Updated for 2020: Development of the Orphan Drug Sector

The orphan drug pipeline continues to grow and the sector has attracted considerable interest.  We have updated this article for 2020 with updated statistics.

Much has been written about orphan drugs in recent times.  These products, developed specifically to treat a rare disease, have become more common in the marketplace.  At the time of writing (May 2020), there are almost 770 orphan drugs commercially available and more than 5600 orphan designated drugs in the biopharmaceutical pipeline.

As the sector continues to grow there has been a tendency for facts about orphan drugs to get lost in the midst of issues concerning specific patients’ needs, pricing and availability.  We look at some facts about orphan designation that companies considering entering the sector should be aware of.

We work with a number of innovative companies in this space and we are always happy to discuss issues relating to the orphan drugs sector with companies considering their next move.

 10 Facts on Orphan Designation

  • Obtaining Orphan Designation early in the development process encourages the development of medicines for rare diseases as it offers incentives for development.
  • Once an orphan medicine is authorised, it will qualify for 10 years market exclusivity. The market exclusivity becomes effective once the Manufacturing Authorisation (MA) is granted. This blocks similar competitive medicines from entering the market for 10 years. However, it does not necessarily block medicines that are targeted at the same disease if they differ from the first medicine on the basis of their molecular characteristics, the way they work or the way they are used. Also, in some circumstances, even a similar medicine could be marketed if, for example, it is manufactured so that it works better than the first medicine.
  • Obtaining Orphan Designation does not necessarily speed up the development time or the evaluation of the MA application.

Can The Drug Demonstrate ‘Significant Benefit’?

  • For a medicine to obtain Orphan Designation, it must be demonstrated that the medicine will bring “significant benefit” to the patient when compared to existing treatments for the targeted rare disease. “Significant benefit” should not be confused with the “benefit-risk” assessment carried out for the MA application which demonstrates that benefits from a medicine are outweighed by the risk it poses.
  • The use of biomarkers to identify a subset of patients for whom medicine could be used is generally not accepted for Orphan Designation.

Obtaining Designation Does Not Permit Use

  • The obtaining of Orphan Designation does not in itself permit the use of the medicine. An MA will only be granted once enough evidence has been obtained to demonstrate that the medicine is effective and acceptably safe. In exceptional circumstances, a doctor in consultation with their patient may consider the use of an unauthorised medicine through a compassionate use programme or the patient may have the possibility to enroll in a clinical trial.
  • Medicines are evaluated for Orphan Designation by the Committee for Orphan Medicinal Products (COMP). It is also their responsibility to determine if Orphan Designation medicines can continue to be classified as Orphan medicines at the time of granting MA. This is separate to the evaluation of the MA application which is conducted by the Committee for Medicinal Products for Human Use (CHMP). The final decision on granting of Orphan Designation and MA is the responsibility of the European Commission. COMP publishes their assessment once Orphan status is designated and the Orphan designation is entered into the Community Register of Designated Orphan Medicinal Products.
  • The medical condition specified in the Orphan Designation is often broader than the “indication” approved in the MA as it will be based on evidence obtained during the development process and post-Orphan Designation.
  • Assessment of Orphan medicines is based on scientific assessment of available evidence. The assessment of costs associated with Orphan medicines is the responsibility of National Health Authorities. Individual countries within the EU will decide whether they will provide an Orphan medicine within their country’s health system.

Differences Between Authorities

  • The rules for granting Orphan Designation can vary between different authorities, therefore, a medicine classified as an Orphan in the EU may not necessarily be classified as an Orphan medicine in another jurisdiction. However, the EMA encourages parallel applications for Orphan Designation with regulatory authorities outside the EU. A common application form is available when applying to the EMA and FDA in parallel. The EMA also has a special arrangement in place with the Japanese health authorities. The application to the EMA is free of charge.

This article from The Scientist looks at the commercial issues impacting upon the orphan drugs sector.  It considers the issue of availability and the commercial considerations for companies operating in the space.

Again, if you would like to have an initial chat with us about issues in the orphan drug space then please call us on 00353 52 61 76 706 or simply complete the form below and we will get back to you.

Reference: Rare Diseases, Orphan Medicines Getting the facts straight, EMA/5513338/2017 28. February 2018.