For academic research to successfully translate into a commercially viable drug or device, clinical trials and development processes need to be smart from the outset and ideally created by a multidisciplinary team of scientists, health economists and market access specialists.

In pharmaceutical and medical device product development, quality clinical data relevant to the disease condition are of major importance and two major hurdles need to be overcome:

Regulatory Approval

Regulatory authorities, such as the European Medicines Agency (EMA) and Food and Drug Administration (FDA), will assess if a product is safe and efficient based on efficacy and safety data from controlled clinical trials in human subjects. The authorities will grant market authorisation for a given medical innovation in a specific patient population if the risk/benefit parameters are met. A trial design which meets the expectations of regulatory bodies is therefore critical to success and early dialogue with regulatory agencies is mandatory.

Market Access

Following regulatory approval, each country carries out a health technology assessment (HTA) which ultimately leads to a price agreement and reimbursement decision. The clinical trial data used to obtain regulatory approval forms the basis of the HTA submission, but these data do not always correspond to payer needs:

  • The comparator in the clinical trial, which may be the current standard treatment, needs to be relevant and available on the market
  • Outcome parameters (primary and secondary) need to be clinically relevant
  • Patient-reported outcome (PRO) data needs to be included to enable a cost-effectiveness analysis (CEA)
  • Clinical follow-up and time horizons need to be well chosen according to disease specific criteria
  • An assessment of the medical resources used in clinical practice alongside the clinical trial can also assist with the budget impact analysis and general health economic research

clinical trial program must, therefore, not only assess the efficacy and safety of product X against product Y, but also be designed in such a way that payer needs are met through good HTA and the avoidance of medical uncertainties.

In recent years, HTAs have often led to innovative managed entry agreements (MEA). These contractual agreements are put in place between the marketing authorization holder and the healthcare payer to offset any clinical or economic uncertainty about the drug or device. As payers are more and more reluctant to pay for every patient, they will often ask the promotor for a risk-sharing agreement (RSA) or request a pay for performance (P4P) contract based on real-world evidence (RWE).  RWE has emerged as a relatively new discipline in clinical research. It is designed to inform payers about the clinical benefit of a new technology and to inform payment mechanisms such as P4P agreements.

Today’s clinical research is far more complex than before. Placebo-controlled clinical trials, considered once to be the gold standard, are no longer sufficient. A broad stakeholder network with different needs and expectations has turned clinical trial design into a strategic decision-making process. It is now clear that early dialogue with regulatory and payer agencies will guide future choices when it comes to developing new technologies. It is therefore extremely important that companies come to the table with an open and future-oriented mindset as new payment mechanisms will demand specific data for each disease and therapeutic domain. In situations where there is a lack of long-term data, or only small patient numbers.

In clinical trials (e.g. orphan drugs), payers become wary. Companies need to be well-prepared and supply an evidence-generating reimbursement proposal.

HCC is a new generation market access consulting company. By partnering with AESCULAPE CRO, we are able to conduct state-of-the-art clinical trials, registries, observational studies and real world outcomes research and combine this with our in-house market access expertise. In parallel with continuous vigilance to meet regulatory and payer needs, we create the data to support successful market access and commercial success.

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