Customer Focus Pty Ltd; Insync Surveys Pty Ltd; GeneMiner
Submission
Review of Medicines and medical Devices Regulation
Tom Holman;tom.holman@customerfocus.com.au +614 1256 6425
1-5-2015
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Contact details:
Tom Holman
Customer Focus Pty Ltd
m +61 (0)412 566 425 | t +61 (0)3 9712 0930
e tom.holman@customerfocus.com.au | w www.customerfocus.com.au
a Mirrm Boolok, PO Box 1104, Kangaroo Ground, Vic 3097, Australia
THIS SUBMISSION IS ON BEHALF OF
 CUSTOMER FOCUS PTY LTD, A CONSULTANCY;
 INSYNC SURVEYS, A FULL SERVICE RESEARCH COMPANY;
AND
 MIRRM PTY LTD, TRADING AS GENEMINER, A START UP
MEDTECH COMPANY
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Table of Contents
Summary .........................................................................................................................................................2
Context............................................................................................................................................................3
Specific comments on Questions for Consideration.......................................................................................4
Specific comments page 5 ..........................................................................................................................4
Specific comments page 6 ..........................................................................................................................4
Questions for consideration: ......................................................................................................................4
Page 13 Are there any additional principles that should be considered? ..............................................4
Page 17 what options are available for determining ‘trusted overseas regulators’? .............................4
Page 23 Should Australia introduce an accelerated approval program? ...............................................5
Comments on Chapter 7 - the regulation of medical devices. ...................................................................5
Page 69 Should the TGA undertake its own assessment of the competency of EU notified bodies? ....8
Page 74 Is the current regulatory framework and classification system flexible enough to
accommodate new and emerging medical device technologies? ..........................................................8
Page 75 Does the current regulatory framework to medical devices in Australia provide an
appropriate balance between managing risk and minimising unnecessary regular regulatory burden?
..............................................................................................................................................................10
Page 77 Does Australia have the balance right between pre-market and post-market regulation of
medical devices? ...................................................................................................................................12
Page 79 Has the regulatory framework for IVDs resulted in a reduced emphasis on clinical best
practice? ...............................................................................................................................................12
Page 83 Is the classification system to medical devices too complex? ................................................13
The survey .....................................................................................................................................................14
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Summary
Information is fundamentally different from medicines and medical devices in that it has the ability to
guide decision making. As developments in healthcare are both increasing consumer centricity and
diversifying the information available about an individual’s health, the lumping of the entire range of
diagnostic processes, devices and reports into one stream will discriminate against individuals who wish
to access their genome and the insights that are now and will be increasingly derived.
Human Genomics testing and analysis is a unique segment within IVDs. As it is primarily about
information, the risk is low and the benefits are high, so it should have a separate and reduced regulatory
burden.
Informed consent is the key to a reduced regulatory burden and the use of genomic information should
be determined by the individual in collaboration with their health professional. The process of informed
consent should be regulated; specific genetic tests should not. The market will monitor the quality of
output.
Global decisions may not apply due to Australia due to legal and cultural differences so a review of any
‘trusted overseas regulator’ should occur through a regulatory committee within the human genomic
stream. To do otherwise may preclude Australia from leadership in this field.
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Context
Thank you to the opportunity to comment on the Review of Medicines and Medical Devices Regulation.
We agree that this is both a timely and necessary review and commend you on the thoughtful and
comprehensive discussion paper.
We support the terms of reference and wish you well in your deliberations to find the appropriate
balance between risk and benefit. We believe this is the core of the issue and this balance is challenged
by two trends that fail to be addressed by the current framework. The first is the disruptive nature to the
existing health system that some of the new innovations carry. It is likely that the existing system will
frustrate their development. Secondly, the trend towards self-care, with the shift in the locus of control
and acceptance of risk towards the individual and away from the health care practitioner, needs to be
addressed through a relaxation of regulatory control and not an increase.
Our submission will focus around the human genome and the information arising from it. We contend
that this area should be a specific consideration within the regulatory environment of medical devices,
specifically IVDUs, but should be regarded as an information service for consumers in partnership with
their healthcare practitioners.
