Top Trends in Orphan Drugs. Brand Acumen. The Global Leader in Pharmaceutical Name Development and Submission Strategy.

1. Top Trends in the
Orphan Drug
Industry
Your guide to the hot topics
influencing the industry now

2. Orphan Drug has become the most valuable designation in the Pharmaceutical
Industry. With each new rare disease focused on, the trends for the pharmaceutical
industry continue to shift towards a more personalized approach. The winds of change
are strong in Pharma – and that makes the trends that make up this movement even
more important. We gathered information from top pharmaceutical companies, patient
groups, regulatory agencies, and healthcare providers to find out what some of the top
trends were within the Orphan Drug movement. Varied in scope, these 17 trends are
where the industry is headed, and are a must-read for any company looking to enter
the rare disease marketplace.

3. The price of Orphan Drugs is increasingly under
scrutiny.
In the United States, The Department of Health and Human Services is fighting to keep
the price of Orphan Drugs discounted when they are not being used for their specific
rare disease designation, citing rule 340B. Meanwhile, industry claims that the same
rule means that all drugs as designated as Orphan by the FDA are free from discounted
pricing. A recent court ruling has put industry’s opinion in the right – but HHS hasn’t
backed down and continues to discount Orphan Drugs in certain cases. In the UK,
NICE and payers alike have begun to ask for more of a basis on pricing.
What is the 340B Drug Pricing Program?
1

4. Rare disease patient groups are forming registries.
Looked at as almost a status symbol by the more established
patient groups, these patient registries allow for a better
understanding of the natural history of many rare diseases. This is
essential for a more efficient development process, and industry
agrees. The fact that many patient organizations are embarking on
this form of data generation is good for the upstream development
of these drugs.
2

5. Breakthrough Therapy Drug Designation is a
boon for the orphan drug industry.
Breakthrough Therapies are defined by the FDA as a drug that, either alone or in
combination with another drug, treat a serious or life threatening disease and based on
preliminary clinical evidence may be far superior to anything in the market. For
Orphan Drugs, meeting those criteria are far easier that their standard counterpart.
Recently, Novartis gained a coveted third Breakthrough designation – meant for
treatment of a rare disease. Meanwhile, other companies, such as Pharmacyclics, have
cited the rule with chopping 7 to 9 months off the regulatory process.
3

6. Rare diseases are increasingly being seen as a tool
to cure the common ones.
Common diseases are often complex, consisting of many pathways
over several systems. Rare diseases, however, usually are far more
limited in their scope – often one gene and one pathway or
mechanism. In fact, many rare diseases account for subsets of
common ones. By studying rare diseases, there is a new thinking in
the field that we are in fact studying ways to cure the common ones
one pathway at a time.
4

7. Pressure is increasing for early access to potentially
lifesaving treatments via FDA Expanded Access and/or
Right to Try laws.
If someone’s prognosis is already fatal, are we justified in fast-tracking them a
proclaimed cure that could have deadly consequences in an attempt to save their life?
The government and industry seems to believe so – though in not so stark a situation.
While Expanded Access and Right to Try laws do not eliminate regulation, they
certainly help to bypass the bureaucracy when someone’s life could be saved by a risky
treatment.
5

8. Patient engagement in Clinical Trials is
increasing.
Both industry and the FDA have been increasingly growing
their desire to have patients actively involved in the clinical
trials process, both through patient representatives and
workshops aimed at educating the test candidates what,
exactly, it is they are looking for. The advent of patient groups
and registries is helping with this tremendously.
6

9. There is a shift toward more curative treatments like
gene therapy versus chronic, lifelong treatments.
Enabled by new technology, the days of taking a pill to live a normal life may be long
gone, and the days of an actual cure may be on the horizon. Gene therapies and other
treatments, such as CRISPR and RNAi, are proving to appear to live up to the hype.
These therapeutics have the potential to treat the disease – and never have to treat them
again.
7

10. Patient organizations are increasingly looking to
shape policies for rare diseases.
Patient groups have more and more influence, particularly as they form registries, and
they can put more pressure on the industry than previously thought – and they know
it. Patient groups have led to companies racing to develop therapies, developed
guidance on risk-benefit, and are increasingly looking to lobby for more favorable
terms to help patients in the long haul. See the AKU Society for a great example of a
patient organization working hard to push for what they need.
8

