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Rare Diseases February 2019

Regulators and payers are ready to talk about orphan drugs

Daniele Severi Bruni

Vice President, Market Access, Two Labs

Securing market access for orphan drugs is littered with challenges, but early collaboration between manufacturers, regulators and payers is the key to overcoming the obstacles.

Daniele Severi Bruni, Vice President of Market Access at Two Labs (formerly MKO Global Partners), believes there is an appetite for overcoming the challenges of bringing expensive, yet potentially transformative, therapies to market.

“The manufacturers and the scientific community are not the only ones who are excited for cell and gene therapies.

“Others in the industry, like payers and regulators, share the same excitement. They know patients with these diseases suffer and that the impact on families and society is enormous,” he says.

Payers simply have limited resources and in providing funding for any drug, they must rely on robust evidence of its efficacy and safety.

Unfortunately in orphan drugs, randomised controlled trials in large patient populations are all but impossible, meaning data on how they work in the real world is limited. This problem is accentuated by new technologies, such as gene and cell therapy. With these technologies, a large upfront cost is necessary, however the benefit is realised over time.

Huge sums of money for a small group of people

It is on this basis that payers are being asked to allocate huge sums of public money to a relatively small proportion of the population for technology whose actual value is uncertain in the real world.

Daniele says: “I believe in the end, all these drugs will be paid for, but it might be that it takes a very long time to negotiate the price and access conditions.

“In the meantime, you have all these patients with high, unmet needs who are suffering or even dying from rare diseases.”

Manufacturers and agencies must collaborate

The solution is manufacturers taking payers’ decision-making process into account and working with HTAs and regulatory agencies “to find a solution that suits everyone.”

Managed access agreements or innovative pricing contracts, which Daniele compared to offering a warranty or insurance policy on the treatments, could be based on any number of models. Outcome-based pricing and staggered reimbursement schemes are just two examples.

Mechanisms to reduce or dilute the financial burden over time and increase payers’ confidence in what they are paying for exist and should be embraced early on. These mechanisms do not only involve payers and manufacturers, but they should also leverage a partnership with regulatory agencies to ensure a flexible but robust evidence generation process.

For example, the European Medicines Agency’s (EMA) new Adaptive Pathways Programme allows for staged conditional approvals, which could be then linked to some conditional or innovative payment mechanisms. Similarly, the EMA also offers joint consultations with EUnetHTA to manufacturers. These approaches allow pharmaceutical companies to gather the information they need to design their evidence development plan in a way that works for both regulators and HTAs and helps payers understand the challenges that manufacturers face.

Daniele says: “This is potentially very advantageous for all parties involved, including orphan drug manufacturers, because it gives them the opportunity to get early advice and understand what needs to be done to secure approval.”

Reducing the perceived risk

Early collaboration with payers and regulatory agencies is important not only to define a reasonable evidence generation programme, they are also fundamental in thinking how to de-risk these innovative technologies for payers.

”While innovative access agreements or innovative payment mechanisms are the potential solution, with many such agreements, the ‘devil is in the detail.'”

He added: “The biggest risk for manufacturers is moving from a risk-sharing to a risk-taking arrangement. Innovative access agreements and innovative payment mechanisms must be designed in parallel with the evidence generation plan and discussed with payers early on. This will ensure that the manufacturer is able to provide an adequate level of insurance for payers, without taking on board the entire risk. These contracts need to reflect the actual value of the therapeutic intervention and, most importantly, they need to be financially sustainable for the manufacturer.

“Ultimately, bringing this technology to the market requires much more planning and much earlier on than for traditional drugs. However, good planning and a collaborative approach will benefit everyone: manufacturers, regulatory agencies, payers and, most importantly, people living with rare diseases.”

If you are a manufacturer working on a rare disease drug and want to learn more about finding a solution that works for payers, please contact Daniele Severi Bruni at [email protected] or visit to learn more.

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