Genetic Alliance UK Patient Charter: Patient perspectives and - - PowerPoint PPT Presentation

A consolidated patient perspective on the HST process

Nick Meade Director of Policy Genetic Alliance UK

Findacure: Navigating the Highly Specialised Technologies Programme, 29th January 2016 Genetic Alliance UK Patient Charter: Patient perspectives and priorities on NICE’s evaluation of highly specialised technologies

Funding for Genetic Alliance UK

• Resource for the running of the
• rganisation and its projects comes

from a wide range of funders, including national governments, the European Union, the pharmaceutical industry, medical research charities, and other grant provision organisations.

• All of our income is reported here:

www.geneticalliance.org.uk/annual-report.htm

Ethical collaboration policy

• All activities are defined and

independently designed by the staff with the approval of our trustees.

• All of our funding relationships

are governed by our ethical collaboration policy, which prohibits funders from influencing the outcomes of our

• work. This can be read here:

www.geneticalliance.org.uk/ethicalcoll aborationpolicy.htm

Declaration of interests

What is a Patient Charter?

• It is a tool that enables us to

collate and communicate a patient-driven policy position

• n a specific issue
• This is expressed in the form
• f recommendations
• Patient groups are invited to

‘sign up’ to show their support

How does it work?

• Patient representatives take

part in a workshop

• They are given a detailed

briefing document

• The day includes informative

talks and facilitated work- group discussion

Developing the HST Patient Charter

• Representatives from 16 patient organisations attended the

workshop

• Each were provided with a comprehensive

briefing document explaining the HST evaluation process and signposting the key issues

• On the day, we had talks from:

The HST Patient Charter workshop

Josie Godfrey NICE Jon Sussex OHE Dr Robin Lachmann Consultant Dr Will Evans Parent

What next?

• Patient representatives the took part in work-group disucssion

sessions facilitated by: Dr Will Evans Dave Ryner Robert Meadowcroft Jayne Spink

• Four key topics were identified – each group addressed at least
• ne of these each and then raised any other issues or concerns

The HST Patient Charter workshop

Scope and transparency Patient evidence submission Evidence gaps

• The minutes from each work group were compiled to ensure all the

issues, concerns and proposals for change raised by patient representatives were captured

• This information was then formatted into a Patient Charter by

Genetic Alliance UK before being made available to work group facilitators for editorial comment:

• Comments were incorporated into the final document
• The Patient Charter was then circulated around the patient group

membership of Genetic Alliance UK for endorsement....

Collating the findings into a Charter

“I thought that the draft was really most excellent and that you captured not only the discussion of the day itself but also place this very neatly into the policy landscape.”

Jayne Spink, Tuberous Sclerosis Association

• Social Value Judgements: Principles for the Development of NICE’s Guidance. NICE, 2005, available at:

www.nice.org.uk/media/c18/30/svj2publication2008.pdf

• M. Rawlins et al., Pharmacoeconomics: NICE’s approach to decision-making. British Journal of Clinical

Pharmacology, 2010, available at: www.ncbi.nlm.nih.gov/pmc/articles/PMC2949905/

• Citizens Council Report, Rule of Rescue. NICE, January 2006, available at:

www.nice.org.uk/niceMedia/pdf/Rule_of_rescue_report_final_0606.pdf

• Citizens Council Report, Inequalities in Health. NICE, June 2006, available at:

www.nice.org.uk/niceMedia/pdf/CCreportonHealthInequalities.pdf

• Governmental willingness for ‘appropriate priority’: House of Lords (2013) Hansard, 28 February, Column 1161

[Online], available at: www.publications.parliament.uk/pa/ld201213/ldhansrd/text/130228-0001.htm

The premise

It is right that HST evaluation exists as a means of ensuring that the NHS can commission effective, highly priced medicines for small populations

There is public, patient and governmental support for the NHS-wide commissioning of highly priced medicines for small patient populations with serious, life-limiting conditions. This is the case particularly when the condition affects children.

