Sarcoma SPAEN Conference – Day 1

This is the third year I’ve attended the SPAEN conference. The three days are always action packed, exhausting, thought provoking and inspirational. The delegates attending are from throughout Europe and most of whom have a personal interest in improving the cancer landscape. There are a great many organisations represented and crossing borders, sharing knowledge, experience and information. Importantly working collaboratively on projects, without ego or personal gain. There’s a great deal that charities and businesses alike could learn from this group!

This blog entry is taken from my extensive notes at the conference and I hope will be useful to many. I am quite sure that SPAEN will soon post their own conference report on their website so please be sure to check back.


The Opening Welcome was given jointly by Roger Wilson and Markus Wartenberg

It is a testament to the organisation that this is the fourth year this conference has taken place and membership, interest and outcomes continue to grow. Despite being a European conference we have this year also attendee representatives from USA, China and Australia. Their aim of attending is to understand how a patient group like SPAEN operates, listen to stories and examples from attendees and take back these learnings to their own countries.


Raz Dewji, Director – Clinical Scientist, GlaxoSmithKline Oncology Global Medical Affairs, UK
GSK Europe welcomes SPAEN to UK London as the main conference sponsor

“A personal perspective on the impact the ‘Patients Voice’ in drug development”

A journey that has been incredibly emotional. I’ve learnt how important it is to understand and hear what patients are asking for. Hopefully some of the work that we are doing and other companies are doing is now reflecting the voice that you collectively and as individuals have been delivering at different levels of the few years that we have known each other. I feel very humbled.

A little bit about GSK
GSK is a science led global healthcare company researches and develops innovative medicines, vaccines and consumer healthcare products.

Mission statement – do more feel better and live longer.

A lot of you have contributed in some shape or form to the journey I’ve had in the past 7 years and hopefully able to share some of those insights with you.

Less than 10% of patients with rare diseases are ‘treated’ on a global basis.

GSK concentrating a team on 200 different rare diseases.

Patient centric medicine is an area that is becoming much more focussed and a growing need to understand the patient.

Partnerships between organisations like GSK (Pharma) and patient advocacy groups are critical to bring new treatment options to those who are in need of them. GSK is very proud to be a long-term supporter of SPAEN.

GSK – Focus on the Patient Initiative

  • Post approval Disease Awareness. Once we have a new medicine approved we then work externally to raise the awareness for the disease.
  • Simplification of the information consent form and process (for clinical trials). Really important to try to get some of those documents into a manageable form where patients feel comfortable and the information they are provided with is appropriate not full of legal jargon.
  • Engagement with patients and physicians at different levels. Clinical trial space – to understand the trial and get feedback. How has a trial been for patients? What was your experience?
  • Treatment adherence initiatives. Helping patients to take medications appropriately. Increasing focus on developing oral medications rather than intravenously… however there can be challenges for side effects and also getting patients to remember to take the medication.
  • Working with the patient support groups.

Post approval Disease Awareness
GSK took on an advertisement raising awareness to physicians to what a challenging disease Soft Tissue Sarcoma is – Raising Awareness of a Challenging Disease advertisement.

My first encounter with sarcoma was in 1991…
At the time I was working as ‘study monitor’ based in Brussels doing a lot of work in different oncology drugs.
I was working with a patient and realised that she had the same date of birth as my brother. It still gives him quite a jolt of reality recalling the challenge in these phase 1 trials. These are usually in older patients except often in sarcoma patients who tend to be younger. A very emotional moment to realise my brother was the same age as this patient and this has stayed with me.

In 2006 I joined GSK as indication lead for pazopanib in advanced soft tissue sarcoma (‘STS’)…
Phase 1 trial had good results of the 6 patients entered… 4 had stable disease by 6 months.

Key milestones for pazopanib in a STS
September 1996 – Program Initiation
Nov 2001 – Candidate Selection
December 2002 – First in Human Dose
October 2005 – 1st STS Patient Dosed (Phase 2)
Sept 2007 – Proof of Concept
March 2008 – Phase 3 Commitment
Oct 2008 – 1st STS patient dosed (Phase 3)
June 2011 – Supplemental NDA Submission
April 2012 – US FDA Approval
August 2012 – EMA Approval

Collaboration with EORTC Soft Tissue and Bone Sarcoma Group Phase 2 and 3
He met with Prof Judson at the Royal Marsden to talk specifically about patient cases. That collaboration has driven the project forward and made him appreciate what a unique community the sarcoma community really is.

Without Sarcoma UK and SPAEN – patients in the UK would not have been able to participate in the PALETTE Phase 3 study. Double blind, placebo controlled trial.

Randomising into placebo without opportunity to cross over within the study.
Without intervention from patient groups it may well have not been able to take place.


