EMA Tries to Assuage Professors on Assumptions Linked to Adaptive Pathways Pilot

The European Medicines Agency Director (EMA) Executive Guido Rasi and high medical Hans-Georg Eichler sent a letter Thursday to clarify some assumptions put in doubt by a group of nine teachers That criticize aspects of track program pilot adaptation of the agency.

Background

First announcement in March 2014, the EMA pilot program seeks to accelerate patient access to medicines for patients with urgent medical needs and then of approval for a strong indication if waiting That clinical data and the additional real world That is used for assess if treatment is really as effective as expected. From November 2015, the EMA has selected 19 proposals to participate in the pilot program.

As Rasi there Eichler describe the of pilot adaptation "way seeks to maximize the positive effect of new drugs balancing timely access to That patients might benefit more from a new drug with the need for adequate information sober evolution benefits there risks . "

Three companies participating include Bluebird Bio, in 2015. That said May that it intends to request the conditional approval for gene therapy to treat beta-thalassemia LentiGlobin BB305 greater; September biotechnology company said in 2015 Immunocore also plans to seek approval conditional Addition lead IMCgp100 biological treatment of patients with metastatic uveal melanoma, a rare and fatal disease with few options available treatment; there August 2015, Pluristem Therapeutics, indicate if found using the guide EMA in the pilot to develop a cellular therapy product That heads a sub-group of patients with critical ischemia of the above tips of look you expand to other sub-groups of patients.

assumptions

In May, nine professors of Oxford, Cambridge, the Medical School London Hygiene and Tropical National French Academy of Medicine, Royal College of Physicians, Toronto, York University, as well as other Barcelona and Italy Copenhagen raised questions with some serious regarding the assumptions made by the development in the EMA pilot ways of adaptation.

"The evidence if based in these cases does not seem very convincing for us and it seems that there is a lot of uncertainty in their potential operational implementation" teachers wrote, noting a list of eight General assumptions, including:

• "New" or "innovative" drugs and biologicals STI more effective and safer That That current
• Current mechanisms of regulation postmarket suffocate market and the market entry and innovation retardation and "new" drugs "Innovator"
• Enter the market on time is beneficial for society
• To that patients have run you will return in a Town medication if the new one just failing regulatory obstacles or later you have marketing and doctors That sober act such subsequent warnings based sober marketing or use restrictions damages
• EU information systems can support the adaptation process with impartial way (or minimally slanted) a day as observation data information
• His acceptable alternative outcomes
• The notion of That "something is better than nothing" is an acceptable principle

And although teachers think That said in the notion of the way of adaptation has "great potential to benefit society," That also claim scientific terms "should be used and correctly, there's possibility That is a misinterpretation. The term "real-world evidence" are a euphemism for observation tests, is That comes from observations That experimentation has always precede production of empirical evidence there. "

They also say that before the use of a means of adaptation leads to the authorization of a new drug, "any subsequent plane to generate evidence must be agreed there legally binding on all parties Following Agreed protocol. This is should you need to ensure of the surrender of accounts by substantial sums of public money they have been and will That invested invested ".
EMA response

In response to the letter, there Eichler Rasi offer point by point summary if agree with the teachers there try of provide more support for adaptation That way form of works and moving forward will work in.

In the event of the "new" or "innovative" drugs could give the impression of That these more effective or safer That existing STI treatments, EMA says that the terms (new and innovative) That said His synonyms his "neutral" respect of a product if innovative or new more or less is given effective or safe That existing treatments.

"The aim of the ways of adaptación're lead to potentially beneficial treatments for the group of patients right as soon as appropriate" officials wrote. "Therefore, evaluation probable of sober benefit the existing treatment options heading of" novelty "must precede has decision to follow paths of adaptation. Note the EMA received 60 requests from sponsors its pilot program pathways of adaptation course, but we selected only 20. Our goal is to '' choose the winners, in the interest of patients No. unmet medical needs. "

And although officials clarified That "real-world evidence" art used by EMA "to refer you evidence from records, electronic health records (EHR) and insurance data, whether in studies specific observation or monitoring continuous use, the risks benefits there, "they also say That" his conscious That observational studies have produced results not reproducible or contradictory, however, also have done other methodologies, including RCTs [randomized clinical trials therefore the concept. pathways of adaptation makes Insisting in the necessity of harvesting planned the observation data, where it may be necessary to supplement trials testing. This collection is based on the advice of experts there Methodologic input of multiple stakeholders. on the other hand, repeated cycles of generation is quickly testing to refine or emphasize decisions when necessary right past. "

And as to be able to change a patient's drug again when the new one Town could become less effective, EMA recognizes the difficulties such a scenario.

"We are conscious disappointing experiences in the past, in fact, doctors have not always acted on post warnings to damage the sober marketing and use restrictions" Rasi there Eichler write. "However, recent experience with flat designed Prospectiva risk sample That is if properly managed, restrictions / warnings can be successful."

In the event of That "something is better than nothing" officials also make clear That "would be unethical and incompatible with the regulatory role of" selling hope rather than aid "[Bianco and Sipp Nature 2014. 510, 336- 337]. This is why special way of adaptation makes Insisting in early enough relevant evidence of patient benefit there a strict control of patient benefit after launch ".