Since we reported on the 2002
annual conference of the International Society for Technology Assessment in
Health Care (ISTAHC), the organisation has metamorphosed into Health Technology
Assessment international (HTAi) - a new organisation with a new Board and one
now very much more interested in including the industry as a major stakeholder.
HTAi's mission is 'to support the development, communication, understanding and
use of HTA globally as a scientifically based means of promoting the
introduction of effective innovation and the effective use of resources in
health care'. For more details please see
www.htai.org. This year's
annual conference was held in Poland with some 600 delegates gathering in the
medieval city of Krakow and opened by Lech Walesa, winner of the 1983 Nobel
Peace Prize and former President of Poland. Here in this conference report, we
will focus on a main conference theme - that of increased collaboration with
the industry. We start with a look at some of the more relevant pre-conference
workshops and satellite meetings.
Pre-Conference Workshop:
Collaboration across the Board in HTA
This year I was asked to
organise a pre-conference workshop on collaborative working within the field of
HTA. Certainly it can be argued that for health technology assessment and
health technology appraisal to be at their most fruitful, collaboration amongst
all stakeholders is essential. But what does such collaboration look like? And
why are there often barriers to such collaboration? The programme is briefly
described in Box 1 and here we give a brief overview.
At this
session, speakers from various HTA Agencies and Industry described their
experiences with regard to collaboration around HTA, lessons learnt and
possible ways forward. I kicked of with some thoughts on partnership and the
differences to sponsorship trying to point out that real collaborative working
was about equal partnership wherever possible. Potential conflict though does
sometime arise from a clash of 'value sets' and the different agendas
operating. Jose Valverde described the history of collaboration within his
Agency and went on to describe how a more transparent framework is being
developed with the purpose of designing new models of collaboration. The Swiss
Network of HTA co-ordinates HTA activities in Switzerland and there has been a
long-standing tradition in working with the industry. Maya Zullig said that
they too were looking at repositioning this to achieve better collaboration and
that this might include collaboration in jointly designing RCTs. She said that
there were still difficulties in understanding each others roles and
responsibilities and that there were real difficulties sometimes when marketing
were too deeply involved!… She also described how increasing budget
constraints in most fields of heath care are making a 'relaxed' relationship
more and more difficult. Andrea Rappagliosi then described some proposals to
enhance the positive experience that can be gained from collaborative working.
He said that in an over regulated system, and with costs rising to market,
appraisal rather than assessment was more the key issue and that common and
transparent methodologies were required. He passionately described the
'conception' of Serono's new 'babies' and their launch onto the market and that
he very much wanted to continue to nurture their growth and development under
the newer 'adoptive parents'….. To continue the analogy, he felt that the
baby may well be thrown out with the bath water if HTA was misused…. He
also suggested collaboration and partnership around the area of trial design
and that HTA should be an opportunity to break down silos. Mike Rawlins
outlined the UK experience, which is likely to be very well known to CRf
readers! Brendan Barnes then outlined the views of EFPIA in terms of moving
forward on collaborative working in HTA. He was clear to point out that
innovation must be rewarded and barriers should not be put in its way and that
there were both merits and limits to HTA as well as 'good' and 'bad' HTA. He
added that the credibility of HTA rested on appropriate processes and
implementation by responsible authorities, and if not, that this would likely
give HTA a bad name. Certainly he worried that political pressures in the EU
had the potential to derail HTA and so dialogue was absolutely important. And
finally here, Chris Henshall outlined some proposals as to how HTAi might
encourage collaborative working in HTA. He was keen to point out that although
there were many challenges facing those working in HTA (in both the Industry
and the public sector), an increased understanding between both parties would
surely help better manage risk and yet maximise the benefits of innovation
within available resources.
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Box 1. |
Before the main conference,
there were also two industry sponsored sessions and it is well worthwhile
reporting on these too. For brief outlines see Box 2 &
3.
MSD satellite symposium
This symposium explored
the 'patient voice' in HTA. The session 'blurb' was keen to point out that
whilst HTA recommendations were based on thorough evaluations of
cost-effectiveness and clinical effectiveness, they often lacked the
perspective of the end-user of those therapies, the patient. The symposium
sought to identify opportunities to include patients in the HTA process,
understand patient preferences in HTA decision-making, provide examples of
effective patient involvement in HTA and to review best practices of patient
involvement. By incorporating patient preferences and values into the
evidence-assessment process, it was suggested that evaluators could then ensure
that their recommendations on the uptake of effective technology gave proper
consideration to society's views on the value of a medicine or therapy.