Markus Wartenberg – SPAEN, Germany
Status report from the SPAEN task force ‘Therapy and side effect management’. Problems and experiences from other advocates – examples of adherence tools?

Oral targeted therapies:

Often patients attitudes/statements:

  •  I am doing fine, I don’t want to spoil my day with feeling unwell.
  • Nausea /diarrhoea reminds me of having this disease – every day.
  • Drug holidays: to have a great weekend or vacations.
  • Doing well after time: Let’s return to normal life
  • Adjuvant settings: No ‘detectable’ tumour. It’s only for prophylaxis…
  • Poor packaging – complicated dosing schedule – forgetfulness.

Often in reality…
Drs attempts at therapy and side effect management. Attitudes/statements:

  • 5 minutes of information: basis for the therapy offered. Is this enough?
  • Let’s see each other in 3 months….
  • If you have any side effects, contact your GP. Would GPs know?
  • Reduction from 12 to 4 different drugs. This many drugs to manage side effects? Needs to be assessed.
  • Others don’t have this problem. This must be psychologically.Underrated: Dermatology, dental treatment, high blood pressure.

There are also side effects that are not visible to the Dr or the patients.. ie high blood pressure (unless it’s measured before and after). Nausea can’t be measured… and lots more.


  • Modern systemic oral treatments will not work in a patient who does not take his medication.
  • Adherence is an issue but it not only a patient issue. Could this have an impact from the media? Is it that the drugs are provided without a side effect management explained and information. Seeking information from other patients or a patient group where a GP perhaps doesn’t know… consequence is that they may take drug holidays ie underdosed with this type of treatment.

Issues for Healthcare Professionals:

  • Misled assumption: Cancer! My patient is taking his medicine.
  • Often: Lack of expertise, experience, time, maintenance (especially in rare cancers).
  • No/less time dedicated to adherence and therapy/side effect management
  • Problem: Do healthcare systems incentivise maintenance?

Patients may not always report:

  • Side effects (or the full extent of the side effects on their Quality of Life)
  • Drugs holidays or modifications to dosing schedules
  • All of the medications, supplements, treatments they are taking for side effect management.

Factors for adherence and persistence: Paper written by Rob Horne – UCL School of Pharmacy)

  • Personal factors such as where people don’t trust medicine
  • Interaction with the system.
  • Treatment factors that might play a role for the patient. (Dosing. Some instances flexible dosing may be prescribed and have a better effect.)
  • Treatment duration – is it possible to treat on a long term basis if this is better. What’s the situation of progression? Should the drug be stopped? – Side effect management. What about prophylactic? All these need to be considered for Optimum Efficacy.
  • How good is the patient/Dr communication on these issues? What kind of questions are raised by the patient and indeed the Dr.
  • We always talk about ‘the patient’… are we always using the same process for every ‘patient’ or perhaps they should be treated individually.

Maximising patient outcomes with targeted agents:
Target agents: a new era in systematic treatment.
-> Patient needs to benefit from a therapy for as long as possible
-> Three key factors for optimum efficacy
-> Adherence
-> Getting the most out of your therapy.

Joining forces for access, quality, innovations and changes.
Communication and cooperation between all groups including healthcare pharma and patients is needed.

When a survey was conducted they discovered that – Less than 50% knew about the standard supportive actions.

Informing and educating the medical people who, in turn educate the patients.
More awareness and support. Brochures at patient and physicians meetings.

Relaunching new website working on a specific tool as an online database of side effect management. What are the real issues? What are the interaction that patients can do? Hopeful that medics will use it also.

  • Working on a patient guide on therapy and side effect management.
  • Also developed another patient guide, about complementary therapy and nutrition. What can I eat if one of my side effect affects my mouth, ulcers or the like? Patients also have to be aware of the side effects that may be because of the complementary therapy or nutrition etc as it could adversely react to the conventional medicine.
  • Engage with industry to be involved with patient materials. They have to be valuable to patients, patient friendly and written in a way that can be understood by the patients.
  • Physician training for teams on dermatology with oncologists/nurses/Drs. They can then best understand these side effects. Need a combination to work on these issues and involve dermatologists in the process.

Potential activities:
Patient advocacy groups

  • Information education of patients and carers
  • Information about influencing factors – complementary medicine and nutrition
  • Hotlines, info-materials, treatment diaries
  • Together with experts: practical recommendation.
  • Own studies/surveys (Evidence) with support of industry and experts
  • Online – Database “Side Effect Management”
    • Mailing list and/or forum exchange
    • Listings: Centres – best therapy care.

Healthcare industry:

  • Are packaging and dose schedules – is it patient friendly?
  • Studies: side effects not only ‘grading’ but also ‘duration’…
  • Real life studies – reg. dosing and outcome…
  • Congresses, conferences, symposia; Awareness!!
  • Information and education (doctors, nurses, pharmacists, etc)
  • Support/train local networks: Oncologists and nurses and dermatologists
  • Health policy: Compensation for time, communication, support
  • Develop material for patients – BUT together with Patient Awareness Groups
  • If care packages not via Medicals via Patient Awareness Groups (make it measurable on the holistic assessment?)
  • Materials and tools to support adherence
  • Not “company specific” Engagements with other pharmaceutical companies.

