Rare diseases

One of our clients, a leading pharmaceutical player, wanted to improve the care of patients with a rare disease by developing a high value-added service offering that improves their well-being and quality of life throughout their care pathways (from diagnosis to palliative care).

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.

Alcimed supported a US biopharmaceutical client to define the launch strategy for its orphan drug in Europe. It then helped its client to structure a concrete and operational plan, both at a European level as well for each target country.

As a real roadmap for each of the key functions of the company (medical, marketing, production, regulatory, sales), the plan extends from pre-launch (before obtaining the MA, the Marketing Authorization) until the first sales.

Our team is also involved in regularly updating this plan, depending on internal or external developments around this launch.

We have supported the European Medical Department of a pharmaceutical manufacturer in optimizing the care pathway for patients suffering from a rare respiratory disease in several European countries. As this rare disease is difficult to diagnose and often confused with more common respiratory diseases, the patient diagnosis time was too long (2 to 3 years). Our client wanted to position itself as a public health player by providing reference hospitals with Alcimed’s service to:

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.

Support for a player in the pharmaceutical industry in understanding the fundamental factors of market access for orphan drugs in 9 emerging countries.

By taking stock of internal knowledge and collecting missing information in terms of the market and regulatory analysis in particular, Alcimed has enabled its client to obtain an overview of the regulations on orphan drugs in emerging countries and to classify these countries according to their level of accessibility.

Alcimed supported a major pharmaceutical player in developing its rare disease portfolio by identifying the best hospital centers to target for its clinical trials in two rare hematological disorders.

First, we deciphered the care ecosystem in these two rare diseases (epidemiology, rare disease care network, care pathway, competitive clinical trials, KOLs) to identify potential investigational centers. Then we collaborated with the client to create a set of criteria for the initial selection of qualified centers for their clinical trials. Finally, we refined the most relevant centers by interviewing them and prioritizing the ones to target first.

On this basis, 5 easily actionable centers were identified as 1st priority centers and have been approached by the client to undertake their clinical trials thanks to the connections we had made with the centers during the project.

Alcimed worked with a top pharmaceutical client to prepare and define their 2030 R&D strategy by understanding the main trends in rare genetic diseases in neurology and pediatric oncology.

To do so, we identified the 4 key steps that lead to a successful launch of new products for rare diseases : Clinical development, Manufacturing, Regulatory and Pricing, Go-to-market models.

Then, thanks to the interviews undertaken by the team with other laboratories working on rare diseases, we gathered all the best practices and successful examples for the launch of new therapies in this area. At the end, our team built an analysis framework summarizing the key insights which led to recommendations about the client’s R&D strategy on rare diseases.

This investigation enabled the client to define the market opportunities for rare genetic diseases and viable business models in adequation to their strategy.