Further, by providing reliable, accurate and balanced information about
medicines and therapies, governments and manufacturers could also ensure that
patients, as experts on their own diseases, could be effective partners in the
HTA process. Please see Box 2.
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Box 2 |
GSK/Lilly satellite
symposium
This symposium aimed to explore partnership in the
evaluation and assessment of the value of medicines. We will describe this
session in a little more detail.
Professor John Gabbay first described
how modern health care was increasingly being shaped by technological
innovations and that in response, HTA had grown rapidly in methodology and
scope over the past three decades. But beset at every stage of its work by a
range of pressures and interests, HTA has had to steer a tense line between
science and policy making. Prof. Gabbay pointed out that whilst it aimed to
meet the needs of the patients and of health care systems, HTA also now
impacted strongly on the industry. To continue its success, he suggested, HTA
must meet an increasing challenge to provide appropriate, usable and
policy-relevant analyses in complex environments, whilst avoiding
'polarisation' and that HTA would 'hold the line' if it managed to hold on to
all the partnerships. He particularly wanted more reciprocal relationships with
the industry.
Professor Adrian Towse explored the use of
pharmacogenetics to target drugs and the potential implications for industry
and HTAs. In reviewing the potential, he said that there were assorted
positives (decrease in R&D costs) and negatives (loss of broad market
utilisation) for the industry to consider. There are also new valuations that
HTAs will need to take into account (e.g. decrease in adverse reactions and
reduced drug spend for patients who will not benefit) as the industry attempted
to provide more targeted and effective medicines.
Dr. Rod Taylor
outlined how in spite of the long tradition of quality, safety and efficacy in
licensing, that such evidence was no longer sufficient to ensure market access
for medicines…… He described how HTA was increasingly being used by
health policy makers to source their additional evidential needs when facing
reimbursement decisions. Dr. Taylor also outlined a changing paradigm on HTA,
which is now moving from a focus on systematic reviews over a 12-24 month
period and driven by a research question to one where the timescale was more
like 6-12 months and was now policy driven. He pointed out that this would
cause problems for the industry unless it was increasingly engaged and
suggested that there was now an increasing requirement for industry to play an
explicit role within the HTA process. Dr. Taylor's presentation was the most
contentious as he heavily criticised placebo controlled trials (not real
world), bias because of non-reporting of 'negative' results and wanted to see
improved (industry) modelling…. Clearly 'value' was in the eye of the
beholder here as a rather 'parent-child' approach was being taken by Dr.
Taylor. The proposed new relationships would appear to be all one way and it
was not clear exactly what was being given in return, with 'terms' seemingly
already set and with little negotiation - doesn't sound like a collaborative
partnership to me! One wonders then what proactive steps agencies and
researchers are actually taking in helping companies respond to these new
challenges and to nurture real partnerships. This is a step change for the
industry- it's revolution, not evolution….
And finally Panos
Kanavos, Lecturer in International Health Policy at the London School of
Economics, discussed approaches to pharmaceutical pricing and reimbursement in
the new accession countries. These approaches were then compared with those
prevailing in the previous EU-15. The emphasis does appear to be on cost
containment as governments attempt restructuring and rationalisation of their
healthcare systems and he suggested that there was a real danger here around
the possible misuse of HTA in the new member states. Please see Box
3.
