Tag Archives: clinical trials

Orphan Drugs: Are They Worth It?

Stormy sky over ocean

The Orphan Drug Act continues to bring hope to patients with rare diseases while providing the guidance and incentives that companies need to develop desperately needed therapeutics.

By:  Kelsey McCormick, MS

Nearly a year ago, I was scrolling through my news feed on Facebook (yes, one of my guilty pleasures!) and came across a very disturbing picture of a newborn with horrible blisters and open sores all over his body.  With immediate heart ache, I had to know his story.  I found out he was suffering from Epidermolysis Bullosa (EB), a rare genetic disorder that causes the skin to be so fragile that the slightest touch or friction can cause severe and painful blistering—internally and externally.  My eyes filled with tears seeing this poor baby and his family.  I have followed him on Facebook for nearly a year.  He struggles with severe pain every day, constant bandage changes, infections, lengthy hospitalizations, etc.  Why had I never heard of this?  What is being done for this poor, helpless baby?

Thanks to Facebook, I am now AWARE.  And awareness is what will ultimately spread the word and help those suffering from these horrible unmet medical need.

The orphan disease challenge
EB is classified as an Orphan Disease because it affects less than 200,000 people in the U.S.  It’s such a horrible disease and therefore low prevalence is a good thing!  But . . . the bad thing is pharmaceutical companies are typically less motivated to develop treatments for diseases that only affect a small number of people.  The generated sales are usually minimal compared to those with a cancer that affects a larger population (e.g. breast cancer or prostate cancer).

But what about this poor baby suffering on a daily basis?   Not to mention the suffering of his parents and siblings (and the family wallet!).  How can we help him?

 A winning solution for patients and pharmaceutical companies
Thankfully, Congress recognized the unmet medical needs and the lack of treatments/therapies for many rare diseases, which led to the passage of the Orphan Drug Act (ODA) in 1983.  The ODA provides incentives for the development of Orphan drugs (See associated Regulations here:  Code of Federal Regulations, Title 21 Food and Drugs, Part 316 Orphan Drugs).  Shortly thereafter, the Office of Orphan Product Development (OOPD) within the FDA was formed to help guide the Sponsor Companies through the complex regulatory pathway of an Orphan drug from discovery to market.  As one would hope, the passing of the ODA and its incentives has led to a major increase in the market approval of new therapies for Orphan diseases.

There is hope, much hope, for my little, precious Facebook friend!

Special opportunities for companies developing orphan drugs
Naturally, one would ask, “What are the incentives of developing a treatment or therapy for an Orphan Disease?”  To list a few:

  • The Prescription Drug User Fee (PDUFA) is waived. And they aren’t cheap! See below for fee rates for FY 2013

Fee Table

  • There is a 50% tax credit to help defray clinical study costs
  • Eligibility to apply for the FDA Orphan grants program
  • 7 years marketing exclusivity

Those are some good incentives!

Now what?  How do we do this?  What are the next steps?

In order to obtain the above incentives, the Sponsor Company must apply for Orphan Drug Designation with the FDA and if “approved,” the FDA will grant the Sponsor Orphan Drug Designation in writing.  Specific instructions can be found here:  How to apply for Orphan Drug Designation

Is it worth it?
It sure is.  Take a few minutes to Google Epidermolysis Bullosa and perhaps a story or two will catch your attention just like my little EB baby Facebook friend caught mine.  Within minutes, I would bet my glass of wine (and anyone that knows me, that’s huge!!) that you will feel the “real” motivation towards finding treatments and cures for the many unmet medical needs.  The incentives from the ODA are just icing on the cake. 

Key Tips for Success with Orphan Drugs

  • Motivation for your company’s goals is critical . . . the path to market for an Orphan Drug has many gray areas . . . there’s no easy answer, so one must have true commitment and passion for the suffering patients whose finger is in the pill vial or arm is at the tip of the syringe.
  • Attempt to obtain Fast Track Designation with this, the FDA will be more proactive and help facilitate the development and expedite the review of drugs intended for an unmet medical need.  All in hopes to get the drug to the patient faster!
  • Defining meaningful biomarkers early can help in selecting a quantifiable clinical endpoint and accelerate completion of this process.
  • Early and consistent communication with the FDA is critical before and during the development of your Orphan drug (or any drug for that matter!).

