Countries: ▼

Capabilities

Tailored, patient-centric development and clinical trial methodology

Rare diseases and orphan drugs undergo the same review processes as other pharmaceuticals and must meet the same high standards of quality, safety and efficacy. The small numbers of patients affected by a rare disease and the variability of any inherited trait (the so-called high phenotypic heterogeneity) means that randomised clinical trials are not always feasible for a rare diseases or orphan drug. Also, the use of placebo-controlled trials may be unethical and for comparisons standardised treatments are often not available.

Recordati Rare Diseases works closely with physicians, centres of expertise and patient organisations to design tailor-made clinical programmes that define the key evaluation criteria for each drug. To this end, we have developed a unique external network of experts who support and cooperate closely with us from a very early stage of development through to market authorisation and post-marketing registries.

Specialist regulatory affairs expertise

Building a complete marketing authorisation dossier based on data from a small number of patients requires specialist know how, experience and a good working relationship with the regulatory authorities. Recordati Rare Diseases has nearly 30 years experience in:

  • Orphan drug designations
  • Marketing authorisation applications
  • Post-approval safety surveillance
  • Named patient use

Highly qualified, highly focused marketing and sales teams

The people in our marketing and sales teams have a personal commitment to improving the lives of people who have a rare disease. We recognise that only through working collaboratively with all stakeholders are we able to tackle the challenges in the orphan drug and rare disease field. These challenges include disease awareness, education and training, and the development of consensus around diagnostic and clinical care guidelines.
Our Rare Diseases Orphan Drug Specialists are highly trained experts working closely with all stakeholders to understand their needs and develop solutions for improving patient management from diagnosis to treatment and to help ensure patient access to our products. 

Market access and focused patient access to treatement

Pathways to enable reimbursement and access for patients to our rare disease treatments are extremely heterogeneous across countries and require longstanding experience

At Recordati Rare Diseases we: 

  • Succeed in providing early patient access to our treatments through local regulations
  • Know how to navigate the various requirements of Health Technology Appraisal (HTA) bodies across countries in the orphan drug space
  • Know how to generate convincing evidence for HTA bodies and payers in insufficiently researched disease areas
  • Are extremely close to the rare disease community to help generate access to our treatments worldwide