Rare Disease & Orphan Drug Development – Cost-efficient trial design to minimise cash burn

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.

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