The Implications of Drug
Development in Practice
Legal and Ethical Issues at a Treatment
Centre
Rebekah Ley, LLB (Hons), MSc
Innovation and excellence in health and care
Addenbrooke’s Hospital I Rosie Hospital
Introduction
• Technology develops quickly, the law changes slowly.
• Medicine is practised with a level of uncertainty.
• Confrontation: medicine, society and law.
• Should medical ethics be formed by the law or the law
formed by medical ethics?
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Introduction
• Medicine operates within broadly stated legal.
• Move away from the duties of the medical professional
towards broad patients’ rights.
• Trust and respect between doctor and patient rather than
adversarial.
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Introduction
• Loss of control and certainty.
• Law and ethics can they help?
• Have in mind the non financial barriers that also come
into play here in particular international property rights.
• Big themes – small answers.
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Topic 1: Example
Lilly is two years old. Her parents count the months as other might
not, because each is previous additional time, bought at high cost.
She was diagnosed soon after birth with a neuroblastoma. She
received early treatment at Addenbrooke’s and was given the all
clear in June 2010. Then, unusually in such a young child, she had
a sudden and violent relapse. The NHS had nothing further to offer.
With the help of their treating oncologist they found the only place in
the world offering treatment for children whose neuroblastoma has
relapsed and spread across the blood/brain barrier. They took Lilly
to the Memorial Sloan-Kettering Cancer Centre in New York for
pioneering immunotherapy. The costs of Lilly’s treatment has so far
cost her family £610,000.
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Topic 1: Example
• Availability of information varies widely, increasingly
digital world with strong parental organisations.
• Differing standards for information across the world.
• Who is going to provide this information?
• As the treating team are you going to let parents and
some patients find out and interpret for themselves?
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Topic 1: Example
• What is the nature of the obligation?
• Legal obligation: provide care that is in the patient’s best
interests. No legal requirement to provide assist.
• Moral obligation is wider. Maintaining the therapeutic
relationship and the value of this.
Topic 2: How to approach families
• Carefully!
• Sources of information; national and organisations.
• NHS Choices provides some advice about seeking
treatment within the European Union but nothing specific
about treatments outside the European Economic Area.
• NHS funding treatment of abroad is variable
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Topic 2: How to approach families
• Unique vulnerability of children as research subjects,
being able to express needs and being able to defend
their own interests.
• Role of parents: decision to participate; thresholds of
understanding and complexity of research.
• Consent: process not a single event. Longer term
studies need to refresh. Child may develop ability to
consent.
• Classification of risk, high, low and minimal.
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Topic 2: How to approach families
• The discussions around care and treatment options
occur in a situation of extreme stress. Parents may feel
a lack of control and variable degrees of choice
regarding their child participating in a clinical trial.
• Informed consent – can parents distinguish between
treatment and trial participation?
• Researcher different from the treating clinician? Do
parents who simply want more time with their child need
that differentiation to help them understand the limits of
what is being offered?
• Losing a sense of normality leading to decisions to
accept or not participation in a trial.
Innovation and excellence in health and care
Topic 2: How to approach families
• Child truly as research participant rather than simply a
recipient of something that he or she may not
understand.
• Understanding the mutual obligations that participation in
research can bring.
• Understanding how research illustrates the expectations
and obligations that society places upon us.
• A child who can understand the suffering of other
children is much more than just a research subject –
altruism.
• Autonomy and medicine’s humanistic roots.
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Topic 3: Affordability NICE, evidence, cost/benefit
analysis
• Affordability or should we look at cost?
• In any health economy choices have to be made
between options when resources are finite (NHS) or
scarce (developing world).
• Not really about affordability, can’t judge affordability
unless you know the cost.
• Opportunity cost – I can’t do y because I am going to do
x.
• What are the benefits that I am giving up rather than the
cost of the thing itself.
• Direct, indirect and intangible costs.
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Topic 3: Affordability NICE, evidence, cost/benefit
analysis
• NICE limited societal perspective – direct costs versus indirect costs
to State.
• Benefits: units: years saved or utility units.
• Quality Adjusted Life Years (QALYs).
• Evidence: Particularly pertinent given the rarity and complexity of
childhood cancer meaning the need to travel often long distances
and the associated costs. Many countries may not be able to offer
some treatments given low volumes. Targeted research on rare
tumours genetically analysed can be effective but the numbers are
low and could be described as truly experimental.
• May be a move away from cost and benefit and instead ask the
question what are the added costs over and above those of the
existing treatment.
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Topic 4: Equity in a limited financial climate?
• Positivist legal approach to human rights embodied in international
law e.g. Universal Declaration on Human Rights and European
Convention on Human Rights.
• I do not believe there is a legal right to health or healthcare.
• Moral right to health and healthcare not an abstract right that is
independent of wider societal values.
• Example at the beginning: Can’t infer from the empirical fact that
because Lilly’s best chance of survival is to travel to New York that
she is entitled to it (has a right to it).
• The entitlement depends on all the other things we have already
talked about, effectiveness (evidence), resources we have and the
priority we give to Lilly as opposed to others; opportunity cost again.
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Topic 4: Equity in a limited financial climate?
• The word most often used in association with healthcare
it utilitarianism – the greatest amount of good for the
greatest number.
• Egalitarian: desire to treat equally or fairly.
• Fairness as Justice.
• Setting a healthcare budget utilitarian general priorities
handing the baton to the frontline Egalitarian.
• Utilitarian v Egalitarian (Fox or Hedgehog?)…..
• Meeting need depends upon evidence and the value
placed upon that evidence.
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Topic 4: Equity in a limited financial climate?
• The economics of research – some things to consider:
• Knowledge is a commodity; research and development
are inputs in the production of knowledge.
• Publicly funded Science? An exclusive dependence on
the public purse to finance R&D is problematic because
no one knows what the end result will be and sometimes
not even how to get there.
• Technology is market driven and enforced by the law;
Science is community driven and thus enforced by
norms – to a certain extent then the scientific community
here can set the “norms”.
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Topic 5: Access to drugs in developing countries
• The better question is, “how can the good or benefits
from medical research be maximised?”.
• This is to move away from the overriding concern to
protect research participants.
• Equal access to the benefits from research (back to
egalitarianism).
• Sustainability: a way of measuring the benefits and
finding ways to improve this.
• Asking: “when are inequalities in healthcare across the
globe unjust?”.
• Obligations on States and International Organisations in
setting priorities.
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Topic 5: Access to drugs in developing countries
• Lymphoma more common in Rwanda than the United
States.
• Rwanda no trained paediatric oncologist.
• In the West 80% of children can be cured of lymphoma,
this requires diagnosis, chemotherapy and follow-up.
• Project twinned Dana-Faber/Children’s Hospital Cancer
Center with a hospital in Rwanda to provide care for 10
children with lymphoma.
• Five are considered cured, two are in remission, two
died from treatment related complications and one from
lymphoma which progressed through treatment.
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Contact details
Rebekah.ley@addenbrookes.nhs.uk
Innovation and excellence in health and care