HomeLatestWhy develop or invest in rare orphan disease treatments? The better question is: Why not?
Why develop or invest in rare orphan disease treatments? The better question is: Why not?
November 29, 2021 at 2:37 pm - by Editor Prism MarketView
“Rare” or “orphan” diseases aren’t so rare anymore. Today, there are about 300 million people worldwide, including one in 10 Americans, living with one of 7,000 rare diseases, according to the National Institutes of Health (NIH).
Many of these diseases are without any effective, approved treatment and while drug development can be both expensive and time-consuming, the FDA’s support for developing new therapeutics and bolstering regulatory approval can mean a faster pathway to market. Interestingly, global orphan drug sales are forecast to grow at double the rate of non-orphan drugs, with sales anticipated to reach $262 billion – capturing one-fifth of worldwide sales by 2024 – according to EvaluatePharma’s Orphan Drug Report.
From rare to well-done
With the trifecta of premium prices, tax breaks and reduced marketing costs, the Orphan Drug Act of 1983 was designed to incentivize companies to pursue the orphan drug market and it worked: The FDA has approved close to 800 new orphan drugs since it took effect, with the lion’s share taking place post-2013. The Act was so successful that it prompted similar laws in the European Union and Japan.
These new therapies are helping those with rare diseases while continuing to climb in value. The global rare orphan disease market is expected to enjoy a compounded annual growth rate greater than 11 percent between 2018 and 2024, based on EvaluatePharma research.
Meanwhile, expanded patient advocacy, medical advances, venture capital interest and industry partnerships are combining to highlight the global importance of rare orphan disease treatments.
Changing the world of rare disease
Companies that develop rare orphan disease treatments and individuals and organizations that invest in them are not only doing good for those in need: They enjoy less competition, since many larger companies are reluctant to pursue treatments for a smaller audience, not to mention a full seven years of patent protection for investing in research and development—two years longer than the exclusivity timeframe for other drugs. Finally, approval times are often swifter for drugs that have orphan drug status.
Notably, a company’s stock price increases by nearly 3.4 percent in correlation with an orphan drug designation announcement, according to the Journal of Rare Diseases.
Further, the unmet need is astronomical: There are only treatments for about 5 percent of rare diseases, according to Global Genes, however, there are more than 560 drugs in development for rare diseases, based on a PhRMA report.
While these drugs are often expensive, they are also life-changing and life-saving. The opportunity to develop or invest in orphan drugs can be invaluable for companies and investors alike.
Share this article:
Disclaimer
This communication was produced by Prism MarketView, an affiliate of PCG Advisory Inc., (together “PCG”). PCG is an integrated investor relations, communications and strategic advisory firm. The information contained on this is ‘Paid Advertising’ for purposes of Section 17(b) of the Securities Act of 1933, as amended (together with the rules and regulations there under, the “Securities Act”). “PCG” and its affiliates are compensated by respective clients for publicizing information relating to its client’s securities. For more information in terms of compensation received for services provided by PCG, see the pertinent advertising materials relating to the respective client. By accessing this Site and any pages thereof, you agree to be bound by the Terms of Use and Privacy Policy…
This communication was produced by Prism MarketView, an affiliate of PCG Advisory Inc., (together “PCG”). PCG is an integrated investor relations, communications and strategic advisory firm. The information contained on this is ‘Paid Advertising’ for purposes of Section 17(b) of the Securities Act of 1933, as amended (together with the rules and regulations there under, the “Securities Act”). “PCG” and its affiliates are compensated by respective clients for publicizing information relating to its client’s securities. For more information in terms of compensation received for services provided by PCG, see the pertinent advertising materials relating to the respective client. By accessing this Site and any pages thereof, you agree to be bound by the Terms of Use and Privacy Policy.
“PCG” and its affiliates are not a registered or licensed broker, dealer, broker-dealer, investment adviser nor investment manager, nor does “PCG” engage in any activities that would require such registrations. “PCG” and its affiliates do not provide investment advice, endorsement, analysis or recommendations with respect to any securities, and its services to or statements about its clients should never be construed as any endorsement of or opinion about any security of any client. No information contained in this communication constitutes an offer to sell, a solicitation of an offer to buy, or a recommendation of any security or any other similar product or service regardless of whether such security, product, or service is referenced in this communication.
Our articles, reports/releases and videos are a commercial advertisement and are for general information purposes ONLY. We are engaged in the business of marketing and advertising companies for monetary compensation. We only publish favorable information because we are compensated to publish only favorable information. We are not objective or independent and have multiple conflicts of interest. The Profiled Issuers and parties hiring us have conflicts of interest.
The contributors may buy and sell securities before, during and after any particular article, report and publication. In no event shall “PCG” or affiliates be liable to any member, guest or third party for any damages of any kind arising out of the use of any content or other material published or made available by “PCG”, including, without limitation, any investment losses, lost profits, lost opportunity, special, incidental, indirect, consequential or punitive damages.Further, nothing in this communication is intended to provide tax, legal, or investment advice and nothing in this communication should be construed as a recommendation to buy, sell or hold any investment or security or to engage in any investment strategy or transaction. Investing in micro-cap and growth securities is highly speculative and carries an extremely high degree of risk. It is possible that an investors investment may be lost or impaired due to the speculative nature of the companies profiled. Never invest in any stock featured by “PCG” and its affiliates unless you can afford to lose your entire investment.We urge Investors to conduct their own in-depth investigation of the Profiled Issuers with the assistance of their legal, tax and investment advisers. An investor’s review of the Information should include but not be limited to the Profiled Issuer’s financial condition, operations, management, products or services, trends in the industry and risks that may be material to the profiled Issuer’s business and other information he and his advisers deem material to an investment decision. We encourage our readers to invest carefully and read the investor information available at the web sites of the U.S. Securities and Exchange Commission (SEC) at www.sec.gov and the Financial Industry Regulatory Authority (FINRA) at www.finra.org.
For full disclaimers, including compensation received for professional services, please visit www.pcgadvisory.com/disclosures.
