GVR Report cover Breakthrough Therapy Designation Market Size, Share & Trends Report

Breakthrough Therapy Designation Market Size, Share & Trends Analysis Report By Application (Oncology, Infectious Diseases, Rare Diseases, Autoimmune Diseases, Pulmonary Diseases, Neurological Disorders), And Segment Forecasts, 2018 - 2025

  • Report ID: GVR-2-68038-070-5
  • Number of Pages: 131
  • Format: Electronic (PDF)
  • Historical Range: 2015 - 2016
  • Industry: Healthcare

Industry Insights

The global Breakthrough Therapy (BT) designation market size was valued at USD 44.1 billion in 2016 and is expected to grow at a CAGR of 14.1% during the forecast period. The BT designation is granted to drugs that bring about substantial improvement in disease conditions compared to existing therapies during preliminary stages of clinical trials for the treatment of life-threatening diseases.

An increasing number of molecules gaining BT status, coupled with a rise in the demand for cancer therapies and drugs for rare diseases, is expected to propel the market over the forecast period. Increasing the prevalence of cancer and the rise in healthcare spending is also expected to contribute to market growth.

U.S. breakthrough therapy designation market

There has been a consistent rise in the number of drugs gaining BT status in the U.S. This is also expected to drive the number of applications submitted for the same, thereby contributing to market growth. An increase in the number of patients preferring innovative medicines in developed countries is also a major factor in boosting the market. The U.S., EU5 countries and Japan are three major markets for BT drugs. The recently expedited approval process in Europe, which is comparable to that of the U.S., is also expected to boost the number of approvals for BT drugs in the region.

FDA-approved drugs in the oncology and rare diseases segment are expected to dominate the market over the forecast period. However, several drugs that have been granted the BT status by FDA have failed to show fruitful results in late-stage clinical trials. For instance, SER 109, a Tylenol-sized capsule for the treatment of Clostridium difficile Infection (CDI), was granted BT designation in 2015. However, in 2016, it failed the placebo-controlled SER-109 Phase 2 ECOSPORTM clinical study for the prevention of recurring CDI. An increase in the number of similar cases is likely to restrain the market.

Rising healthcare expenditure in developing countries and the development of Intellectual Property (IP) laws are also among factors expected to drive the demand for innovative medicines for the treatment of life-threatening diseases. At present, developing countries such as India and China hold a larger share of the generic medicines market owing to unfavorable IP laws and relatively low healthcare spending. Countries such as Saudi Arabia and South Africa have relatively high spending power, owing to which they are lucrative markets for BT drugs.

Application Insights

The infectious diseases segment dominated the market in 2016. This can be attributed to an increase in sales of blockbuster drugs such as Harvoni and Sovaldi. In addition, a significant number of anti-infective drugs are gaining BT status. However, the decline in sales of blockbuster medicines, coupled with an increase in competition in the hepatitis C market, is expected to restrain the infectious disease segment over the forecast period.

The oncology segment is expected to surpass the infectious disease segment over the forecast period because of growth in the number of anticancer drugs gaining BT status. As of 2016, anticancer drugs were the largest of a number of drugs that gained BT status. The rise in the prevalence of cancer across the globe is anticipated to boost the oncology segment.

The rare disease segment is expected to exhibit the fastest growth over the forecast period. This segment does not include rare cancer diseases as they are covered under the oncology segment. Drugs for rare diseases receive the “Orphan Drug” designation if they are able to diagnose, treat, or prevent diseases that occur in less than 2,00,000 patients in the U.S. 50.0% tax credit is granted for the clinical research for these drugs.

According to the report titled “Insights into Rare Disease Drug Approval: Trends and Recent Developments” by U.S. FDA, rare disease noncancer approval accounts for 23.0% of total BT approval since 2013. Canakinumab (Ilaris) and Asfotase Alfa are among the highest selling medicines for rare diseases as of 2016. The number of molecules approved for rare diseases is expected to increase, and this is expected to boost the market over the forecast period.

Regional Insights

North America held the largest share of the global BT designation market owing to favorable IP laws and regulatory environment that rewards innovation through patent and data protection. High per capita income, improved access to healthcare, an increase in the prevalence of chronic diseases, and a rise in preference for innovative drugs by consumers are among major factors driving the market.

Global breakthrough therapy designation market

The European market is the second-largest market for BT drugs owing to better infrastructure and reimbursement policies. Consumers in this region prefer innovative medicines. According to the European Federation of Pharmaceutical Industries and Associations, diseases related to the cardiovascular system, cancer, respiratory system, digestive system, and nervous system are among major causes of death in Europe.

Asia Pacific and Latin America are expected to exhibit lucrative growth over the forecast period owing to the development of favorable IP laws and a rise in per capita healthcare expenditure in these regions. Preference for generic products in countries such as India and China is estimated to be one of the major challenges for the growth of innovative medicines.

Breakthrough Therapy Designation Market Share Insights

Some of the key players in the BT designation market are F. Hoffmann-La Roche Ltd; Gilead; Novartis AG; Pfizer, Inc.; AbbVie, Inc.; Janssen Global Services, LLC; Bristol-Myers Squibb Company; Eli Lilly and Company; Sanofi; Regeneron; Acadia Pharmaceuticals, Inc.; Boehringer Ingelheim GmbH; Amgen, Inc.; AstraZeneca; and GlaxoSmithKline plc.

Roche is one of the companies that produce the largest number of BT drugs in the market, and this is expected to significantly boost its revenue over the forecast period.

Report Scope



Base year for estimation


Actual estimates/Historical data

2015 - 2016

Forecast period

2017 - 2025

Market representation

Revenue in USD Million & CAGR from 2017 to 2025

Regional scope

North America, Europe, Asia Pacific, Latin America, & MEA

Country scope

U.S., Canada, U.K., Germany, France, Japan, China, Brazil, Mexico, Saudi Arabia, and South Africa

Report coverage

Revenue, competitive landscape, growth factors, and trends

15% free customization scope (equivalent to 5 analysts working days)

If you need specific market information, which is not currently within the scope of the report, we will provide it to you as a part of customization

Segments Covered in the Report

This report forecasts revenue growth and provides an analysis of the latest trends in each of the sub-segments from 2015 to 2025. For the purpose of this study, Grand View Research, Inc. has segmented the global breakthrough therapy designation market report on the basis of application and region:

  • Application Outlook (Revenue, USD Million, 2015 - 2025)

    • Oncology

    • Infectious Diseases

    • Rare Diseases

    • Autoimmune Diseases

    • Pulmonary Diseases

    • Neurological Disorders

    • Others

  • Regional Outlook (Revenue, USD Million, 2015 - 2025)

    • North America

      • The U.S.

      • Canada

    • Europe

      • Germany

      • The U.K.

    • Asia Pacific

      • Japan

      • China

    • Latin America

      • Brazil

      • Mexico

    • The Middle East and Africa (MEA)

      • South Africa

      • Saudi Arabia

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