Euro Roundup: Pharma reform could cause loss of 3 new orphan drugs a year, report claims

The European Commission’s plan to reform pharmaceutical legislation could cause three fewer rare disease drugs to come to market each year, according to a report commissioned by pharma trade group EFPIA.

Dolon, a strategic pricing and market access consultancy that specializes in rare diseases, deployed risk-adjusted net present value (rNPV), a model used by drug developers to inform investment decisions, to analyze how changes to pricing, reimbursement and regulation may affect spending decisions at pharma companies.

In the report, Dolon revised a base case model it created in 2020 and assessed the impact of plans to introduce a cap on the validity of an orphan designation and change orphan market exclusivity.

The revised base case, which Dolon said better reflects the current economic picture for orphan drugs, suggests the investment case for rare disease R&D is more marginal than previously thought. Factoring in the planned pharma legislation reforms further weakens the case.

According to Dolon, shortening the exclusivity period for orphan drugs and putting a seven-year limit on the validity of orphan designations would decrease innovation based on European incentives by 12%. The Commission has estimated that the EU will approve 375 orphan drugs from 2020 to 2035. Based on that figure, Dolon calculated the reforms could cause 45 fewer rare disease therapies to come to market over the 15-year period.

“Losing about a fifth of future orphan medicines represents a significant dip in innovation and contrasts with the Commission’s ambition to boost therapeutic options for rare disease patients, as well as the Union’s aspiration to remain a leading industrial hub,” according to the Dolon report. The pace of innovation may remain the same if other countries continue to support rare disease R&D, but “European patients would depend on the goodwill of other countries – goodwill that may be eroding.”

A second scenario assessed the potential impact of more extensive regulatory updates. If the EU were to reduce exclusivity further and restrict eligibility to orphan designations for products with multiple indications, the consultancy expects innovation to reduce by 36% compared to the base case. The figure translates into an estimated 135 fewer rare disease products from 2020 to 2035.

Publication of the report coincided with the release of other notices and reports about the planned EU pharma reforms. The European Parliamentary Research Service (EPRS) published a report about the Commission’s assessments of the impact of the reforms. EPRS concluded the impact assessments appear “solid in substance, underpinned by a seemingly sound evidence base.”

The off-patent trade group Medicines for Europe updated its position, too, calling for the EU to prioritize five improvements that will support the creation of a streamlined, digital regulatory framework and lead to equitable access to medicines. Medicines for Europe’s priorities include simplifying the framework to reduce delays and investing in a digital, interconnected network of medicine agencies.

Dolon Report, EU Report, More

Commission discusses MDR transition for devices without a medical purpose

The European Commission has answered questions about how the transitional provisions of the Medical Device Regulation (MDR) applies to certain products without an intended medical purpose.

An annex of MDR covers products such as dermal fillers that have a similar function and risk profile to medical devices but have an aesthetic or other non-medical purpose. The devices, which were previously unregulated, were included in MDR to introduce manufacturing and surveillance requirements. Common specifications (CS) for risk management of Annex XVI products have applied since June.

In a new question-and-answer document, EMA explains that MDR has applied to the products since the CS took effect in June. The CS had a 6-month implementation period, which began in December, and includes transitional provisions for specific cases when more time is needed to conform to MDR.

“Such cases include when a notified body needs to be involved in the conformity assessment procedure, when the manufacturer considers carrying out a clinical investigation followed by a conformity assessment procedure that involves a notified body, and when the Annex XVI product is covered by an MDD certificate that is no longer valid,” the Commission wrote.

Subsequent questions and answers go into more detail about the specific situations when a longer transition applies. As the Commission explains, the transition period for when a notified body needs to be involved in the conformity assessment procedure, and the manufacturer decides to carry out a clinical investigation runs until the end of 2029.

Commission Q&A

HPRA clarifies oversight of medtech industry as Irish pediatric spinal surgery scandal grows

The Health Products Regulatory Authority (HPRA) has clarified its role in the oversight of medical devices amid an escalating scandal about the use of implants in pediatric surgeries at a hospital in Ireland.

An independent investigation found 19 children with spina bifida suffered serious complications after a single surgeon operated on them using an implant know as compression strings. One child was readmitted to the operating theater 33 times. A top health official said the implants were “unauthorized, uncertified,” leading HPRA to put out a statement about what it does and does not do in relation to medical devices.

“The HPRA does not approve or certify medical devices for sale in Ireland or any other EU member state,” the agency wrote. “The HPRA's role is in relation to monitoring the safety of CE marked medical devices is activated once a medical device is placed and becomes available on the Irish market.”

HPRA added that, “at this point,” it understands compression springs reportedly used in the pediatric procedures “were not intended to be sold or supplied as medical devices.” The agency does have a role in approving clinical trials, but that requires a sponsor to seek authorization. HPRA never received a filing to study compression springs at the hospital.

HPRA Notice, The Guardian

EDQM updates policy on the acceptability of CEP applications for sterile active substances

The European Directorate for the Quality of Medicines and HealthCare (EDQM) has updated its policy on the acceptability requirements of applications for sterile active substances.

Under the old policy, acceptability requirements only applied to the manufacturing process when the producer of a non-sterile active substance carried out the sterilization operations in-house. Sterilization operations are required to obtain the sterile grade.

Now, the requirements will also apply when the active substance sterilization process is outsourced to an external manufacturer and when a crude or non-sterile substance is bought from an external supplier and “subjected to purification and/or salt formation before sterilization.” The change applies to new Certification of Suitability (CEP) applications and requests for revision of existing CEPs.

EDQM Notice

Other news:

The Swiss Agency for Therapeutic Products (Swissmedic) has published the results of a medical device survey. The survey gathered more than 450 responses about the Swiss Database on Medical Devices to inform the development of the module for registering medical devices. Swissmedic Report

Euro Roundup: Pharma reform could cause loss of 3 new orphan drugs a year, report claims

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