A discussion of the roles patients can play. Nick Meade Director of Policy Genetic Alliance UK

1. Patients’ voice in access decisions
A discussion of the roles patients can play
Nick Meade
Director of Policy
Genetic Alliance UK

2. Genetic Alliance UK is the national charity working to improve
the lives of patients and families affected by all types of
genetic conditions. We are an alliance of over 180 patient
organisations.
Our aim is to ensure that high quality services, information and
support are provided to all who need them. We actively
support research and innovation across the field of genetic
medicine.
Who are we?

3. • Patient voice as information providers
• Patient voice in decision making
• Patient voice in policy development
• Patient voice as an individual
Patients’ voice in access decisions
A discussion of the roles patients can play

4. “Health technology assessment is the systematic evaluation
of the properties and effects of a health technology,
addressing the direct and intended effects of this
technology, as well as its indirect and unintended
consequences, and aimed mainly at informing decision
making regarding health technologies.”
World Health Organisation
Raw material: information
Patient voice type 1
Information providers

5. Some types of information required by HTA bodies:
•Population size? How many people are affected? How many people need / want
the intervention?
•What is the impact of the condition? Burden of illness?
•How does the disease progress? At what stage should the treatment be given?
•What are the adverse events associated with the treatment? What is their impact?
•How will the treatment be delivered within the health service?
•How effective is the medicine? Is it uniformly effective? Does it work better in
certain circumstances?
•How much does it cost? What is the value of the improvements it makes?
Are these questions valid? Can we answer them in the case of the treatment under
discussion?
Patient voice type 1
Information providers

6. Example from:
The Society for Mucopolysaccharide Diseases (the MPS Society) is the only UK
charity supporting individuals and families affected by MPS, Fabry and related
diseases.
Response to scoping for NICE’s Highly Specialised Technology evaluation of
Elosulfase alfa for treating mucopolysaccharidosis type 4A
Patient voice type 1
Information providers

7. What is the population size?
NICE’s DRAFT Scope for Elosulfase alfa for treating mucopolysaccharidosis type 4A
“The exact prevalence of mucopolysaccharidosis type IVA is not known. It has been
estimated to affect approximately 1 in 250,000 people in the UK, with 1 new
diagnosis per 220,000 live births. This equates to about 200 people living with
mucopolysaccharidosis type IVA, and approximately 3 new diagnoses per year, in
England.”
MPS Society’s response:
“The MPS Society has had an MPSIVA registry since 1984 and has collaborated
with clinical sites to ensure accuracy and completeness. 94 people living with a
biochemical diagnosis in England are on the MPSIVA registry and we do not
believe any are missing.”
Patient voice type 1
Information providers

8. What outcomes should be considered?
NICE’s DRAFT Scope for Elosulfase alfa for treating mucopolysaccharidosis type 4A
“The outcome measures to be considered include:
MPS Society’s response:
“Please consider:
But is it easy for patients to play this vital role?
Patient voice type 1
Information providers
• mobility
• respiratory and cardiac function
• growth and development
• vision and hearing
• sleep apnoea
• mortality
• adverse effects of treatment
• health-related quality of life (for patients
and carers).”
• fatigue
• pain
• endurance.”

9. Patients are a key stakeholder in healthcare. They should play a role in decision
making.
Patients and patient groups understand the potential beneficiaries point of view
• Is the patient submission being considered appropriately?
• Are all potential benefits from this medicine being valued equally?
• Just how valuable is a shift from daily treatments to weekly?
Patient participation in marketing authorisation decision making has changed the
culture of EMA scientific committees.
• At least one trained disinterested patient should be part of the decision making
panel.
Patient voice type 2
Decision maker

10. NICE’s HST Evaluation Committee has:
3 lay members (including someone with knowledge / experience of
ethical issues)
• Professional ethicist
• Member of public
• Patient representative (conflicted for one of the products!)
Scottish Medicines Consortium
2 public partners
• One of whom has been public partner for Healthcare Improvement
Scotland for more than 10 years
Patient voice type 2
Decision maker

11. European Medicines Agency committees:
• Pharmacovigilance Risk Assessment Committee – one patient member
and one alternate
• Committee for Orphan Medicinal Products – three patient members
(including the co-chair)
• Committee for Advanced Therapies – two patient members and two
alternates
• Paediatric Committee – three patient members and three alternates
• Committee for Medicinal Products for Human Use (CHMP) – one patient
observer (new this year)
The membership structure of all of these committees are the result of
primary legislation at the European level.
Patient voice type 2
Decision maker

12. Patients are a key stakeholder in healthcare. They should play a role
in designing decision making processes.
Developing the system in isolation disenfranchises the patient
community and is likely to result in a poorer design.
Patients just like other stakeholders will have experience of these
programmes and how their voice has been dealt with in the past. This
should be made use of.
This is how we change how patient voice contributes to HTA decisions!
Patient voice type 3
Policy maker

13. The most important patient voice in the whole process is that of the
individual making a choice to take a medicine.
Where possible, patients should be empowered to make an informed
choice as to whether to take a medicine, as part of a shared decision
with their clinician.
Patient voice type 4
The individual

14. Patient voice:
• as information providers
• as decision makers
• as policy makers
• as individuals
Conclusion
At least four types of patient
voice: ALL must play a role

15. You can download a copy of our Patient Charters on access to
rare disease medicines in England here:
www.geneticalliance.org.uk/hstcharter.htm
www.geneticalliance.org.uk/nhsenglandcharter.htm
Acknowledgements
• All those that participated in the workshops
• All colleagues that have worked together with us
• NICE and NHS England
Links and acknowledgements

16. Thank you!
Nick Meade
Director of Policy
+44 (0) 207 704 3141
nick@geneticalliance.org.uk
Registered charity numbers: 1114195 and SC039299
Registered company number: 05772999