Early stakeholder dialogues crucial for success of rare orphan medicines

By ensuring all voices are included early in the process, this streamlines the development of the product while also serving to increase transparency and build trust among the respective groups. [SHUTTERSTOCK]

Early dialogue between stakeholders is crucial for the effective development and success of rare orphan drugs, used to treat patients with rare diseases, Anna Bucsics, project adviser at the mechanism of coordinated access to orphan medicinal products (MoCA), told EURACTIV in an interview.

“Nobody wants to develop a drug that is not able to be used or to be sold. The earlier you find out what is needed, the easier it is for everyone to make important decisions and to ensure that the drug is fit for purpose,” she said, adding that dialogue also serves to increase transparency and build trust among the respective groups.

Around 246,000 people suffer from rare diseases across the EU. 

MoCA is a voluntary coordinated mechanism which works to facilitate dialogue between key stakeholders, including member states, orphan medicine developers, payers and patients, to evaluate the technical aspects of an orphan medicinal product, including distribution, logistics and pricing.

By working to support this exchange of information, MoCA aims to enable informed decisions on pricing and reimbursement at the member state level.

It is particularly important to facilitate this kind of discussion early on in the process of developing an orphan drug, Bucsics stressed.

“Bringing all involved parties around the table to discuss a new orphan product in the early stages of development is crucial given that these treatments often pose more varied problems than others and can be particularly difficult to deal with,” she said.

She added that this is also important for developing treatments for other diseases given that these orphan products often serve as a “canary in the coalmine” for other diseases.

This is because orphan medicinal products (OMPs) are usually at the cutting edge of innovation, and can therefore offer insights into problems that other medicines may encounter later in development.

Bucsics added that OMPs also often serve as a model for other new treatments, such as new cancer drugs.

Reviewing orphan drugs law: A tough equation

The Orphan Regulation was introduced in 2000 and its main objective was to address the challenge of regulating medicines which treat patients with rare diseases.

Around 246,000 people suffer from such diseases across the EU,

The European Commission is expected to present …

Patients few and far between

But bringing all involved parties is particularly challenging for patients, Bucsics stressed. 

“It is very valuable to have patients’ input and support early in the discussions, as this allows companies to take their concerns into consideration,” she said, adding that patients that are involved early on in the procedure are also more likely to engage with patient-reported outcomes later.

Patient-reported outcomes are defined as any report of the status of a patient’s health condition that comes directly from the patient, without interpretation by a clinician or anyone else, and these are a key tool to measure the effectiveness of a drug.

But getting enough patient input is a challenge in the case of rare orphan drugs given that the pool of patients is often very small and patients are often spread across Europe.

Payer participation a ‘challenge’

Equally, ensuring payers participation in these dialogues presents a major challenge, but those are “vital voices” in the conversation which ensure discussions about pricing and reimbursement happen at an earlier point in time.

But there is currently a reluctance to get involved in such discussions. 

One of the reasons is that there are often concerns that payers will be held to what they say, according to Buscics, driving concerns that early constructive dialogue could later turn into constraints.

But Buscics stressed that these early dialogues are not binding and are “completely informal”, adding that this is something that must be made clearer to participants.

“People are worried that in these discussions, they will be held to whatever they say, and that this could have repercussions later down the road. But the European Medicines Agency makes it very clear that all advice is not binding,” she emphasised. 

Another reason for a low level of participation among payers is because they often do not have enough resources to participate, as this is technically “outside their remit”.

One way this could be alleviated is by holding discussions virtually, making it easier, cheaper and less time-consuming for everyone to be able to attend discussions and thus encouraging a wider variety of voices to the table. 

Data sharing critical to close 'innovation blackhole' for rare diseases

Access to data and ‘real world evidence’ obtained from observational data is needed to help push research and innovation on rare diseases, with support needed to encourage and harmonise this across Europe, stakeholders have said. 

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