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Box 3 |
Some reflections on the main conference
The opening plenary session included a fascinating talk by Dr. Chris
Henshall, HTAi President, who looked at HTA - past, present and future. The
present position seems to be one of sophisticated methods for trials,
meta-analysis and economic modelling of therapeutic and preventive
interventions but where the area is weaker on diagnostics procedures and on the
assessment of the 'wider consequences' of technologies. There are varying
levels of engagement by clinicians, patients and industry in assessments and
health care systems are increasingly seeing HTA as a key input to decisions on
use of resources. Dr Henshall was clear that he wanted HTAi to be a meeting
place for all disciplines, including folk like ethicists, lawyers and
sociologists but particularly the industry, which he felt needed to up its
'game' in terms of understanding and engaging with the HTA agenda. He then
outlined the world ahead where public-private partnerships would place an
increasing emphasis on collaboration between public and private sectors. The
challenge ahead for HTA then was one about being a tool that allowed folk to
balance the desire for effective and affordable healthcare with the need for an
innovative and profitable healthcare industry sector. But to do this, he
suggested, there would need to be further developments in the way that HTA was
undertaken and used in decision-making. So in terms of doing HTA, he felt there
was now a need to put the 'T' back into HTA - i.e. to put a fair assessment on
the innovation component, particularly in an increasingly knowledge-based
economy. And in terms of using HTA, there needed to be more and earlier use by
industry in order to identify the most marketable innovations. There also
needed to be more consistent and flexible use of HTA in healthcare systems in
order to assess known and possible benefits, costs and the wider implications
at different stages of intervention/technology life cycles. But Dr Henshall
again felt that current HTA methods do not yet meet the needs of
decision-makers trying to address these challenges and that the necessary
methodological developments required a call for collaborative work between all
stakeholders. There was a need too to use HTA along the development path of an
intervention, not simply at the point of launch and to share risks in cases of
uncertainty. The next steps then should be collaboration between manufacturers
and health systems to better manage and share risk and to maximise the benefits
of innovation within available resources. So HTA needs to become "a process
undertaken and used in collaboration by HTA experts, industry, patients, and
health care systems to inform decisions about the development, marketing,
uptake and use of drugs, equipment and procedures throughout their life-cycle,
with the aim of promoting useful innovation and value for money in health
care". In closing, Dr. Henshall expressed his wish for HTAi to provide a forum
for all stakeholders to come together to discuss these necessary developments
aimed at achieving both innovation and the cost effective use of resources in
health care and the development of HTA methodologies to support such decisions.
And on this whole area of innovation and HTA, one slide put up the conference
and which created a great deal of interest was that of the proposed new
National Health Technology Strategy in Canada. This nicely positions HTA within
the whole R&D process.
Parallel session on Industry
Collaboration in HTA
During the main conference, there was also a
parallel session on Industry collaboration in HTA. My role was to 'provoke'
debate and discussion after the speaker slots and that certainly happened!
Several of the speakers already mentioned were again involved. Please see
Box 4. Just to pick up on a few highlights here. Dev Menlon described
the places in the HTA 'cycle' where collaboration was possible and outlined
examples of 'meaningful' collaboration, including identifying priorities and
defining the questions, as well as conducting assessments, funding and
disseminating results. Certainly the Institute of Health Economics appears to
very much put partnership into practice as the organisation involves all
stakeholders as equals including industry as well as government, universities
and health authorities. And he closed by saying that there was certainly room
in Canadian health technology strategy for industry participation.
Dr
Taylor looked at the pros and cons of HTA-Industry collaboration, the added
value of industry collaboration and examined why there were concerns with
industry collaboration. Like many he agreed that there was a major opportunity
for collaboration in implementation and he talked about co-marketing where
companies might support the NHS in implementing technology decisions. There was
also probably more room for risk-sharing schemes as with UK Department of
Health MS scheme - this about outcome guarantees and outcomes audit. Dr Taylor
though was again keen to point out that there was evidence which appeared to
show bias in industry sponsored trials and that issues still remained over
confidentiality versus transparency.