Key message

Genetic Alliance UK’s Patient Charter shows patients are concerned that, while greatly needed, the existing process is opaque, potentially disruptive to the wider NHS and risks downplaying the patient voice.

Patients are worried about the government and NICE's new approach to evaluating new medicines for very rare diseases. The Patient Charter makes 29 recommendations for change and is endorsed by 78 patient groups. Genetic Alliance UK hope it will act as a reference point for the patient perspective on HST evaluation and inform NICE’s review of the process, which is expected before the end of 2014.

• Potential of straying into risk / benefit analysis
• Risk of downplaying the patient voice
• Definition of clinically distinct is outdated
• Potential disruption to the wider NHS
• Lack of transparency in the topic selection process

Key findings

The process of choosing which drugs to evaluate is a black box. In contrast to the remainder of HST evaluation, the topic selection process lacks transparency. Patients are neither involved in the process nor able to access the deliberations of NICE’s topic selection committee.

• Recommendation 7 calls for the process, methodology and deliberations
• f the topic selection committee to be made available to patients and

the public online in a format that can be searched by the name of a condition or a medicine.

• The recommendation includes the need for clarification on the role of the

NIHR Horizon Scanning Centre in the process of topic identification.

Lack of transparency in topic selection

Topic selection at the beginning of a maze...

Transparency of Highly Specialised Technology topic selection at NICE

Pre 2015 2015 HST Topics considered 24 9 Results Proceed to develop topic 7 8 Do not proceed 5 20.8% 1 11.1% Confidential 12 TA Topics considered 311 120 Results Proceed to develop topic 181 115 Do not proceed 16 5.1% 2 1.6% Confidential 114 Filtered out 3

Transparency of topic selection at NICE 2015 onwards

Following the consultation on the ‘guide to the processes of technology appraisal’ one of the clear messages that came through was from pharmaceutical companies, professionals and patient groups that there was a lack of information publically available on the progress of topics through topic selection at NICE. Therefore the reporting of topic progress on the NICE website is now more open and transparent for technology appraisal and highly specialised technology topics, supporting NICE’s core aim of openness and transparency. This has been agreed following extensive negotiations with the Department of Health, Association of the British Pharmaceutical Industry (ABPI) and the Ethical Medicines Industry Group (EMIG) (as the representative organisations for pharmaceutical companies in the UK). All decisions from January 2015 onwards will be published in this format. NICE website

Transparency of topic selection at NICE 2015 onwards

Transparency of topic selection at NICE 2015 onwards

Transparency of topic selection at NICE 2015 onwards (Single and Multiple Technology Appraisals and Highly Specialised Technologies)

129 topics considered 123 have the following entries: Decision: Topic prioritised for potential technology appraisal guidance production, or Topic appropriate for potential highly specialised technologies (HST) guidance development Rationale: Anticipate the topic will be of importance to patients, carers, professionals, commissioners and the health of the public to ensure clinical benefit is realised, inequalities in use addressed, and help them make the best use of NHS resources Next steps: Topic passed to scoping team to prepare for a consultation exercise 114 of those have the following next step: Further detail to be added after consultation exercise

Transparency of topic selection at NICE 2015 onwards (Single and Multiple Technology Appraisals and Highly Specialised Technologies)

6 negative decisions in 2015 1 HST – Topic not prioritised Reason: Anticipate NICE guidance will not add value. 5 Single and Multiple Technology Appraisals 3 x Filtered out: out of scope, another work programme, duplicate 2 x Topic not prioritised Reason: NICE has to prioritise its topic selection decisions as it has limited resources and cannot consider everything. The level of the burden of disease and patient numbers may mean conducting an appraisal would not

• ffer good value for money

Transparency of topic selection at NICE

Proposals:

• Link the topic selection for all programmes, and be flexible with capacity

for both.

• Describe why each topic is suitable or unsuitable for a next step.
• Increase transparency of (and ease of access to information about) steps

before and after the topic selection step.

