Access to treatment, care and clinical trials by patients with rare cancers

Jan Geissler

Co-founder, CML Advocates Network

Vice President, Leukemia Patient Advocates Foundation Secretary, European Forum For Good Clinical Practice

Member, EU Committee of Experts for Rare Diseases (EUCERD) Director, European Patients’ Academy on Therapeutic Innovation (EUPATI)

Rare but not alone? The journey of (rare) cancer patients

1 in 3 Europeans will get cancer in their lifetime

1/3 of them develop a rare cancer

~261 cancers are rare - and very different

Cancer, rare cancers, rare diseases – is there a difference?

Rare Cancers: often lost between 17 common cancers and 1000s of rare diseases

Rare Cancers share many challenges with rare diseases

~6000

Rare

Diseases

203

Cancers

~1

86

RA

RE

CA

NC

ER

S

Patients with rare cancers face specific challenges

1. Prevention and screening mostly irrelevant

2. Late or incorrect diagnosis very common

3. Experienced doctor not available locally, lack of access to clinical expertise

4. Lack of appropriate therapies

5. Slowness of research (lack of trials & commercial interest),

6. Facing stigma and inequity

7. Lack of information & local patient groups

Patients with rare cancers face specific challenges

1. Prevention and screening mostly irrelevant

2. Late or incorrect diagnosis very common

3. Experienced doctor not available locally, lack of access to clinical expertise

4. Lack of appropriate therapies

5. Slowness of research (lack of trials & commercial interest),

6. Facing stigma and inequity

7. Lack of information & local patient groups

Rare Cancers: best to be treated in an experienced center

Misdiagnoses common before diagnosed appropriately

Number of „cases“ in a center, conduct of studies: Indicator for up-to-date expertise and treatment outcome

Study clinic (n= 56 pts)

Standard Inferior therapy

52%

48%

75%

25%

Non-study clinic (n= 68)

3 times higher

chance to

receive a better

therapy!

Source: Standard therapy in ,FIGO I-IIA: Study- vs Non-Study clinic, German Quality program QIII 2001 AGO OVAR

Special challenges crossing country borders

Currently, crossing country borders for health services is challenging (E112 form, prior authorization, unclear rules)

„Patients‘ Rights in Cross-Border Healthcare“ Directive 2011/24/EU, in national law by Oct 2013

• To provide clarity and legal certainty on access to diagnosis & healthcare in other EU Member States, especially when condition requires particular expertise or resources (e.g. rare diseases)

• National contact points

• Rules for reimbursement

• Procedural guarantees

• Prior authorisation system & reasons to refuse & time limits

• Cooperation between health systems

Patients with rare cancers face specific challenges

1. Prevention and screening mostly irrelevant

2. Late or incorrect diagnosis very common

3. Experienced doctor not available locally, lack of access to clinical expertise

4. Lack of appropriate therapies

5. Slowness of research (lack of trials & commercial interest),

6. Facing stigma and inequity

7. Lack of information & local patient groups

Unmet medical needs for most patients with rare cancers

“Success stories” available only to small numbers of rare cancers

Patients in urgent need of therapeutic innovation

Source: RareCare (2012)

Barrier: Public image of clinical trials

Lack of public confidence in research

leads to

delayed / lack of trial recruitment

delayed generation of meaningful clinical data

slow progress or lack of research in Europe

Good information and support of patient groups can help to understand

?

Potential benefits of a clinical trial

Personal benefit:

• Some new therapies only available in trials

• Closer monitoring

• Therapy optimization trials with more effective or more tolerable regimens

Altruistic reasons:

• Achieving progress – beyond our personal case

Understanding risks of clinical trials

Uncertainty always brings risks, but trials are strongly regulated & assessed

Risks of participation in trials • Risks of the unknown: will it work? Will it do harm?

• Impact on quality of life, unpleasant/more diagnostics

• Protection from unauthorized use of data and tissue

Good information is essential for informed consent (of the whole family)

Where to find trials?

Research networks websites

EU Clinical Trials Register https://www.clinicaltrialsregister.eu

Doctors & Patient organisations

Patient organisations are ‚navigators‘ for patients

Most „official“ information portals fail on rare cancers: Budget for the "Top 17".

Patient organisations inform, help, support

• Explanation of disease, therapy, side effects

• Managing anxiety about disease and doctor

• Information on clinical trials (centers, doctors, results)

• Working in partnership with researchers

• Trustable web resources

International Rare Cancer Advocacy Organisations (just to name some)

International Brain Tumour Alliance (IBTA) - http://www.theibta.org

Sarcoma Patients Euronet (SPAEN) - http://www.sarcoma-patients.eu

CML Advocates Network - http://www.cmladvocates.net

International Kidney Cancer Coalition (IKCC) - http://www.ikcc.org

European Cancer Patient Coalition (ECPC) – http://www.ecpc-online.org

European Rare Disease Organisation (EURORDIS) - http://www.eurordis.org

European Waldenström Network (EWMNetwork) - http://www.ewmnetwork.eu/

Myeloma Patients Europe (EMP, http://www.emp-myeloma.eu and ME, http://www.myeloma-euronet.org)

Lymphoma Coalition - http://www.lymphomacoalition.org

Myelodysplastic Syndromes Foundation - www.mds-foundation.org

Carcinoid & Neuroendocrine Tumor Society - http://www.cnets.org Carcinoid Cancer Foundation - http://www.carcinoid.org/

International Confederation Of Childhood Cancer Parent Organizations (ICCCPO) - http://icccpo.org/

…and many more…

Patients – as co-researchers?

"More needs to be done: rare cancers will never be a

priority unless the patients make it one. Patients

themselves must therefore play a larger role in

driving forward the search for therapies. They are

able to see connections that have eluded scientists."

Patients have a complementary expertise that is invaluable to research

Patients experience is complementary, not substitutive

Examples

• What it means to live with cancer

• Explain the value and barriers of participation in cancer trials

• Improve informed consent

• Make trial results widely known

• Provide insight on adherence, CAM use, QoL

• Fundraise to make research happen

Research subject

Information provider

Advisor

Reviewer

Co-researcher

Driving force

PatientPartner

FP7 Project (2010)

Launched Feb 2012, runs for 5 years, 29 consortium members, PPP of EU Commission and EFPIA

will develop and provide objective, credible, correct, up-to-date knowledge about medicines R&D

will build competencies & expert capacity among patients & public

will facilitate patient involvement in R&D to support industry, academia, authorities and ethics committees

The Patients’ Academy: empowering patients on medical R&D

Rare Cancers: Rare, but not alone.

Jan Geissler

jan@patientsacademy.eu

Twitter @jangeissler

http://www.patientsacademy.eu

http://www.cmladvocates.net