Rare Disease & Orphan Drug Development: Cost-Efficient Trial Design to Minimize Cash Burn

Life Sciences, Clinical Trials, Pharmaceutical, Drug Discovery & Development,
  • Thursday, June 01, 2017

Discussion topics include:

  • Key challenges to improve cost-efficiency in clinical development of orphan drugs
  • Regulatory evolution in the review process of Orphan Drugs
  • After examples like Sarepta’s Exondys51 approval, how will the evaluation of clinical benefits change based on endpoints?
  • What aspects of commercially planned clinical trials can contribute to cash-burn during orphan drug development? How do you plan for scale up while improving cost-efficiency?
  • What role does multi-stakeholder collaboration in rare disease research play in improving cost efficiency of clinical trials?

Despite the benefits of orphan drug development, cash burn minimization is one of the major challenges faced by developers. While guidelines exist for efficient orphan drug development, they should be adapted to challenges presented by each of the more than 7,000 recognized rare diseases.

The clinical development of orphan drugs represents a very interesting challenge that demands thoughtful and innovative designs. Orphan Drug clinical developers must become serious reliable players in the orphan space to attract investors and motivate all the stakeholders. The regulatory process of orphan drugs is becoming more transparent and faster in the review step.

Commercial success is not a guarantee and the regulatory and access demands are rising. As a consequence, orphan drug developers are focusing their interest in the “ultra-rare” diseases field which is characterized by conditions that are chronic or degenerative with  poor quality of life and/or shortened lifespans.

Given the increasing scrutiny of market access for orphan drugs, high quality clinical data, clinically meaningful endpoints rather than surrogate endpoints, life expectancy/survival, health economics evaluations and properly labelled indication must be part of the clinical trials for orphan drug development. While historically orphan drugs have been rewarded with very high prices, the current annual price per patient faces tougher demands. However, the median current cost per patient is still 5.5 times higher for orphan drugs compared to non-orphan Drugs.

The main objective of research and development of orphan drugs is to bring a safe and effective treatments to patients in the shortest period of time while protecting the investment by increasing certainty and prioritizing the quality of data through innovative procedures. There are many regulatory benefits related to the orphan drug designation designed to bring a safe and effective therapeutic option to patients who are often children suffering from devastating rare diseases.

Three main factors will affect the final cost of the entire proposed clinical development programme: Time, quality and activity. The purpose of clinical development is to bring into the market innovative treatments to overcome the unmet medical need. Therefore, the clinical trial design should enable generation of the highest quality data that would facilitate the regulatory review and approval, as well as answer the market access requirements. The efforts allocated to cope with the development scope must be maximized considering that the efforts per “rare disease” patient may be much higher than with more conventional conditions. The completeness of the non-clinical experience and the adequate product development/production campaign are relevant factors at the time of launching the clinical development program.


Carlos R. Camozzi, MD, Chief Medical Officer, Simbec-Orion Group

Group Chief Medical Officer world-wide responsible for the Clinical Research, Development and Pharmacovigilance activities of Simbec-Orion Group (London/Slough, United Kingdom); a clinical research organisation bringing outstanding solutions to the most challenging clinical research and development projects of the biotechnology and pharmaceutical industry.

A physician by training, with specialisations in Paediatrics, Clinical Pharmacology and Neuropsychiatry, Carlos has extensive experience in clinical research and development for paediatric and rare diseases therapeutic options, He also brings experience from successful regulatory interactions and outstanding products approvals achievements, both by the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA), including the first ever approval of a Gene Therapy (Glybera) in the western world. He also actively participated in financial activities, due diligences, fundraising rounds, and collaboration with several worldwide VCs in products review and analysis of innovative “orphan drugs” development strategies.

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Fabrice Chartier, PhD, Chief Operating Officer, Simbec-Orion Group

Fabrice was appointed COO of Simbec-Orion upon completion of the merger of Simbec Research and Orion Clinical Services in June 2014.

In 1998, Fabrice and Dr Alan Irvine, founded Orion Clinical Services Ltd, an international CRO operating internationally and specialised in Rare and Orphan diseases, oncology and other indications with high medical need. In 1994, Fabrice founded for Fournier Laboratories, a medium-size French pharmaceutical company, their UK International Clinical Development Unit, specialised in Gene Therapy and Immunotherapy. Previously, he was one of the directors of an international CRO based in Paris.

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Who Should Attend?

C-level and senior professionals from Biotechnology companies involved in the research and development of Orphan Drugs

Procurement and outsourcing professionals with a focus on Rare and Orphan Disease, and Oncology

Xtalks Partner


Orion Clinical is the full-service clinical development division of Simbec-Orion Group.

Founded 20 years ago as a European CRO we have expanded, through consistent delivery of studies in complex areas. Our operations are based in the United Kingdom, France, USA, Germany and Italy. We have operational staff across mainland Europe (Western, Eastern & Central) and throughout North America.

We have in-depth experience of the most challenging therapeutic areas including rare and orphan diseases, Oncology, respiratory, dermatology, vaccines and anti-infectives. We are a boutique CRO offering a true full service with an international reach, combined with the level of quality, flexibility and customer service that you would associate with a company whose mission is to:

“Use our skills elegantly to design, execute and deliver our clients’ clinical development needs.”

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