• ...wait for the Accelerated Access Review findings!

You can download a copy of the Patient Charter here: www.geneticalliance.org.uk/hstcharter.htm Our NHS England Patient Charter is here: www.geneticalliance.org.uk/nhsenglandcharter.htm For more information please contact: Nick Meade (Director of Policy): nick@geneticalliance.org.uk

• All the patient groups that helped inform and shape the Patient Charter,

especially all those individuals who attended the HST workshop.

• Dr Will Evans (Niemann-Pick Disease Group (UK)), Robert Meadowcroft

(Muscular Dystrophy Campaign), Dave Ryner (CML Support Group) and Jayne Spink (Tuberous Sclerosis Association), for facilitating work group discussions and providing editorial comment during the development of this Patient Charter.

• Josie Godfrey, Dr Robin Lachmann (National Hospital for Neurology and

Neurosurgery) and Jon Sussex (Office of Health Economics) for their informative contribution to our HST workshop day and afterwards.

• The ABPI and BIA for hosting the Patient Charter launch event, particularly to

Steve Bates (BIA) and Frances Macdonald (ABPI) for their helpful contribution to the panel discussion.

• Finally, our thanks go to Alexion and Pfizer for funding the project .
• Genetic Alliance UK team, past and current.

Acknowledgements

Thank you!

Nick Meade Director of Policy 020 7704 3141 nick@geneticalliance.org.uk Registered charity numbers: 1114195 and SC039299 Registered company number: 05772999

The impact that HST Evaluation will have on other aspects of care for rare disease patients is unclear. Patients are anxious that HST evaluation may sometimes hinder drug access, either directly with poor quality decisions, or indirectly due to NICE- mandated commissioning pushing other, currently prescribed treatments beyond the reach of the NHS budget.

• Recommendation 2 asks that consideration be given to the potential

impact of a NICE recommendation on access to medicines during topic selection, when determining whether HST evaluation is the most appropriate route to commissioning.

• Recommendation 3 calls for continued dialogue between NICE and NHS

England, and specifically for representatives of the NHS specialised services team to be present at all Evaluation Committee meetings.

Potential disruption to the wider NHS

NICE is using a definition of ‘clinically distinct’ does not recognise patient subgroups defined by symptoms, genetics or biomarkers This means that many medicines are currently ineligible for HST evaluation as only if the overarching condition is sufficiently rare to qualify for HST is it eligible – regardless of the size of the relevant patient subgroup.

• Recommendation 11 calls for the definition of ‘clinically distinct’ to be

updated to better reflect our understanding of diseases and to ensure the framework is ‘future proof’

• The need to robustly and effectively appraise medicines for small

patient populations will become increasingly important as we move towards more stratified medicines.

NICE’s definition of clinically distinct is outdated

Without targeted support, the patient voice will be absent from HST evaluations. This will happen if a patient group does not exist and/ or if small, poorly resourced groups are left to develop their evidence submissions alone. NICE has yet to make provision for any contracted, third-party support.

• Recommendation 4 asks support for patient communities involved in HST

evaluation to form patient groups if one does not already exist

• Recommendation 5 calls for NICE-contracted third-party support to be

made available to all patient groups to ensure a ‘level playing field’

• Recommedation19 asks for NICE to feedback to patient groups how

their evidence submission was used so that patient groups can learn from each other and share best practice

Risk of downplaying the patient voice

Is it appropriate that national HTA bodies such as NICE are able to effectively overturn a decision already made at the EMA by blocking patient access on the basis of perceived unacceptable risk? At the workshop. Patient representative were keen to ensure that NICE’s decision not to approve a drug would not be based on it being ‘too risky’ for the NHS to spend money on.

• Recommendation 28 calls for NICE to explicitly justify and involve

patients in any reexamination of risk they think is necessary beyond that already carried out at the EMA

• Recommendation 29 states that patients should ultimately decide

whether the benefit of a new treatment outweighs the risks

The role of NICE in risk versus effectiveness