Oral target therapies in Sarcomas/GIST:
Professional therapy – and side effect management as the main factor for adherence. Our patients must get the BEST benefit for as long as possible from these kind of treatment with an acceptable quality of life.



Dr Francesco Pignatti, Scientific and Regulatory Management Department, European Medicines Agency, UK (“EMA”)
The process of authorisation for new generic drugs

What are generics?
Defined as essentially similar products. Must contain the same qualitative or quantitative composition in active substances as the originator. Same pharmaceutical form and studies to prove it’s working in an appropriate way.

Approx a 40% saving at the time a generic medicine is introduced.

What data are required?
Chemical, and pharmaceutical aspects (Quality)

  • Same requirements as for the product.

Non-clinical studies (in vitro studies, animal studies)

  • Often no studies required for generic product

Clinical studies

  • Only bioequivalence required for generic product (no need for traditional phase 1-3 development)

In essence: “if the fraction of the dose absorbed is the same, the human body should always do the same with the absorbed compound”

It may be that the ‘packaging’ ie the tablet form or coating makes it’s absorption being different.

Issues related to bioequivalence

  • Biowavier
    • For different strengths
    • Using the BCS classification approach
  • Parent compound versus active metabolite
  • Investigation in fed versus fasting state
  • Handling of outliers

EMA experience

  • Initial evaluation application by type
  • Average active time taken for evaluation is 171 days… no different from other drugs.
  • Rejection rate is about 10%
  • Whenever generic or another drug is approved, it goes on their website and more general information about what generic drugs are

Q. What about the psychological impact of changing from a drug that works to a ‘generic’ drug that they don’t know works?
A. Would have to understand there is a risk and if so what is it.
Q. Why is the patient responding to that drug? Will it be the same with the generic.
A – The active ingredient is the same and it is hitting the target. Objectively it should have no different response. Psychologically it would have to be considered on an individual basis and perhaps needs to be managed accordingly. Patient preference issue should be reviewed by the Dr.


Dr Karen Facey, Honorary Senior Research FelLow, University of Glasgow, Introduction to Health Technology Assessment (“HTA”)
How can patient groups be involved in the process of access to new therapies? – Health Policy Consultant, UK

Health Technology Assessment

  • What do patients have to contribute?
  • Providing evidence to HTA
  • Participating effectively in the HTA process
  • Principles of patient involvement in HTA

Difficult decisions

  • Do new treatments and procedures add value compared to current treatment?
  • Should the health system invest in them?
  • If we invest in a new treatment there is an ‘opportunity cost’ – we must take investment away from somewhere else in the system.
  • How do we decide what the priorities are?

In times of austerity we need to look at how to make a fair decision about how to spend the budget fair for the population we are serving.

How do we decide what to stop investing in?

Since NICE involvement there has been an increasing use of this process called Health Technology Assessment. It’s meant to be a fair systematic evaluation of the clinical effectiveness and/or cost effectiveness and/or social and ethical impact of a health technology on the lives of patients and the health care system. Should we invest to get that amount of benefit?

Health Technology Assessment International

Ethical issues come in strongly for trials.

HTA is being used more and more for where to spend money.

Health Technologies.
A ‘health technology’ is any intervention that may be used to promote health, to prevent diagnose or treat disease or for rehabilitation or long term care.

This includes education, vaccines and much more.

Another part of assessment should be to determine ‘which patients will benefit most from it?’ How long they should get it and if they don’t respond what next?

HTA and decision making is often considered a bridge between scientific evidence and decision-making.

Patients have unique knowledge and perspectives that can inform.

Patients and carers experiences:
Living with an illness

  • No one knows better what it is like to live with an illness day in day out, than those who are doing this – the patients and their family and friends who care for the.

How can patients perspectives in HTA be a route to robust evidence and fair deliberation?

Patients and carers can contribute to HTA

  • By providing EVIDENCE about their experiences and preferences
  • Through PARTICIPATION in the HTA process.

Within the Scottish Medical Commission’s Submission of Evidence Template – SMC 04/12.

Section 3 – views of patients. Carers and families.

Patient evidence:

  • Ideally, concise and balanced overview that reflects the range of patients perspectives
  • Variations in clinical practice
  • Personal perspectives about benefits and difficulties with the technology.
  • Views on rules for starting and stopping treatment
  • Evidence and facts NOT emotions.

Watch the lay people present their case to the Scottish Parliament – Health and Sport Committee Scottish Parliament on 29th January 2013. This demonstrates the importance of presenting evidence, facts and presenting as if a well run business.

