for the next few years. Survival benchmarks are reaching points that were once thought unattainable and products are often more tolerable. More products are becoming available for patients harboring specific biomarkers, creating subpopulations within tumor types. This creates vast improvements in outcomes for patients and raises important issues. Growing competition within specific indications is allowing payers to manage oncology more thoroughly. But how will payers manage the different agents, indications, and costs? Clearly, companies have to be even more mindful of the commercial hurdles that lie beyond regulatory approval.

Figure 1. Use of Differential Tiering in Multiple Myeloma

Use of Differential Tiering in Multiple Myeloma

*Based on primary benefit design formulary.
Source: Pharmspectice; Pharmspective Policy View. http://pharmspective.com/

The importance of examining these questions in the context of a product’s clinical development plan can be clearly illustrated by pricing and reimbursement in Europe, where payers from different countries often have unique perspectives and data needs to take into consideration. Kadcyla® (trastuzumab emtansine, Genentech/Roche), which received European Medicines Agency (EMA) approval in December 2013, is an example of this. In France, the Commission de la Transparence (CT) assessment of Kadcyla had few criticisms of its trial. Determining that the correct active comparator was used in the trial, the product received a very positive Improvement of Medical Benefit assessment (amélioration du service médical rendu, or ASMR) rating (II), representing a “substantial improvement in actual benefit.” In contrast, in Germany, the Gemeinsame Bundesausschuss (G-BA) considered three different comparators. Because the clinical trial was designed to address a comparison with Tyverb® (lapatinib, Novartis) plus capecitabine, two of the three comparisons resulted in a designation of Kadcyla as offering “no additional benefit.”

Meanwhile, in comparison with Tyverb plus capecitabine, G-BA offered only a “hint of significant benefit,” predominantly due to the use of a progression-free survival endpoint based on imaging alone and no symptom data.1 In turn, Kadcyla’s price in Germany was discounted about 20%, and a 160 mg vial is almost €300 less than in France and the lowest in the 5EU.

Even in the United States, payer management beyond a prior authorization for a labeled indication is growing. For example, in the increasingly crowded market of multiple myeloma, around one-third of community oncologists reported an increase in payers asking them to change their prescribing for Revlimid® (lenalidomide, Celgene), Velcade® (bortezomib, Millennium/Takeda) and Pomalyst® (pomalidomide, Celgene), in particular.2 Reasons for these requests varied, but step edits and pathways are certainly evidence of increased payer management of this category. In addition, a sample of plans operating on the national, regional and state levels indicates that differential tiering is also being leveraged to manage products differentially. In fact, around one-third of patients across payer types are being affected by this type of management, and smaller plans operating within a region and in a single state are the most likely to manage in this way (Figure 1).


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