In narrative for first bullet point mention that trainees will be doing a similar exercise on Friday afternoon.
Patient interactions between Industry, HTAs, Academia & Regulators
“Patient interactions between Industry, HTAs,
Academia & Regulators”
The United Parent Projects Duchenne Muscular
Dimitrios Athanasiou, UPPMD Board member & EMA expert patient
POs Placement in the Medical Development equation
Grey areas of POs relations with the industry in the past
• Lack of trust from all sides increases the complexity of the environment and delays drug
Almost all other stakeholders have a clear code of conduct and compliance regulations *
• POs need a professional approach and a specific frame of conduct with the other stakeholders.
• Legal & compliance advice is most needed in POs.
There is great need for more directive towards patient involvement
• This can only be achieved in an organized and transparent environment
POs need to have a clear position in the medical development equation
• We need to be clear about our interests , our positioning and find effective ways to tackle
possible challenges in the equation with the other stakeholders
* EMAs code of conduct, EFPIA code, EURORDIS code of practise ,HTAi code of conduct etc
Lack of knowledge and skilled expert patients
Lack of transparency and a common code of conduct
Limitation of current models for rare and ultra rare diseases
Limitation in understanding the “bigger” picture and long-term stategy
Limitations and obstacles at the national and international level
Duchenne Muscular Dystrophy (DMD) is one of the most common, devastating and
lethal genetic disease of childhood. DMD occurs in ~1:3500 male births world-wide
with no treatment or cure and life expectancy between late 20s and early 30s.
What is Duchenne Muscular Dystrophy (DMD)
Strategy to tackle the obstacles of the DMD model
Map the environment and identify specific areas of POs strategic interest (Academic
research, industry, regulatory work, training, venture capital & funding etc.)
Identify key stakeholders per area and place specific goals
Define which POs of the community are more effective per area
Help develop new organizations internationally and integrate them, they will be a constant
stream of highly skilled patient advocates
Develop working groups and a group leader with various umbrella organization PO reps
Identify highly skilled patient advocates specialized in different areas of interest within the
umbrella organization and train new specialized patient advocates per area
Manage possible conflicts by developing alternative skilled patient advocates per area
Distribute knowledge and best practises under the umbrella organization
POs and Industry
Benefit/Risk Study in Duchenne Muscular Dystrophy (DMD) regarding pulmonary
therapies in the disease with Santhera and Parent Project.
Decode Duchenne PPMD Launches Next Phase of Genetic Testing Program,
Decode Duchenne in collaboration with BioMarin Pharmaceutical, PTC Therapeutics, &
Sarepta Therapeutics is a nationwide program to assist individuals with Duchenne
muscular dystrophy in accessing genetic testing, interpretation, and counseling.
DMD Patient Advocacy Group (PAG) advisory board with PTC looking at challenges
and solutions in DMD, best practices and knowledge transfer.
NICE accreditation on Duchenne guidelines as produced by the highest standards
and we are in constant contact offering real life expertise for Qualis and receiving
pharma economics advice for our projects
POs and HTAs
POs and Academia
TREAT-NMD It started some years ago as a network of registries and now is involved in
the biggest project leader in Neuromuscular diseases with offshore activities. Patient
Reps are always in the Board of Directors and the Steering Committee
• Since 2007 the network's focus has been on the development of tools that industry, clinicians
and scientists need to bring novel therapeutic approaches and on establishing best-practice
care for neuromuscular patients worldwide.“
RD CONNECT connects, databases, biobanks and ‘clinical bio-informatics’ for rare
DMD IMAGING works on development of new Magnetic Resonance Imaging (MRI) and
Magnetic Resonance Spectroscopy (MRS) technologies for Skeletal Muscle and as a
biomarker for DMD therapeutic development
PPMD and LEIDEN partnership created a breakthrough Exon skipping technology for
DMD Exo-Skeleton International Project works on how technology can assist DMD
POs and Regulators
European Medicine Agency (EMA) As Draft Guideline on the clinical investigation of
medicinal products for the treatment of Duchenne and Becker muscular dystrophy
US Food & Drug Administration (FDA) Draft Guidance on Duchenne PPMD and a
broad coalition of stakeholders has submitted the first-ever patient advocacy-initiated
draft guidance for a rare disease to the FDA to help accelerate development and review
of potential therapies for Duchenne muscular dystrophy.
• More than 80 representatives of the Duchenne participated in seven working groups
that met over the past six months to draft the guidance.
Common points of " Interest " by the regulatory authorities for POs
• Consulting, Strategic Advisory Work for example participation in advisory boards,
steering committees etc with or without remuneration and
• POs funding should be declared!
New models of patient involvement
New models of drug development
• Patient driven research consortiums, Cross disease consortiums, Patient owned registries, Post
Marketing Registries (PMRs) , Patient Reported Outcomes (PROs) , Patient Reported Outcome
Measures (PROMs) etc.
New models of drug development financing
• Philanthropic venture capital, hedge funds, crowdfunding, etc.
Non profit Pharmas and Non Profit Contract Research Organizations (CROs)
• Drug repositioning and DMD Mapping project with a non profit Pharma (Data is owned from
Hybrid models of Intellectual property (IP) ownership and/or animal model ownership
• Leiden Exon skipping 51 and DMD mouse