If some characteristics of high quality information include accuracy, consistency, completeness,
uniqueness, and timeliness, those characteristics are better judged from different perspectives. While
accuracy and consistency may be best judged by regulated and independent quality systems, the
characteristics of uniqueness and timeliness are more important to the individual who uses the
information. Completeness is a matter of transparency which can also be made apparent to the
individual. So who is in the best position to judge the quality of the genomic information available to an
individual about their genetics? We contend it is a tripartite decision where timeliness and relevance to
the individual are higher priorities.
With these things in mind, we present some arguments and evidence from consumers to assist you in
your review. We welcome further discussion if any of the issues we raise are unclear or require further
detail.
All consumer comment and quantified feedback are from a survey run between January 2nd and January
5th 2015. More details are found in the appendix.
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Specific comments on Questions for Consideration
In document order, please find following some commentary:
Specific comments page 5
In the first paragraph, the asymmetry which you observe is now not as significant as it has been
principally due to better information sources and a more literate health consumer population. While
there still will be a requirement to care for the more vulnerable members of our community, this is a
more likely to be done through judicious regulation and moderation via the health care practitioner and
not through stronger regulation.
Specific comments page 6
We suggest that post-market monitoring systems via reporting of adverse drug reactions and registers be
mandatory and should be extended. There is still a relatively weak proactive monitoring of consumer
experience in an ongoing manner that equates quality of life outcomes with interventions.
Questions for consideration:
Page 13 Are there any additional principles that should be considered?
We contend that there are two elements that should be articulated in the principles. It may well be
argued that these elements would be considered within the principles but we suggest they should be
overtly mentioned. Principle one should be reworded to read “The role of regulation is to manage risk in
order to maximise public health and safety”. The reason for this variation is that to do no harm to a single
individual risks not allowing the benefits that may be realised for many more. Protection is a risk adverse
strategy and generally works against new innovations. To replace “protect” with “maximise” is a more
contemporary orientation. The second element would be to include the trend towards self-care and
autonomous responsibility for health. Although Principle five refers to the ultimate responsibility
remaining with the Commonwealth, it should also reference to the partnerships that exist with
healthcare practitioners and consumers. Therefore it is suggested to rework Principle five as “Through
partnerships with healthcare practitioners and consumers, the ultimate responsibility for medicines and
medical devices regulation should remain with the Commonwealth”.
Page 17 what options are available for determining ‘trusted overseas regulators’?
This is a difficult situation in that it presumes that ‘trusted overseas regulators’ make decisions
appropriate for the Australian environment. It is contended that in many areas, Australia will lead
developments and therefore not be subjected to the lagging regulations determined from overseas. This
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determination should be made by looking outside the regulatory system to include respected bodies in
academia, ethics and commerce.
The observation that trusted overseas regulators will judge the situation in Australia better than may be
judged by those actually in Australia is based on the risk assessment more applicable to implantable
medical devices. A stent implanted in a coronary artery creates similar risk profiles across any geography
assuming other medical support parameters are consistent. Apart from individual differences and some
ethnic types, the human population potentially receptive to cardiac stents is relatively homo-genius. But
where information differs from the stent is in the cultural context. Religious, legal, historical and ethical
environments differ widely. Compared with Australia, some countries are more litigious or religiously
fundamental which has significant bearing on availability and providence of information and are attracted
to risk and become bound up with an unwieldy and bureaucratic regulatory process that fails to respect
the rapidity of change in medical and technological revolution.
So it is contended in the area of information to follow the path of trusted overseas regulators will not be
as relevant to the Australian situation when compared to implantable medical device. This situation also
fails to anticipate and respect the fact that unique innovation and leading developments particularly in
the area of human genomic analysis can first occur in Australia. The uniqueness of the Australian
environment therefore encourages a localised approach to the regulation of human genomics.
Page 23 Should Australia introduce an accelerated approval program?
Australia should introduce an accelerated approval program via multiple pathways, particularly
recognising software, informatics and genomic sequencing as distinct from tangible medical devices.
Comments on Chapter 7 - the regulation of medical devices.
The remaining comments relate specifically to chapter 7 the regulation of medical devices.
The main issue is whether information poses risk. It is clear that in certain circumstances it certainly does.
