In the pharmaceutical marketplace, time-to-market is crucial, with companies seeking viable strategies to help hasten the review process in the United States. Manufacturers must meet the requirements of the Food and Drug Administration Safety and Innovation Act or FDASIA for short, signed into law on July 9, 2012, which expanded the FDA’s authorities and strengthened the agency’s ability to safeguard and advance public health. The Advancing Breakthrough Therapies for Patients Act was incorporated into a Title of FDASIA in order to expedite the review process of novel therapies that are showing very promising results in early phase clinical trials. [1]


According to a peer-reviewed article published in Clinical Cancer Research, a breakthrough therapy designation (BTD) must meet specific criteria. First, the disease in question must be life-threatening and highly debilitating. Also, it must not have a standard of care treatment, or the standard of care treatment has failed to show efficacious clinical improvement. In addition, the product in clinical development must demonstrate substantial improvement compared to a therapy in the same class, or be superior to the current standard of care. Finally, the product must show promising or superior outcomes in early stage trials.[2] If the product meets these criteria, it may be granted the BTD.


Fig 1.0 – Overview Granted of Breakthrough Therapy Requests.

1.0 Impact on Oncologic Market Access
Breakthrough Therapy Designations (BTDs) have captured the attention of many hematology and oncology drug manufacturers. According to EP Vantage, 141 BTDs have been requested, and 37 applications have been accepted by the FDA since 2012.[3] The Center for Drug Evaluation and Research or CDER reported that 42% of all granted BTDs fall into the disease categories of hematology and oncology, as of December 2013.[4] Clearly, pharmaceutical companies specializing in these disease areas are taking notice of this accelerated market access program.

While 37 applications have been accepted, only four products have had NDA approval. Of these four products, three are indicated for oncology use. Obinutuzumab (Gazyva®; Genentech) and ibrutinib (Imbruvica®; Pharmacyclics/Janssen Biotech) are both indicated for chronic lymphocytic lymphoma (CLL). In addition, ibrutinib is indicated for mantle cell lymphoma. As for the oncology market, BTDs represent the best option for expedited market access. On average, a product takes approximately 88 months to gain market approval. With a BTD, the approval process can take as little as 53 months to gain market access.[5] For example, if a BTD is granted to a drug in Phase II, this gives the product the potential to reach market three years faster when compared to a product without a BTD. This kind of accelerated market access will lead to a greater length of market exclusivity, and give manufacturers increased time for sales and marketing.

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Fig 2.0 – For Serious and Life-threatening diseases, including cancer, the U.S. Food and Drug Administration (FDA) can grant specific designations to trial drugs that may help accelerate their time to approval. If the FDA grants accelerated approval, patients may receive a trial drug while ongoing Phase III studies confirm safety and efficacy. Source: FDA.

2.0 Pricing and Market Access Considerations for Oncology Products
The accelerated review of products with a BTD requires additional planning for pricing, reimbursement and market access (PR&MA). On most occasions, PR&MA planning is done during Phase III of product trials. With a BTD, a company must be ready to start this process during Phase II. According to Ram Subramanian, a pricing, strategy and marketing expert at Simon-Kucher & Partners, “Payers will find it more difficult to restrict access to a drug whose therapeutic value has been singled out by the FDA, and which may have already generated excitement among physicians and patients.[5]

In other words, an oncology product gaining a BTD can potentially ease payer scrutiny. Nevertheless, the company must still construct a compelling value proposition to present to these stakeholders.

An article written by Simon-Kucher & Partners published in OBR Oncology, identified three value drivers that will help develop a meaningful value proposition. Overall Survival (OS), Progression-Free Survival (PFS), and Safety Profile (SP) are the most compelling components of clinical results that payers want to see5. In the case of oncology products, OS and PFS data demonstrate efficacy to payers. Without this data, payers may be skeptical of the product and will require alternative and compelling evidence. A company may need to consider this challenge, and find ways to compensate and demonstrate value — in contrast to the lack of data generated by a product undergoing an accelerated review process.


3.0 Payer Uncertainty with Breakthrough Therapy
A certain amount of skepticism and uncertainty exists surrounding how BTD products undergoing clinical trials will be priced upon market approval. At a conference sponsored by Friends of Cancer Research in September 2013, an Aetna representative stated that payers are “nervous” about these products.[6] Michael Kolodziej, national medical director of Oncology Solutions, stated, “We recognize unmet need. We recognize the therapies that are being thrown at these diseases are not very effective. We would like much more effective treatment. We are not fools, however, and there are no new drugs to come to market that are cheaper than old drugs… So, we have to find the way to get the right drug to the right patient.”[6]

In addition, reimbursement surrounding products with BTD is uncertain. Primarily, payers are concerned about the prices that these new products will bring to the market.


4.0 Conclusion
For manufacturers, BTDs could bring potentially significant benefits to their oncology pipelines. With market approval, a product’s market access will significantly increase which could cause significant challenges when gaining support from various market access stakeholders. Payers in the oncology market, as well as all other therapeutic areas, are generally concerned about the prices that these novel therapies will demand.


March 31, 2015 | Corresponding Author Sophie Murdoch | doi: 10.14229/jadc.2015.3.31.001

Received: March 30, 2015  | Published online March 31, 2015 | This submitted editorial has not been peer reviewed.

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