Bridgitte Casteels stated that some
kind of mutual agreement about methodologies was required. She certainly felt
that the increased burden on the industry was a problem in terms of faster
patient access to innovative therapies as the burden of proof was often placed
exclusively on the manufacturer. But she felt the industry just had to 'ride
the wave' and must get more proactively involved in the evolution of HTA and
its uptake in policy making. She challenged the audience on bias (why should we
produce biased data?, she asked) at which point one delegate exploded with some
anger…. The interactive session then followed and I adopted a 'Kilroy-Silk
style' taking an 'active' microphone into the audience…. The discussion
continued to be very lively!…
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Box 4 |
Submitted Papers Session on Effective
Dissemination of HTA
In this parallel session there was a very
interesting paper presented by Kevin Loth, Director of European Public Affairs,
Novartis, well worth picking up on. Kevin had done an MBA on NICE and its
impact and effects on local prescribing decisions within Primary Care
Organisations (PCOs) in England and Wales. It created a lot of interest as the
study illuminated quite clearly the area of so-called NICE blight. Briefly, a
structured questionnaire was sent out to all PCOs at the end of 2002. The
results showed that some 95% of PCOs thought that NICE had changed the way that
local prescribing was managed and around 50% had curbed investment in products
not subjected to a NICE appraisal. Of particular interest was the fact that 50%
of PCOs said that there was a moderate to strong influence at DH referral point
to NICE; 70% moderate to strong at FAD stage and 77% strong at final guidance
stage. One of the main conclusions appeared to be that NICE has had a high
'cognitive' impact on prescribing within PCOs and that if this led to
behavioural change, as suggested by change management theories, the true impact
of NICE on the use of pharmaceuticals may yet to be realised. But also clearly
NICE seems to having too big an impact too early. And Kevin thought to date
that the industry has applied a 'surprising lack of imagination' and resources
to the issue of NICE implementation!…
And very finally, there were
several interesting poster sessions. Just to pick up on one from York
University's Centre for Reviews & Dissemination (CRD). Their poster was on
'Newer drugs for epilepsy in adults: how good are the trials from a systematic
review?'. As folk may know, NICE has issued guidance on these drugs but still
the CRD heavily criticised the studies they found, "….much of the
available literature is of poor quality and has limited applicability when it
comes to informing clinical practice about the optimal use of newer AEDs. Many
trials failed to meet international recommendations on clinical trials (number
of patients, duration, etc)…The true methodological quality of trials was
difficult to assess due to poor reporting….". So despite all the stuff on
collaboration at the conference, some folk still seem to have antibodies' to
industry-sponsored' trials….
Some implications in all of
this
So what to make of all of this? Well bottom line, perhaps
clinical researchers should be more aware of these wider developments around
HTA. Also with HTAi now saying that it wants a more constructive relationship
with the Industry, perhaps clinical researchers should get engaged here too.
HTAi President Dr Chris Henshall is based in London and is very happy to talk
to folk about this.
More generally, clinical trials are of course
increasingly being conducted in the CEE countries where HTA is definitely now
on the map… Transferability of data is still a hot issue within the HTA
fraternity as is generalisability of RCTs submitted to regulatory authorities.
Perhaps this needs factoring into trial design?.. Also what about the balance
between UK-based data for UK consumption and UK-based data for 'export'…..
The data maybe OK for registration but it will certainly be examined with a
fine toothcomb for HTA purposes and it may well be dismissed….. Folk
should perhaps be aware of this when designing clinical trials?
It was
interesting that Prof. Kent Woods made similar comments to Dr. Henshall in the
June issue of CRf regarding the importance of a thriving and innovative
pharmaceutical and medical devices industry in a modern, knowledge-based
economy like the UK. He also outlined how the MHRA lay at the very centre of
rapid changes in UK national healthcare and healthcare policy and it is this
author's belief that clinical researchers perhaps need to lock into this area
too. For example, just as most folk were getting ready to go off on their
summer holidays came the decisions by government over the rationalisation of
the so-called 'Arm's Length Bodies'. Although the MHRA will continue its role
unchanged, do please note that the National Patient Safety Agency will now take
responsibility for the Central Office of Research Ethics from the DH and take
the national lead in supporting the development of ethics committees. And this
is not to mention the brand new NHS payment by results financial flow system
and national reference prices and healthcare resource groups… possibly to
be factored into clinical trials?…
As in previous years, industry
attendance was disappointingly low (apart from Poland!) and I just don't
understand this. There were in fact around 60 delegates from the UK including
many significant 'movers and shakers' like Andrew Dillon and Mike Rawlins from
NICE (in fact NICE had a very high exposure at this year's conference) and Kent
Woods from the MHRA. There were just two UK pharmaco delegates, who seem most
pleased to have been at the conference, viz:
"This conference proved
to one of the best networking conferences that could be attended. HTA officials
stated they have a lot to learn from industry in how to promote their messages.
This congress provided an exciting forum to engage in". John Littlefield,
NHS Strategy Manager, Altana Pharma, UK
"This conference was
invaluable in terms of sharing ideas and engaging with a wide spectrum of HTA
stakeholders. It is vital that all elements of the UK HTA community actively
participate in the ongoing debate on the future shape and direction of HTA"
Sarah Cotton, NICE Policy Manager, MSD, UK
Next year's Annual Conference
of HTAi is in Rome from 20th-22nd June 2005. Please put it in your
diary…....
Article first pulished in Clinical Research focus - October 2004