1. Home
  2. > Pharmaceutical Market Trends
Vital Signs The Analyst's Perspective - April 2016 Issue 

Vital Signs The Analyst's Perspective - April 2016 Issue 

  • May 2016
  • 6 pages
  • ID: 3898037
  • Format: PDF
  • Frost & Sullivan


Table of Contents

Trends in Orphan New Molecular Entities – Half are First in Class Source: Health Affairs, March 2016

Orphan drugs are defined as “drugs that are used to treat rare diseases or conditions which (A) affect less than persons in the United States or (B) more than in the United States where there is no reasonable expectation that the cost of developing and marketing in the United States for such a disease or condition will be recovered from sales in the United States for such a drug.”1 In 2015, a total of drugs were approved by the FDA, of which , or %, were for orphan indications. Over half of the new orphan indication approvals were oncology related.

The Analyst’s Perspective by Barbara Gilmore, Senior Industry Analyst, Life Sciences According to the FDA, once an orphan drug designation has been granted, the company developing the drug is eligible for numerous financial incentives, including but not limited to tax deductions for clinical trial expenditures, waiver of FDA user fees for the orphan indication, and seven extra years of exclusivity once the drug is approved for the orphan indication.

The FDA has recently reviewed trends in orphan new molecular entities (NME) for the period 1983–2014. From the time Congress passed the Orphan Drug Act in 1983 until 2014, the FDA approved drugs as NMEs. According to the FDA economist Kathleen Miller, over were first in class and % received a priority review. In total, the group of approved drugs did address the unmet medical needs of many diseases. Trends that emerged from the FDA study indicated that although orphan approvals have spanned all therapeutic categories, there has been a much greater focus on oncology therapies ( %) in recent years, followed by metabolic disorders ( %), infectious diseases ( %), neurological disorders ( %), and hematological disorders ( %). It was speculated that the advances in the identification of genomic subgroups in the oncology tumor milieu were responsible for driving the increase in orphan oncology NMEs.

There is growing belief in the reimbursement segment of the healthcare industry, as well as in the US Congress, that the costs of drugs are out of control, resulting in an enormous drug spend that is benefiting only a fraction of the population. When the prices for approved drugs garner blockbuster status, it is an understandable concern. It is safe to conclude that revenues generated from orphan drugs will be scrutinized as more of these drugs are approved as secondary indications, where a non-orphan indication already is in place.

Get Industry Insights. Simply.

  • Latest reports & slideshows with insights from top research analysts
  • 150+ Million searchable statistics with tables, figures & datasets
  • More than 25,000 trusted sources
  • Single User License — provides access to the report by one individual.
  • Department License — allows you to share the report with up to 5 users
  • Site License — allows the report to be shared amongst all employees in a defined country
  • Corporate License — allows for complete access, globally.

ReportLinker may already be registered as a supplier with your company. If you want to Order by PO, check with us first and we'll let you know if we are a registered supplier and what the vendor number is. Otherwise, we'll provide you with the necessary information to register ReportLinker as a vendor.

Grace helps you find the right report:

The research specialist advised us on the best content for our needs and provided a great report and follow-up, thanks very much we shall look at ReportLinker in the future.

Kate Merrick

Global Marketing Manager at
Eurotherm by Schneider Electric

We were impressed with the support that ReportLinker’s research specialists’ team provided. The report we purchased was useful and provided exactly what we want.

Category Manager at

ReportLinker gave access to reliable and useful data while avoiding dispersing resources and spending too much time on unnecessary research.

Executive Director at
PwC Advisory

The customer service was fast, responsive, and 100% professional in all my dealings (...) If we have more research needs, I'll certainly prioritize working with ReportLinker!

Scott Griffith

Vice President Marketing at
Maurice Sporting Goods

The research specialist provided prompt, helpful instructions for accessing ReportLinker's product. He also followed up to make sure everything went smoothly and to ensure an easy transition to the next stage of my research

Jessica P Huffman

Research Associate at
American Transportation Research Institute

Excellent customer service. Very responsive and fast.

Director, Corporate Strategy at

I reached out to ReportLinker for a detailed market study on the Air Treatment industry. The quality of the report, the research specialist’s willingness to solve my queries exceeded my expectations. I would definitely recommend ReportLinker for in-depth industry information.

Mariana Mendoza

Global Platform Senior Manager at
Whirlpool Corporation

Thanks! I like what you've provided and will certainly come back if I need to do further research works.

Bee Hin Png

CEO at
LDR Pte Ltd

The research specialist advised us on the best content for our needs and provided a great report and follow-up, thanks very much we shall look at ReportLinker in the future.

Kate Merrick

Global Marketing Manager at
Eurotherm by Schneider Electric

  • How we can help
    • I am not sure if the report I am interested in will fulfill my needs. Can you help me?
    • Yes, of course. You can call us at +33(0) 4 37 65 17 03 or drop us an email at [email protected] to let us know more about your requirements.
    • We buy reports often - can ReportLinker get me any benefits?
    • Yes. Set up a call with a Senior Research Advisor to learn more - [email protected] or +33(0) 4 37 65 17 03.
    • I have had negative experiences with market research reports before. How can you avoid this from happening again?
    • We advise all clients to read the TOC and Summary and list your questions so that we can get more insight for you before you make any purchase decision. A research advisor will accompany you so that you can compare samples and reports from different sources, and choose the study that is right for you.

  • Report Delivery
    • How and when I will receive my Report?
    • Most reports are delivered right away in a pdf format, while others are accessed via a secure link and access codes. Do note that sometimes reports are sent within a 12 hour period, depending on the time zones. However, you can contact us to escalate this. Should you need a hard copy, you can check if this option is offered for the particular report, and pay the related fees.
  • Payment conditions
    • What payment methods do you accept?
      1. Credit card : VISA, American Express, Mastercard, or
      2. You can download an invoice to pay by wire transfer, check, or via a Purchase Order from your company, or
      3. You can pay via a Check made out in US Dollars, Euros, or British Pounds for the full amount made payable to ReportLinker
    • What are ReportLinker’s Payment Terms?
    • All payments must normally be submitted within 30 days. However, you can let us know if you need extended time.
    • Are Taxes and duties included?
    • All companies based in France must pay a 20% tax per report. The same applies to all individuals based in the EU. All EU companies must supply their VAT number when purchasing to avoid this charge.
    • I’m not satisfied. Can I be refunded?
    • No. Once your order has been processed and the publisher has received a notification to send you the report, we cannot issue any refund or cancel any order. As these are not ‘traditional’ products that can be returned, reports that are dispatched are considered to be ‘consumed’.
  • User license
    • The license that you should acquire depends on the number of persons that need to access the report. This can range from Single User (only one person will have the right to read or access the report), or Department License (up to 5 persons), to Site License (a group of persons based in the same company location), or Corporate License (the entire company personnel based worldwide). However, as publishers have different terms and conditions, we can look into this for you.
Purchase Reports From Reputable Market Research Publishers

Product Design & Development Services Market Research Report by Services, Application, End-Use, State - Cumulative Impact of COVID-19, Russia Ukraine Conflict, and High Inflation - United States Forecast 2023-2030

  • $ 3949
  • January 2023
  • 148 pages

Product Design & Development Services Market Research Report by Services (Concept & Requirements Development, Design Verification, and Detailed Design & Process Development), Application, End-Use, State ...

  • United States
  • Medical Device
  • Marketing Research
  • Industry analysis


Reportlinker.com © Copyright 2023. All rights reserved.

ReportLinker simplifies how Analysts and Decision Makers get industry data for their business.

Make sure you don’t miss any news and follow us on