The Alcimed Healthcare team has been exploring the rare disease market, often called orphan diseases, for more than 25 years, and supports its clients in their innovation projects, in understanding the orphan drug market and in their market access approaches.
THE CHALLENGES RELATED TO RARE DISEASES OR ORPHAN DISEASES
What is a rare disease and what is an orphan drug?
A “rare disease” is when a disease affects a very small proportion of the population, generally less than 8 people in 10,000.
An “orphan drug” is a drug that obtains privileged regulatory status from the authorities (the “orphan designation”) intended to encourage the development of therapies for these orphan diseases.
What are the challenges related to rare diseases?
The rare disease market is experiencing strong growth and transformation across the world. Over 230 orphan drugs have been approved on the European market since early 2000.
As a result, the typology of rare diseases, their challenges and their market situations are evolving significantly. This term now includes:
- ultra-rare pathologies without any suitable medical solution,
- relatively unknown rare diseases still awaiting solutions,
- rare but well-known pathologies, with several treatment options, representing considerable progress for patients in established markets that are more similar to so-called “specialty” markets.
Even though this market is developing, manufacturers, biotechs and research centers are facing many challenges, including:
What are the most relevant new technologies to continue transforming the field of rare diseases? Which technological approach should target which disease and how can the current limits to address all sub-populations be overcome?
How many patients are affected and how can patients be found for a clinical trial?
How can healthcare professionals and authorities find out about this rare disease and the existence of a treatment?
What is the regulatory framework and what path should be taken to obtain and accelerate the marketing of an orphan drug?
How will a drug be assessed and possibly reimbursed in a given country? At what price should the treatment be positioned? How can its value be shown?
How can care pathways be organized and optimized to improve the level of diagnosis and accessibility to treatment?
HOW DO WE SUPPORT YOU IN YOUR RARE DISEASES OR ORPHAN DISEASES PROJECTS?
For more than 25 years, Alcimed has been supporting its clients on many issues related to orphan and ultra-orphan diseases, in Oncology and outside Oncology. Indeed, we have carried out more than 150 projects in this field on more than 100 rare diseases for different actors such as:
- National and European institutions and research centers (for example, the European Commission, INCa, AFM / Genethon)
- European and North American biopharmaceutical and pharmaceutical companies, biotechnology companies and professional associations (e.g. Genzyme (Sanofi), Shire (Takeda), Endo, Amgen, Chiron, Actelion (J&J), Alexion (Astrazeneca), PTC Therapeutics, Vertex, Sanofi, J&J, Roche, Merck-Serono, Novartis, Novagali (Santen), Spark Therapeutics (Roche), Insmed, Bone Therapeutics, Cellectis, NPS Pharma (Takeda), Intermune (Roche), EFPIA, EBE, EuropaBio etc.)
- Patient associations (e.g. SFPA).
The diversity of our clients (industrial companies, biotechs, research institutes, professional associations, national or European institutes, etc.), the geographic areas we explore, and the types of projects we develop, give us a global and in-depth understanding of the issues related to rare diseases.
Our projects cover areas as diverse as new technologies and therapeutic approaches for orphan diseases, search for funding opportunities, preparation for the launch of new orphan drugs, understanding of disease management and patient care pathways, assessment of market and licensing opportunities, market access, pricing and reimbursement models, regulatory developments and operational support (patient identification, care pathways optimization and many more).
The types of projects we carry out for our clients in this field are:
EXAMPLES OF RECENT PROJECTS CARRIED OUT FOR OUR CLIENTS IN RARE DISEASES
To do this, our team interviewed patients, caregivers and associations in each country concerned in order to identify the levers for improving their well-being and their quality of life that could be addressed by the development of services. Following this, our team pre-tested these service ideas with healthcare professionals, before organizing a workshop to co-construct a service development roadmap with the medical and marketing teams of our client. Ultimately, several services were deployed in pilot countries and then extended to other countries in the area, improving the patient journey and positioning our client as a major player in public health.
1. Diagnose the current coordination of care between the different health actors registered on the patient journey
2. Co-define with this multidisciplinary team an ideal patient care model and an associated action plan
3. Implement actions defined at local and national level.
The result for our client? A reduction in the time taken to diagnose patients and a general improvement in physician coordination, an increase in therapeutic support and a change of perspective for the sales teams.
Our purpose? Helping both private and public decision-makers explore and develop their uncharted territories: new technologies, new offers, new geographies, possible futures, and new ways to innovate.
Located across eight offices around the world (France, Europe, Singapore and the United States), our team is made up of 220 highly-qualified, multicultural and passionate explorers, with a blended science/technology and business culture.
Our dream? To build a team of 1,000 explorers, to design tomorrow's world hand in hand with our clients.
- Proof of concept
- Voice of Customer (VOC)
- Business case
- Business development
- Business models
- Business plan
- Cluster study
- Collaborative projects
- Commercial strategy
- Competitive analysis
- Customer experience
- Dossier creation
- Due diligence
- Go to market
- Innovation consulting
- Innovation process
- Innovation strategy
- Learning expedition
- Market access
- Market study
- New offers
- New services
- Open innovation
- Opportunity evaluation
- Patient pathway
- Product innovation
- Product launch
- Regulatory framework analysis
- Search for funding opportunities
- Search for partners
- State of the art
- Strategic audit
- Strategic foresight
- Strategic positioning
- Test and Learn
- Value proposition