Winter 2014 – BIO375H5S – Midterm Answers Key

(Alvin’s  Marking Notes; Questions 1 to 10):

1.) What right does a patent confer to an inventor? (1)

The right to exclude other from selling (or using is fine) your invention.
**Students  only  stating  “the  right  to  exclude”  and  nothing  else  received  0.5  marks.

2.) In  drug  discovery,  what  is  meant  by  a  “target”?  (1)

The molecule (protein/ gene is fine) that is defective in the disease that is used to screen potential
drugs.
**Students that did NOT mention WHAT the molecule (i.e. protein/gene), defective/disease, and/or
screening potential received 0.5 marks.

3.) If there is a business case to support a technology, what does this mean? (1)
It means the venture is profitable enough, meeting or surpassing return on investment levels, (to justify
pursuit by a business, which they may not add).
**Any things along the lines of ROI, profit, money yielding, etc. received FULL marks for this
question. Several students were off on this question stating business or action plans as answers – NO
marks were awarded!

4.) What is the difference between the CEO and President in terms of their roles? (1)
The CEO acts as the liaison between the board and management (0.5 marks), while the President
supervises the senior management on day to day operational issues (0.5 marks).
** Students needed to EXPLICITLY define the DIFFERENCES in terms of the ROLES assumed. Students
who had the answer above received full marks. Students on tangents (i.e. face of the business,
bringing in the money, etc.) were NOT awarded any marks. Here students FAILED to DEFINE the
DIFFEENCES assumed in ROLES by the CEO and President.

5.) What are the typical routes of administration for a biologic? (1)
Iv, IM, and/or Sc – anything but an oral route of administration. If they say oral, give them zero for this
question.
**Students received FULL marks for listing ANY of the three routes (i.e. IV, IM and/or Sc). Students
stating an ORAL route received ZERO. Students listing ANY of the three routes + an ORAL route also,
received a ZERO!

6.) What is the difference between a primary and secondary endpoint? (2)
Primary endpoint is: the single outcome measure that the FDA will evaluate the trial on and it is the
endpoint the study is statistically powered to detect (1 mark). All remaining endpoints are secondary
endpoints (1 mark).
**Students received FULL marks if the answer clearly DIFFERENTIATED between PRIMARY and
SECONDARY endpoints. Students failing to include DIFFERENCES or lacked explanations received a
ZERO for this question. Students answering the question in full, but failed to mention statistically
powered/approval body (FDA, etc.) received either a 1 or 1.5 (dependant on answer and what was
forgotten).

1
7.) What is the relevance of a predicate in a new product approval? When is it used with respect to the
product and regulatory pathway? (2)
The use of a predicate allows a new medical device to get to market faster and more cheaply (less data)
(1 mark). It is used in the 510K pathway (1 mark).
** Student needed to EXPLICITY state the RELEVANCE of a PREDICATE in NEW product APPROVAL. A
great deal of students spent a lot of time DEFINING a predicate WITHOUT answering the question.
These students received a ZERO for this question. Students who answer the question in full, however
failed to mention the regulatory pathway (i.e. 510K pathway) received a 1 mark deduction.

8.) [Twitter Feed] What wearable technology space has Rogers entered? What is the name of the
product? (2)
A wearable technology that is a diaper (1 mark) called Exmobaby (1 mark).
**A POORLY answered question!!! This was an ALL-or-NONE situation. 1 mark for stating DIAPER (i.e.
wearable technology) and the EXMOBABY (i.e. NAME of the product – spelling counted!!!).

9.) If a manufacturer wishes to add a new dose for use for its already approved drug, what clinical trial
step must it likely return to, if any, and why? (2)
They must return to phase II testing (or they could say phase III – that’s  fine)  (1 mark). There is no point
returning to phase I as the full dose range was already tested in that phase so repeating that step will
provide no new dosing data (1 mark).
**Students answered this question quite well! 1 mark for the CORRECT phase (i.e. Phase II or Phase
III ONLY) and the RATIONALE (1 mark). The rationale needed be explicit in your understanding of the
drug phases and a discussion of full dose range, redundancy and dosing data were ALL needed in
your answer for FULL marks.

10.) You  file  a  patent  on  a  new  medical  device  but  you  are  concerned  about  “work  around  solutions”.    
What  are  “work  around  solutions”?    Why  are  they  a  problem  for  medical  devices  but  not  drugs?    (2)
Works around solutions allow you to create a product that meets the same customer need but does not
infringe on the patent claims of an already existing product (1 mark). This is possible for devices as we
understand these products well and can modify them to get around existing claims, but drugs we do
not understand well enough to modify and predict the outcomes (1 mark).
**Students needed to clearly DEFINE   “work   around   solutions”   making   sure   to   include   the   need   to  
meet the same customer need WITHOUT the infringement on patent for the first full mark. Students
forgetting the infringement on patent received a 0.5 mark deduction. The PROBLEM needed to be
explicit in your understanding of medical devices and drugs – the understanding of medical devices
to allow for modification to surpass existing claims (0.5 marks) AND the LACK of understanding of
drugs such that we cannot PREDICT outcomes or understand it well enough for modification (0.5
marks) were BOTH needed for the second FULL mark.

2
11.) In the graph above it depicts data from a recently completed clinical trial for patients suffering from
angina, a form of chest pain experienced during physical exertion. Remission refers to patients who no
longer feel such pain. The treatment group received the new drug while the control group received
placebo. A binomial test between the groups yielded a p-value of 0.03 . What clinical trial phase is this
most likely to be and why? Do you find this data convincing that the drug in question works? Would
the FDA find this data convincing enough for approval? Explain. Make assumptions as necessary to
answer this question. (5)

(Dr.  Parker’s  marking Notes; Question 11)

3
4
5