11. Policies continue to impact the rare disease space.
In 1982, nobody developed for orphan drugs. Starting with the passage of the Orphan
Drug Act in 1983, the rare disease space will always be tied to regulation and legislative
action due to the nature of the risk-benefit surrounding developing therapies for them.
In 2012, the Breakthrough Therapy laws changed the face of the game again, and future
laws will continue to shift the playing field in ways that we can’t even predict yet.
9

12. Governments are trying to further help companies
to work on rare diseases.
There are many means for governments to innovate to help spur growth in the rare
disease space, and adaptive licensing in Europe is just one example. As the impacts of
this research on the population as a whole increases, government interest will only
grow.
10

13. Rare disease is increasingly seen from a global
perspective.
Disease doesn’t end at our borders – and regulatory
agencies are starting to take note. The FDA and EMA are
working together more on rare disease topics than ever
before, and more countries are getting involved yearly.
This will help spur orphan drug laws in countries where
they do not currently exist.
11

14. Mergers will continue to be common.
Just recently, it was revealed that AbbVie was looking to acquire Shire for their Orphan
Drug focus – a merger that seems to be likely to take place. In an industry where one
breakthrough drug can make or break your finances, expect to see mergers and
acquisitions continue to be a major player in the marketplace, particularly as Big
Pharma knows they can purchase a smaller Orphan Drug pharmaceutical manufacturer
and limit their costs while reaping all of the rewards.
Find out more about the merger between AbbVie and Shire here >
12

15. Drugs will be increasingly produced via
Bioprocessing.
Biologics are becoming more and more common every day, and the advantages can’t be
ignored. The biologics space – and the cell culture field in general – is growing at a
staggering rate, and as more and more pharmaceuticals shift to biologics production, so
too will the orphan drugs.
13

16. Therapy areas are getting increasingly focused.
Long gone are the days where pharma companies decided to target
every single disease known to man. It is becoming more and more
common for companies to have therapeutic “areas of focus”,
specific diseases or areas that they are targeting for one strategic
reason or another. This increase in focus coupled with the intrinsic
advantage is driving an increase in Orphan Drug production, as
companies explore every aspect of their core competencies. For
example, if a company is focused on the rheumatics, rare disease
rheumatics are a natural extension.
14

17. R&D Centers and Innovation Clusters will spur
growth in the industry.
Countries are recognizing the benefit to catering to pharma and biotech companies –
and it is shown in the growth of R&D centers across the globe. This will help spur
orphan drug development and cooperation among typical competitors.
15

18. As new countries pass orphan drug legislation,
emerging markets will increasingly play a large role in
the industry.
The countries that currently have Orphan Drug legislation have spurred
unprecedented growth, but there are still several huge untapped markets – including
China – that will become an enormous boon to the industry as payers and patient
groups in those countries begin to push for the same rules as in the US or EU.
Attendance by regulators and payers from these countries at recent conferences, such
as the World Orphan Drug Congress, signals interest on the part of those countries in
expanding their legislation. This will open up a huge additional market for the already
booming orphan drug producers.
16

19. Big Data is helping speed up clinical trials and
promote faster access to information on all sides.
Big data is helping the entire biotechnology industry, and Orphan Drugs is no
exception. In fact, several big data and analysis companies attended last year’s World
Orphan Drug Congress. Being able to properly analyze data rapidly is speeding up
clinical trials as well as speeding up the opportunities to look into new challenges.
Likewise, the advent of patient registries is bringing forward new challenges to be
solved by Big Data as the field progresses.
17

20. Exploring all areas of rare disease- through strategic keynote presentation, focused
tracks, collaborative roundtable discussions and abundant 1-2-1 partnering; the
World Orphan Drug Congress gives you the opportunity to tailor your
participation and experience to your needs.
Taking place between April 24-25, 2015, the World Orphan Drug Congress remains the place to
discuss the hot topics currently being discussed within the industry.
Find out more at: www.terrapinn.com/orphandrugsus