For example, the careless communication of Huntington's disease to a young individual is likely to create
stress and behaviours that withholding of information would alleviate. On the other hand, when a
therapeutic intervention for this condition is possible – which could well be in vitro – withholding this
information would most likely be judged inappropriate.
Consumer sentiment at present is spread but the overwhelming thrust is for the individual to have access
to the totality of the information and to control its use.
5
We contend that the regulatory approach to information of potential risk is to place it in situ with both
education and duration via a health care practitioner.
According to consumers, while the preference for locus of control is for it to lie with the individual, an
input from the doctor is anticipated.
Consumer comments from Survey
I have recently been diagnosed with acinic cell
carcinoma - and am preparing for a course of radiation
therapy. Knowing this, I take the view that it would have
been better to know if there were potential 'red flags' in my
genome that might have flagged this earlier - rather than
waiting to have a tumour appear.
For us at the moment, the more information we have the
better off we are. We lost our newborn daughter in July due to
seizures and significant brain malformation. We conceived her
via IVF and are continuing to try and have a live child to raise.
Anything we can look for at early stages (via embryo testing,
amniocentesis testing etc.) to prevent going to term on
another severely affected child would be to our advantage.
Access to health information is a
personal decision with expert
advice from the doctor and not
from authorities or from family.
6
When matters become more serious, the advice of experts associated with the consumer in
understanding the situation is preferred.
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Page 69 Should the TGA undertake its own assessment of the competency of EU
notified bodies?
We contended the answer is yes and should occur through a committee of representatives that have
interests in the specific stream under consideration. For example, if this area was in the stream of human
genome testing and reporting, the membership would include geneticists, genetic counsellors, genetic
technicians, ethicists, gene sequencing commercial providers, GPs and consumers. This group would
reflect the Australian cultural environment and so be better placed to provide a view of the competency
of the EU notified body rather than just an administrative compliance.
Page 74 Is the current regulatory framework and classification system flexible
enough to accommodate new and emerging medical device technologies?
We contend that the answer is no and it should be improved by having a separate stream for those
services that focus on information. We recommend that there should be an additional subsection that
focuses on human genomic information as this is now a large, complex and rapidly emerging field. To
lump this area into existing structures will frustrate this emerging disruptive and potentially significant
technology. It is likely – and important - that the information will be provided with a lighter regulation
particularly during its formative stages. If a heavy regulation is mandated in Australia, services will be
provided to consumers from other countries.
Consumer do not have a good view of the regulation of medical technology – and it can argued why
should they have to? Never the less, only 19% are of the opinion that the system is flexible enough.
Human genomics is revolutionary. To a degree now and certainly to a higher degree in the next decade
and then very likely to a much greater degree subsequently individuals will be able to determine their
quality of health and life based on a dataset which is unique to them – their genome. This will allow
predictive health measures to be taken prior to the onset of symptoms based on specific personalised
evidence to overturn the bureaucracy of health and reverse the trend from the locus of control of health
professionals to the providence to the province of the individual and family.
8
To realise the potential in Australia, we need to reorient the locus of control and the responsibility of risk
by devolving the regulatory burden away from a central authority to a localised partnership between
health care practitioner and individual consumer. We contend that the risk of this reorientation is
minimal in the area being considered – information provision.
The key issue in this development is informed consent and the curatorial partnership with healthcare
practitioner.
It is a sobering observation that the conduct of human genomic sequencing and reporting can occur in
Australia from overseas sites in a relatively unregulated manner but as soon as that service is offered in
Australia, local and anachronistic regulations apply.
It is anticipated that the regulator may wish to consider separately and specifically each test for a gene or
a genetic condition. We contend that this is impractical and should be replaced by a lower regulatory
hurdle for a process, commercial oversight and declaration of tests and ultimately the judgement of the
healthcare practitioner and individual. To do otherwise will create an unnecessarily high burden on the
regulator, the commercial developer and delay availability for important information to the consumer.
To regulate each test would be analogous to virus checking software to have regulatory approval for each
new threat prior to implementation. In this situation, the service is provided and the judgement to adopt
is made by the consumer but the specific tests are updated regularly by the commercial provider who is
more immediately aware of the threats. To otherwise delay the upgrade of the virus checking software
would be to expose the individual to threats of security breaches.