Picture Credit:  zoomion via photopin cc

Never Giving Up After Failure Will Lead to Success

By: Kelsey McCormick, M.S.

Sky with sun and clouds

The passion of your team with the guidance of a regulatory expert will be critical factors in getting the approvals you need for commercial success.

You have to work through the “wrong” ones to get to the “right” one. What does that make you think of? It makes me think of 2 things: 1) Relationships, and 2) Drug Development. I know, you’re thinking, “huh?”

Think about it though . . . dating . . . how many people did you date before you found your “Mr.” or “Mrs. Right?” (Those who are still looking don’t give up!) Think of all the time, emotions and money spent on the “wrong” ones! In my case . . . there was a lot of “sifting” until I found my loving hubby (Brownie points if my husband actually reads this! Better yet, if he doesn’t mention this, he’s sleeping in the shed tonight because it means he didn’t read my newsletter!). It was from those “Mr. Wrongs” that I learned what I ultimately wanted (or didn’t want!) in a life partner! I am now happily married (10 years already!) with two wonderful kids (cue cheesy love song).

The regulatory challenge of drug development
Drug development . . . as you all probably know (and experience!), there is much failure in this amazing, but challenging industry we live in! It can be frustrating and draining to spend so much time, money and even emotion on a product, to then have it fail in Clinical Trials and never make it to market (cue depressing break-up song).

“In general, it costs an average of $800 million and takes 12 to 15 years before a drug makes it from the lab bench to your medicine cabinet!”(1)

The regulatory roadmap
It’s tough to summarize activities over 11-14 years, but in short (as most of you know), the process goes like this:

Drug discovery begins with an idea for a new disease target, often licensed from a university laboratory.

–Industry researchers start sifting through hundreds (sometimes thousands!) of compounds, looking for one that will hit the biological target.

–The lead candidate is tested in animals to look for toxic side effects and potential efficacy.

–IND Phase: clinical trials in humans. These trials eat up much of the development costs.

  • Phase 1: small number (about 20-80) of healthy volunteers to assess safety
  • Phase 2: medium number (about 100-300) of patients (affected by the disease the drug is indicated for) to assess efficacy, safety and pinpointing acceptable dose range
  • Phase 3: large number (about 1000-3000) of patients to assess efficacy, safety and identify side effects.

And even more depressing . . . only about 1 in every 5,000 drugs in development actually make it to market.

What you need to successfully navigate the regulatory roadmap
Talk about a high failure rate (kinda like youngins dating)! YUK! There’s a ton of detail that goes into the above lengthy process. It takes a lot of brains (like yours), a lot of hard work (like mine), and a lot of hope (optimists) to get to market.

In trying to find the bright side of each of these depressing realities, which in turn will help motivate us: as you see, the cost to develop a drug is ridiculously high . . . this helps me feel a little better when I go to buy an OTC or prescription drug and my wallet is quickly emptied! And with the high failure rate, I’m very comforted in knowing how detail-oriented, diligent and CAREFUL drug companies and the FDA are when developing these products and reviewing the data. Makes me feel a little safer knowing the process isn’t rushed just to get a drug on the market.

So how do we continue on with our daily jobs knowing the painfully low success rate in our industry? In my mind, it’s all about perspective, persistence and positive thinking.

Quick Tips:

  • As in dating, we have to weed through the bad ones to get to the good one! DON’T RUSH IT. There’s no turning back (well, not easily and pain-free anyway)!
  • Each failed drug or failed relationship is NOT a waste of time or money. TONS of useful information is gained from these processes. Stay positive no matter the results, and be proud of the work accomplished.
  • Take advantage of all the years of knowledge that’s out there (literature, colleagues), and consider similar products that have failed and/or been approved. You’ll have a head-start!
  • Know your competition . . . risk of failure is that much higher when someone else is developing a similar product with the same disease indication.
  • Have passion for the product you are developing . . . that helps maintain the positive attitude and “I won’t give up” mentality!

I won’t give up, every day that passes; every lesson learned . . . we are that much closer to ‘the one’.”


  1. Cost and time line estimates based on over 15 years of industry experience of McCormick LifeScience Consultants, LLC.

Picture Credit:  © Mihalec | Dreamstime Stock Photos & Stock Free Images