Breakthrough therapy designation was introduced by the FDA in 2012 to expedite the drug development process for promising novel therapies for serious or life-threatening conditions. In 2013, of 28 breakthrough therapy designations publicly announced, 12 were for oncology indications, and the first breakthrough therapy designations to gain regulatory approval (obinutuzumab [Roche/Genentech’s Gazyva] and ibrutinib [Janssen Biotech/Pharmacyclics’ Imbruvica]) were for chronic lymphocytic leukemia (CLL). These designations have been awarded for both hematological and solid tumor types, including multiple myeloma, non-small-cell lung cancer, malignant melanoma, and breast cancer. Breakthrough therapy designation therefore appears set to become an important accolade in oncology drug development and, as such, has received close attention from drug developers. Because the breakthrough therapy designation program is aimed at quickening drug review times, therapies may achieve earlier regulatory approval compared with standard review, a key advantage in increasingly competitive and fragmented oncology markets.

The breakthrough therapy designation program potentially impacts drug developers planning for novel therapies; if an agent gains regulatory approval more quickly, considerations such as appropriate clinical trial design to secure favorable market access and optimal positioning in the treatment algorithm will need to be considered at an earlier stage of drug development.

Questions Answered in This Report:

  • Physician and payer perceptions of oncology agents with breakthrough therapy designation. How do physicians and payers view currently approved breakthrough therapy drugs? How successfully have they penetrated the market? Have these agents achieved favorable formulary status? Are they preferentially prescribed? Do they experience off-label use?

  • Impact of breakthrough therapy designation on market penetration. Do attitudes toward breakthrough therapy designation differ between physician specialties? Could breakthrough therapy designation result in more rapid uptake? Will physicians preferentially prescribe a breakthrough therapy if available? Do physicians think that emerging breakthrough therapy drugs deserve this designation? How do physicians plan to prescribe emerging breakthrough therapy drugs?

  • Formulary placement of novel agents with breakthrough therapy designation. What are managed care organization pharmacy/medical directors’ (MCO PDs/MDs’) attitudes toward drugs with breakthrough therapy designation? How familiar are they with breakthrough therapy designation? What prescribing controls have been placed on existing breakthrough therapies, and will they change in the future? What prescribing controls are likely to be placed on emerging breakthrough therapies? What will their tier placement be?


This U.S. Physician & Payer Forum report contains insights from a survey of 50 medical oncologists, 50 hematological oncologists, and 30 MCO directors regarding the advent of breakthrough therapy designation in the United States. We explore the factors that shape current and future treatment patterns as well as reimbursement trends for therapies with this regulatory designation.

Markets covered: United States.

Primary research: Online survey of 100 physicians (50 medical oncologists and 50 hematological oncologists) and 30 MCO directors.

Author(s): Amy Duval, M.Res.

Related Reports

Multiple Myeloma - Landscape & Forecast - Disease Landscape & Forecast

The multiple myeloma therapy market continues to grow, fueled by the disease’s increasing incidence and long treatment durations. The availability of premium-priced branded agents prescribed...

View Details

Malignant Melanoma - Landscape & Forecast - Disease Landscape & Forecast

The introduction of immune checkpoint inhibitors Keytruda (Merck & Co.), Opdivo ± Yervoy (Bristol Myers Squibb), and Tecentriq (Roche / Genentech; United States only), as well as BRAF/ME...

View Details

Breast Cancer - Landscape & Forecast - Disease Landscape & Forecast

The breast cancer therapy market is rapidly evolving and presents new commercial opportunities for drug developers. CDK4/6 inhibitors (Pfizer’s...

View Details

Breast Cancer | Disease Landscape and Forecast | G7 | 2020

The breast cancer market is rapidly evolving and presents new commercial opportunities for drug developers. CDK4/6 inhibitors (Pfizer’s Ibrance, Novartis’s Kisqali, and Eli Lilly’s Verzenio / Verze...

View Details