Clinical trials vs patient benefit

Some tips about how to gather patient experiences and preparing the evidence:

  • Review helpline questions
  • Survey/questionnaires
  • Social networking
  • Patient stories (videos)
  • Qualitative research (interview, focus groups…)

Build an evidence base!

There is a research project of the HTA – Patient/Citizen Involvement Sub-Group (PCISG)

Through this sub-group there is participation in the HTA process at every stage:

  • Study design to produce evidence
  • HTA topic selection
  • Scoping
  • Submission of evidence
  • Presentation of patient experience to expert committee
  • Sitting on an HTA decision making committee
  • Commenting on recommendations
  • Patient friendly summaries
  • Dissemination/communications
  • Designing and reviewing patient engagement processes
  • Use HTA to inform charity investments
  • Contribution to governmental review of HTA.

Increasing there are opportunities for patients to get involved in designing the patient engagement policies.


  • Population
    • What patients?
    • When an agency starts a process it looks to what patients should be considered.
  • Intervention
  • What medicine, what dose, duration, how’s it administered etc.
  • Comparators
  • Outcome – what matters to patients?

Communicating to a committee

  • Understand your audience and how you are allowed to participate
  • Identify what other experts will be involved and seek to provide unique knowledge.
  • Be prepared with written evidence to refer to
  • Know how long you have to speak – top 3 messages
  • Facts, not emotions.

Tips for writing your consultation response (NICE-IPP)

  • Evaluation of efficacy and safety – not costs.
  • Doesn’t need to be lengthy or comment on everything
  • Short, focussed response
  • Be specific about the procedure
  • Balanced positive and negative
  • No local issues.


Eric Lowe, Chief Executive Myeloma UK
Build your own evidence base – experiences with NICE in the UK

Eric speaks about his involvement on behalf of Myeloma UK with HTA. Taken part successfully in 20 HTA appraisals. Very steep learning curve and some of the toughest experience.

Reimbursement increasingly a huge issue – tremendous problem across the world even in wealthy countries.

HTA is expanding and evolving.

My view is that in 5 years time the UK is going to be a great place as HTA continues to evolve.

Dumbing down health economics, public health doctors and government – empowering HTA.

Biggest mistake of working with patient groups is that they don’t appreciate the wider agenda ie not just about patients.

Work with NICE and help them do their job properly. Help them to create a new environment rather than butt against them.

Being involved…

  • Patient groups need to be crystal clear on what their ole is and isn’t. It’s a huge commitment to be involved in an appraisal so it is important that patients and patient groups are as effective as they can be.
  • They must also try to represent at the patient view beyond the emotional and angry. Got to behave a s a professional cognisant of all the aspects and being able to represent the case.

Being listened to…

  • Need to be seen as both a patient and a key opinion leader
  • They must understand the broad and competing issues around the table and then take up a compelling but balanced and credible position.
  • Early engagement is critical. It is important to go into an appraisal with a detailed understanding of the technology and the opinions and positions of other stakeholders.
  • Patient groups should engage with the company and clinicians to find out their views and opinions.

How early should you get involved?… Phase II study group or before!!

Must ensure that all representatives engage prior to that point to get consensus and agree on the issues and objections. They should work collectively to submit a cohesive and consistent submission rather than sending in several potentially very different submissions which may show that there is a poor understanding and little agreement about the benefits and/or impact of a new technology.

Building evidence…

  • Patent groups need to think about conducting research to provide evidence for their arguments.
  • Anecdotal is not good enough so by conducting studies of patient preference, values and impact etc. puts you in a much stronger position and gives the approval committee more certainty around what impact a new technology may or may not have.
  • Be careful about quality of life.
  • Need to think way I advance of the beginning of the appraisal to give time to conduct, analyse and write up their evidence/data
  • Patients also have a role in ensuring the clinical evidence is fit for purpose.
  • Patient groups also need to encourage industry to work with NICE Scientific Advice and also attempt to influence the clinical trial designs of registration studies or to encourage additional evidence development to address the evidence gaps. The service is available to help… only 10% of companies use the service!!!

Myeloma UK have established a national clinical trials network. We worked with pharma ahead of time, looked at the trial compared to NICE and then designed a phase 2 academic study to deal with.


  • Reimbursement issues are increasingly challenging
  • HTA is here to stay, is evolving and expanding.
  • Specialist expertise is needed
  • Inputs needs to be better, crap in crap out. Much more important than that… it’s based on a Phase 3 Global Licensing study… quite often that is very different to clinical practice… even if NICE say yes to the drug we need to make sure we can translate it into effective clinical practice.
  • Done properly it’s a good thing and patient groups should embrace it and make it work for them.
  • HTA is only part of the process and not the end.

Be partners and help involve HTA from the inside.

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