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Page 75 Does the current regulatory framework to medical devices in Australia
provide an appropriate balance between managing risk and minimising
unnecessary regular regulatory burden?
We contend the answer is no. An example of this unnecessary burden would be to impose regulatory
approval on each new genetic test rather than simply approve the process of genomic sequencing and
analysis. We contend the provision of this information is relatively low risk when provided in
collaboration with a curatorial healthcare practitioner and consumer and so should not be included on
the ARTG. The solution is to have a sophisticated informed consent process which manages the provision
of information and its communication. The informed consent process should be under regulatory control.
While it is a concern that the level of trust in science is relatively low, regulatory approval is second
lowest with the concept of personal informed consent having most importance when judging the risk.
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In particular, the disclosure of information is preferred to be comprehensive or certain and determined
by the individual.
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When compared with a similar cohort of respondents asked in 2011, talking with experts was the
preferred way to gain information, apart from using the Internet.
Page 77 Does Australia have the balance right between pre-market and postmarket regulation of medical devices?
We contend the answer is no. The level of post-market regulation is too low and could be improved by
proactively seeking feedback from consumers about their quality of life and experience post-interaction
with the medical device. At present this post-market regulation is done through adverse event reporting
which does not engage the consumer at an adequate level. The approach could be a simple, light touch
through online or phone reaching out to consumers placed on the register or commercially operated
database to ask a handful of simple questions that could be escalated if a negative situation is noted.
Essentially, the process of post-market surveillance is self-regulating but it is generally absent activity and
so its occurrence should be regulated.
Page 79 Has the regulatory framework for IVDs resulted in a reduced emphasis
on clinical best practice?
We contend that the question assumes that clinical best practice is determined through regulation. In an
environment that is moving toward self-care and autonomous decision-making, the market is likely to
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include an element of judgement by the consumer and not just on the accreditation of the process by the
regulator. Self-regulation should be considered but there needs to be also an element of science-based
evidence that is judged by an independent authority. As an example, we have relatively unregulated
environment of complimentary medicines making claims with very little scientific evidence. On the other
hand, it can be argued that the level of potential harm is low even though the level of potential benefit is
also low. The main risk is a waste of money! So the issue is how an appropriate balance is to be
established for information that is now available from diagnostic devices.
Our view is that judgement will occur as a partnership between the healthcare practitioner and the
consumer.
Page 83 Is the classification system to medical devices too complex?
We contend that the answer is yes and it might be simplified by reducing the concern for individual risk
and balancing it with the potential for public well-being. While this is contentious, it is argued that in the
area of information resulting from diagnostic medical devices, the benefits to the public far outweigh any
small risk to an individual. Furthermore, the risk to the individual can be minimised through the informed
consent process regulated through a partnership between the healthcare practitioner and the consumer.
The simplification can also occur through a focus on outcomes and not just the process. It may also be
simplified through grouping a battery of tests rather than segmenting out each individual test. While the
legal system has encouraged the patenting of individual tests, it is likely that the future will see a sharing
of this intellectual property and not a further segmenting of it into private ownership. Australia could
take a lead in this area by accrediting the process and not each specific test.
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The survey
The online survey was conducted across a potential cohort of 2150 respondents from January 2nd to
January 5th. A convenience sample was used but it is held to be generally reflective of the Australian
population, with a bias towards better education and technically literal people.
Further details are available on request.
Here are the questions used:
Insync Genetic Information Review
Regulation in the health industry
The community creates standards to safeguard itself and minimise risk. Many products and practices
in health are regulated.
Regulation in health protects the community against harm by checking and evaluating risk. New
technologies are not available until approved.
In your view, is the current regulatory system flexible enough to accommodate new
medical technologies?
Not sure
Yes
No
When making important DECISIONS about your health, how do you prefer the LOCUS
OF CONTROL to be spread?
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With the doctor
%
With me
%
With my family/carer
%
%
When having information about your health, who do you prefer to CONTROL ACCESS
to the information?
The doctor
Me
My family/carer
The regulator
On balance, which of the following statements do you prefer when determining the
QUALITY of the information regarding your health?
The regulator, at all times
Me, at all times
The regulator, when the risk of negative impact is significant
The doctor, at all times
The doctor, when the risk of negative impact is significant
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Your genome
Inside the cells of your body exists DNA, which is a code which strongly influences the way you grow
and function. It is now possible to read that code and gain a new level of understanding of the health
issues ahead in the hope of being able to do something about the quality of life for that individual.
The totality of your genetic information is called your genome.
Assume money and availability is not a limiting factor.
How interested are you in having your genome analysed?
1 = very low; 5 = very high
1
2
3
4
5
NA
Level of interest
The analysis of your genome may disclose information about your health that may be not otherwise
known. Some of this information may be potentially distressing.
What level of risk do you see in you HAVING INFORMATION about your genome?
1 = very low; 5 = very high
1
2
3
4
5
NA
Level of risk
In helping you evaluate the risk associated with information about your genome,
which of the following factors regarding the process of generating the information
would be more important?
Popular acceptance
Regulatory approval
Control of who has the information
The support of your doctor
Plain language communication
How securely the data is stored
Ethical acceptance
Full disclosure of the confidence underlying the information
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Full detail behind the information
Scientific acceptance
You can select up to 8 factors.
The analysis of your genome may be subject to error.
What level of concern do you have about potential errors in the information about
your genome?
1 = very low; 5 = very high
1
2
3
4
5
NA
Level of concern
Do you have any dependent children under 18 years of age living with you?
(an answer is required)
Yes
No
17
Your preferences
Preferences can differ widely, often with there being no 'right or wrong'.
To what extent do you agree or disagree with the following statements?
Level of Agreement
(1=Strongly Disagree; 5=Strongly Agree)
1
2
3
4
5
NA
I feel healthy
I have enough energy
I feel calm and peaceful
My health is consistent with my life goals
I feel confident in my ability to be healthy
It is very important for me to be as healthy as possible
I have decided that I want to be healthy
I have thought carefully about my health and believe it is important
for many aspects of my life
I try to do things that I believe are best for my health
I actively try to prevent disease and illness
I seek out health information that answers my health questions
Before making a decision about my health, I find out everything I can
about the issue
I know how to use the health information I find to help me
I feel confident in using information to make decisions about my
health
I really enjoy learning about health issues
18
Assume your genome is analysed. You can be told everything that has been found, ask to have only
certain information disclosed or have nothing told to you. For example, some people might prefer to not
know they have a life limiting condition.
What level of disclosure of your genome would you prefer TO YOU?
Full disclosure; all known shared
Partial disclosure; only information known for certain shared
Partial disclosure; only information which is determined by the regulator shared
Partial disclosure; only information which is determined by me in advance is shared
Partial disclosure; only information which is harmless shared
Partial disclosure; only information which is determined by the doctor shared
Partial disclosure; only information for which something can be done shared
No disclosure; nothing shared
Yes, you can only choose one! Apart from you, assume full confidentiality. Your doctor may also not
know.
Assume your child's genome is analysed.
What level of disclosure of your child's genome would you prefer TO YOU?
Full disclosure; all known shared
Partial disclosure; only information known for certain shared
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Partial disclosure; only information which is determined by the regulator shared
Partial disclosure; only information which is determined by me in advance is shared
Partial disclosure; only information which is harmless shared
Partial disclosure; only information which is determined by the doctor shared
Partial disclosure; only information for which something can be done shared
No disclosure; nothing shared
About you
Finally, so we can segment
the responses, here are some
simple demographic
questions.
In which year were you born?
What is the highest level of education you have completed?
Secondary school (Year 10 or below)
Secondary School (Year 11 or 12 equivalent)
Certificate or Diploma (e.g. TAFE)
Bachelor degree
Postgraduate qualifications
20
At the moment, how satisfied or dissatisfied are you with your general health?
Level of Satisfaction 1=Very Dissatisfied; 5=Very Satisfied
1
2
3
4
5
NA
Satisfaction with Health
Thank you. You now know we are interested in how you might wish your genetic information handled. Of
course this is a short survey in a complex area and we have been very specific.
Optional question: Please note any issue that you would like considered.
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